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BACKGROUND: Pruritus is a common condition in chronic kidney disease (CKD), especially for patients receiving haemodialysis. CKD-associated pruritus (CKD-aP) can be distressing and have a negative impact on quality of life (QoL). This post hoc analysis aimed to assess the relationship between pruritus relief and QoL. METHODS: Data from phase 3 trials [(NCT03422653, NCT03636269 grouped), and NCT03998163] of the novel antipruritic difelikefalin (N = 914) were used to assess the relationship between reductions in pruritus intensity at Week 12 (24-h Worst Itching Intensity Numeric Rating Scale; WI-NRS), perceived improvement in itch (Patient Global Impression of Change, PGI-C) and pruritus-related QoL (Skindex-10 questionnaire). RESULTS: Patients receiving difelikefalin had greater improvements in Skindex-10 total scores than those receiving placebo [LS mean treatment difference -3.4; 95% confidence interval (CI) -5.5, -1.3; P = .002] and greater improvements across Skindex-10 domains (disease, mood and social functioning) at Week 12. In patients receiving difelikefalin, those with clinically meaningful improvements in pruritus (≥3-point reduction in WI-NRS score) at Week 12 had a greater improvement in Skindex-10 total score (mean difference 14.2; 95% CI 11.0, 17.3; P

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes. Children (2-19 years) with genetically confirmed CDD and ≥ 16 major motor seizures per month were enrolled in a double-blind randomized placebo-controlled trial. Ganaxolone or placebo was administered three times daily for 17 weeks. Behaviour was measured with the Anxiety, Depression and Mood Scale (ADAMS), daytime sleepiness with the Child Health Sleep Questionnaire, and quality of life with the Quality of Life Inventory-Disability (QI-Disability) scale. Scores were compared using ANOVA, adjusted for age, sex, number of anti-seizure mediations, baseline 28-day major motor seizure frequency, baseline developmental skills, and behaviour, sleep or quality of life scores. 101 children with CDD (39 clinical sites, 8 countries) were randomized. Median (IQR) age was 6 (3-10) years, 79.2 % were female, and 50 received ganaxolone. After 17 weeks of treatment, Manic/Hyperactive scores (mean difference 1.27, 95%CI -2.38,-0.16) and Compulsive Behaviour scores (mean difference 0.58, 95%CI -1.14,-0.01) were lower (improved) in the ganaxolone group compared with the placebo group. Daytime sleepiness scores were similar between groups. The total change in QOL score for children in the ganaxolone group was 2.6 points (95%CI -1.74,7.02) higher (improved) than in the placebo group but without statistical significance. Along with better seizure control, children who received ganaxolone had improved behavioural scores in select domains compared to placebo.

Context: The purpose of this study was to compare short-term clinical outcomes between meniscus procedures performed with anterior cruciate ligament reconstruction (ACLR), ACLR (ACLR-only), ACLR with meniscectomy/resection (ACLR-resect), and ACLR with meniscal repair (ACLR-repair) for bone patellar tendon bone grafts (BPTB) and hamstring tendon grafts, separately. Design: This was a cross-sectional study conducted in a controlled laboratory setting as part of a large point-of-care collaborative research program. Methods: This study included 314 participants (168 females; mean [SD]: age, 19.7 [4.8]) with primary unilateral ACLR with a BPTB or hamstring tendon. Patients were divided into 3 groups depending on meniscal procedure (ACLR-only, ACLR-resect, and ACLR-repair). Postsurgical testing included: isokinetic assessment of knee extension and flexion, single-leg hop tests, and patient-reported outcomes. Multivariate analysis of covariance compared differences between meniscal procedures on the battery of tests, and for each statistically significant variable an analysis of covariance assessed the effect of meniscal procedure within each graft type. Chi-square analysis assessed the influence of meniscal procedure on tests' pass rates defined as 90% of limb symmetry index. Results: BPTB: ACLR-only had greater hamstring strength than ACLR-resect (P = .05) and ACLR-repair (P = .005). ACLR-only had the highest proportion of participants to pass the hamstring strength test (P=.02). amstring tendon: ACLR-only (P=.03) and ACLR-resect (P=.003) had higher International Knee Documentation Committee scale scores than ACLR-repair. There was a significant difference in the proportion of participants who scored >90% limb symmetry index on the timed hop test (P = .05). Conclusions: The influence of meniscal repair on clinical outcomes is dependent on the graft choice. Following an ACLR with BPTB and a meniscal procedure, hamstring function should be more closely monitored for optimal short-term recovery. [ABSTRACT FROM AUTHOR]

BACKGROUND: Following a breast cancer diagnosis, it is uncertain whether womens breast density knowledge influences their willingness to undergo pre-operative imaging to detect additional cancer in their breasts. We evaluated womens breast density knowledge and their willingness to delay treatment for pre-operative testing. METHODS: We surveyed women identified in the Breast Cancer Surveillance Consortium aged ≥ 18 years, with first breast cancer diagnosed within the prior 6-18 months, who had at least one breast density measurement within the 5 years prior to their diagnosis. We assessed womens breast density knowledge and correlates of willingness to delay treatment for 6 or more weeks for pre-operative imaging via logistic regression. RESULTS: Survey participation was 28.3% (969/3,430). Seventy-two percent (469/647) of women with dense and 11% (34/322) with non-dense breasts correctly knew their density (p

AIM: This paper describes the trajectory during 1 year of four patient-reported outcomes (PROs), namely, sleep, depressive symptoms, health-related quality of life (HrQoL), and well-being, in patients with heart failure (HF), their relationship and the patient characteristics associated with changes in these PROs. METHODS AND RESULTS: Data analyses of PROs from 603 patients (mean age 67 years; 29% female, 60% NYHA II) enrolled in the HF-Wii study. On short term, between baseline and 3 months, 16% of the patients experienced continuing poor sleep, 11% had sustained depressive symptoms, 13% had consistent poor HrQoL, and 13% consistent poor well-being. Across the entire 1-year period only 21% of the patients had good PRO scores at all timepoints (baseline, 3, 6, and 12 months). All others had at least one low score in any of the PROs at some timepoint during the study. Over the 12 months, 17% had consistently poor sleep, 17% had sustained symptoms of depression, 15% consistently rated a poor HrQoL, and 13% poor well-being. Different patient characteristics per PRO were associated with a poor outcomes across the 12 months. Age, education, New York Heart Association, and length of disease were related to two PRO domains and submaximal exercise capacity (6 min test), co-morbidity, and poor physical activity to one. CONCLUSION: In total, 79% of the patients with HF encountered problems related to sleep, depressive symptoms, HrQoL, and well-being at least once during a 1-year period. This underscores the need for continuous monitoring and follow-up of patients with HF and the need for dynamic adjustments in treatment and care regularly throughout the HF trajectory.

Self-report data are essential in health psychology research where an individual's perception is critical to understanding one's health and psychological status. Intensive data collection over time, including daily diary assessments, is necessary in understanding within- and between-person variability in health and psychological processes over time. An "initial elevation or latent decline" (IELD) effect, inherent of self-report data, is increasingly acknowledged in the social psychology literature, but awareness of this effect in health psychology research is lacking, particularly in studies that emphasize within- and between-person variability in self-reports. The IELD effect is a pattern in which responses tend to be more extreme at the initial interview relative to subsequent responses. This paper illustrates the impact of IELD in applications of mixed-effects models based on observational self-reports and concludes that researchers take such effects into account in data analysis or in the research designing phase to help mitigate such effects.

INTRODUCTION: The 2021 US approval of ruxolitinib cream for treatment of atopic dermatitis (AD) in patients aged ≥ 12 years was based on the results of two pivotal phase 3 studies. Currently, real-world data to describe effectiveness of ruxolitinib cream and physician satisfaction with treatment remain limited. Our objective is to describe disease control among adults with mild to moderate AD prescribed ruxolitinib cream and physician satisfaction with treatment. METHODS: Data were from the Adelphi AD Disease Specific Programme™, a US real-world, cross-sectional survey of physician-reported data, undertaken between August 2022 and March 2023. For patients aged ≥ 18 years, physicians reported patient demographics, clinical characteristics, treatment patterns, and physician satisfaction with disease control. Descriptive analysis of data for patients with mild to moderate AD prior to the initiation of ruxolitinib cream and treated with ruxolitinib cream for ≥ 1 month was undertaken. RESULTS: Among physician-reported data from 1360 patients with AD, 149 patients had received ruxolitinib cream (in combination or as monotherapy) for ≥ 1 month, including 59 patients receiving monotherapy. Prior to treatment with ruxolitinib cream, 84.6% of patients had moderate AD (Investigators Global Assessment, IGA of 3), whereas after treatment (median duration, 26 weeks), only 21.5% had an IGA of 3, with 48.3% of patients having clear or almost clear skin (IGA of 0/1). For these patients, 81.2% were not currently experiencing a flare, and physicians were satisfied with disease control for 87.3%. Results were similar in patients receiving monotherapy. The most frequent physician-reported reasons for prescribing ruxolitinib cream included relieving itch, improving lesion redness/thickness, achieving disease control, and reducing/controlling flares. CONCLUSIONS: These real-world findings demonstrate effective disease control and physician satisfaction with ruxolitinib cream for the treatment of AD in adults in a clinical practice setting. Outcomes were similar whether ruxolitinib cream was prescribed as monotherapy or in combination regimens, suggesting a role for ruxolitinib cream across the spectrum of disease.

Context: An easy-to-administer, function-based questionnaire to assess patients with various foot problems was required for a Greek-speaking population. This study aimed to translate and cross-culturally adapt the Quick Foot and Ankle Ability Measure (Quick-FAAM) into Greek and evaluate its reliability and validity. Design: Cross-sectional study. Methods: Established international guidelines for the cross-cultural adaptation of questionnaires were followed. The face and content validity of the Greek version of the Quick-FAAM (Quick-FAAM-GR), as well as the internal consistency and test-retest reliability upon repeated administration after 5 days, were examined. In addition, the construct validity of the scale was examined via exploratory factor analysis as well as by testing for associations with the Manchester Foot Pain and Disability Index, the 12-item Short-Form Survey (version 2), and a functional balance assessment test--the Y-Balance Test. Results: Sixty participants (18 women) with self-reported chronic ankle instability symptoms, with a median (interquartile range) age of 27 (7.7) years, participated in the study. Half of the participants were included in the test-retest reliability study. The Quick-FAAM-GR demonstrated face and content validity. Excellent internal consistency (Cronbach a =.961) and intrarater test-retest reliability (intraclass correlation coefficient ICC[2,1] = .93) were demonstrated, with a comparable error margin to the original version (standard error of the measurement = 2.1, 95% minimum detectable change = 5.9). Associations of the Quick-FAAM-GR scores to other questionnaires ranged from weak to strong (Spearman rho), all being statistically significant (Manchester Foot Pain and Disability Index from -.26, P = .04, to -.67, P< .001, and 12-item Short-Form Survey, version 2, between .41 and .72, P<.001), and to the Y-Balance Test between lower-limb differences (-.35 to -.58, P< .001). The exploratory factor analysis confirmed the single- factor structure of this scale. No floor/ceiling effects were observed. Conclusions: The Greek Quick-FAAM has proven to be a valid and reliable tool for evaluating chronic ankle instability and can be used for clinical and research purposes in Greek- speaking individuals. [ABSTRACT FROM AUTHOR]

Heart failure (HF) is characterized by a progressive clinical course marked by frequent exacerbations and repeated hospitalizations, leading to considerably high morbidity and mortality rates. Patients with HF present with a constellation of bothersome symptoms, which range from physical to psychological and mental manifestations. With the transition to more advanced HF stages, symptoms become increasingly more debilitating, interfere with activities of daily living and disrupt multiple domains of life, including physical functioning, psychological status, emotional state, cognitive function, intimate relationships, lifestyle status, usual role activities, social contact and support. By inflicting profuse limitations in numerous aspects of life, HF exerts a profoundly negative impact on health-related quality of life (HRQOL). It is therefore not surprising that patients with HF display lower levels of HRQOL compared not only to the general healthy population but also to patients suffering from other chronic diseases. On top of this, poor HRQOL in patients with HF becomes an even greater concern considering that it has been associated with unfavorable long-term outcomes and poor prognosis. Nevertheless, HRQOL may differ significantly among patients with HF. Indeed, it has consistently been reported that women with HF display poorer HRQOL compared to men, while younger patients with HF tend to exhibit lower levels of HRQOL than their older counterparts. Moreover, patients presenting with higher New York Heart Association (NYHA) functional class (III-IV) have significantly more impaired HRQOL than those in a better NYHA class (I-II). Furthermore, most studies report worse levels of HRQOL in patients suffering from HF with preserved ejection fraction (HFpEF) compared to patients with HF with reduced ejection fraction (HFrEF) or HF with mildly reduced ejection fraction (HFmrEF). Last, but not least, differences in HRQOL have been noted depending on geographic location, with lower HRQOL levels having been recorded in Africa and Eastern Europe and higher in Western Europe in a recent large global study. Based on the observed disparities that have been invariably reported in the literature, this review article aims to provide insight into the underlying differences in HRQOL among patients with HF. Through an overview of currently existing evidence, fundamental differences in HRQOL among patients with HF are analyzed based on sex, age, NYHA functional class, ejection fraction and geographic location or ethnicity.

Conventionally, peripheral nerve stimulation (PNS) for treatment of chronic pain has involved a two-stage process: a short-term (e.g., 7 days) trial and, if significant pain relief is achieved, a permanent PNS system is implanted. A percutaneous PNS treatment is now available where a coiled lead may be implanted for up to 60 days with the goal of producing sustained relief. In the present review, published prospective trials using percutaneous PNS treatment were identified and synthesized. The collected evidence indicates that percutaneous PNS treatment for up to 60 days provides durable clinically significant improvements in pain and pain interference. Similar efficacy across diverse targets and etiologies supports the broad applicability for use within the chronic pain population using this nonopioid technology.

PURPOSE: To compare the outcomes of the Tecnis Eyhance ICB00 IOL, designed to enhance intermediate vision, to a conventional Tecnis Monofocal ZCB00 IOL. METHODS: This retrospective analysis compared two cohorts of patients undergoing lens replacement surgery with bilateral implantation of the Tecnis ICB00 or the Tecnis ZCB00 IOL (383 patients in each group). Monocular and binocular uncorrected distance (UDVA), intermediate (UIVA; 66cm), and near (UNVA; 40cm) visual acuities, refractive predictability, and patient-reported outcomes were compared. A sub-analysis of patients with the Eyhance IOL was performed to compare patients who achieved bilateral emmetropia to those with mini-monovision. One-month postoperative outcomes were analyzed. RESULTS: Both groups had comparable UDVA outcomes. On average, both monocular and binocular UIVAs were approximately one Snellen line better in patients implanted with Eyhance IOL (monocular UIVA: ICB00 0.23 ± 0.18 logMAR, ZCB00 0.33 ± 0.19 logMAR; binocular UIVA: ICB00 0.18 ± 0.18 logMAR, ZCB00 0.26 ± 0.20 logMAR, p < 0.01). Likewise, the mean UNVA was also one Snellen line better with the ICB00 model (monocular UNVA: ICB00 0.51 ± 0.20 logMAR, ZCB00 0.61 ± 0.18 logMAR; binocular UNVA: ICB00 0.42 ± 0.19 logMAR, ZCB00 0.51 ± 0.22 logMAR, p < 0.01). There was no difference between the two groups in overall satisfaction or visual phenomena. A subgroup of patients who achieved mini-monovision with Eyhance IOL had, on average, one Snellen line better UIVA and UNVA compared to patients with bilateral emmetropia. CONCLUSION: Patients receiving the enhanced monofocal IOL had better intermediate and near vision compared to those receiving the conventional monofocal IOL, with similar levels of patient-reported photic phenomena in both groups.

Background Methods Key results Conclusions and Inferences Cyclic vomiting syndrome is a chronic disorder of gut‐brain interaction that is present in both adults and children. It is characterized by severe nausea, vomiting, abdominal pain, and several non‐GI symptoms. It is also associated with several comorbid conditions such as anxiety and depression, which affect overall health care outcomes.This article delineates treatment principles, encompassing both abortive interventions and prophylactic regimens currently recommended for CVS. However, it underscores a critical concern: the absence of FDA‐approved medications for CVS treatment, with existing therapies relying on retrospective and open‐labeled trials.This article emphasizes the pressing need for the development of CVS‐specific outcome assessment tools to facilitate more accurate evaluation and robust data collection for the future studies. In exploring this deficiency, the manuscript also presents the up‐to‐date data and development that enhances our comprehension of patient‐centric concepts, and the challenges faced in creating CVS‐specific tools, and presents a roadmap for their development. Addressing this gap is crucial for advancing our understanding of CVS and optimizing patient care.This elucidates the current state of CVS management but also advocates for a future where tailored tools enhance our ability to measure and improve the outcomes for individuals with this debilitating disorder. [ABSTRACT FROM AUTHOR]

Introduction: Surgery, even on an elective-basis, often induces significant stress in patients, characterized by preoperative anxiety and heightened stress levels due to anticipation of the unknown. However, the primary objective of preoperative counseling is to mitigate these concerns, particularly when delivered in a structured and comprehensive manner. While previous research has highlighted the beneficial impact of preoperative counseling on patient-reported outcomes, none have specifically explored the implementation of a structured checklist-based approach during counseling sessions. To bridge this gap in the literature, our study aims to investigate the effects of implementing a checklist-based structured counseling approach on patient-reported outcomes following elective surgery. Methods: In this prospective cohort study conducted over one year from January to December 2023, a total of 600 patients undergoing elective surgery across three specialties—orthopedic surgery, general surgery, and urology—were examined. The patients were divided into two groups: an intervention group consisting of 300 patients and a control group with an equal number of patients. The study evaluated three key outcomes—postoperative pain, anxiety, and satisfaction—at three specific time points following surgery: 24 h, 48 h, and 72 h. Results: Patients receiving structured checklist counseling showed significantly lower pain scores (24 h: 6.6 vs. 7.03, p = 0.041; 48 h: 5.62 vs. 6.55, p = 0.029; 72 h: 2.54 vs. 2.90, p = 0.035) and anxiety scores (24 h: 8.58 vs. 9.25, p = 0.039; 48 h: 7.50 vs. 8.45, p = 0.030; 72 h: 4.53 vs. 5.98, p = 0.031), as well as higher satisfaction scores (24 h: 5.99 vs. 5.06, p = 0.043; 48 h: 6.99 vs. 6.02, p = 0.033; 72 h: 9.10 vs. 8.20, p = 0.039) compared to controls. These improvements were consistently significant across all three surgical specialties studied (p < 0.05). Conclusion: The structured checklist-based counseling method proves to be effective and essential. This method is associated with reduced postoperative pain and anxiety levels, along with increased patient satisfaction, when compared to the standard approach. Level of evidence: Prospective non-randomized study, Level II. [ABSTRACT FROM AUTHOR]

Study Design. Multicenter, prospective registry study. Objective. To clarify minimal clinically important differences (MCIDs) for surgical interventions for spinal metastases, thereby enhancing patient care by integrating quality of life assessments with clinical outcomes. Background. Despite its proven usefulness in degenerative spinal diseases and deformities, the MCID remains unexplored regarding surgery for spinal metastases. Patients and Methods. This study included 171 (out of 413) patients from the multicenter "Prospective Registration Study on Surgery for Metastatic Spinal Tumors" by the Japan Association of Spine Surgeons. These were evaluated preoperatively and at 6 months postoperatively using the Face Scale, EuroQol-5 Dimensions-5 Levels (EQ-5D-5L), including the Visual Analog Scale, and performance status. The MCIDs were calculated using an anchor-based method, classifying participants into the improved, unchanged, and deteriorated groups based on the Face Scale scores. Focusing on the improved and unchanged groups, the change in the EQ-5D-5L values from before to after treatment was analyzed, and the cutof f value with the highest sensitivity and specificity was determined as the MCID through receiver operating characteristic curve analysis. The validity of the MCIDs was evaluated using a distribution-based calculation method for patient-reported outcomes. Results. The improved, unchanged, and deteriorated groups comprised 121, 28, and 22 participants, respectively. The anchor-based MCIDs for the EQ-5D-5L index, EQ-Visual Analog Scale, and domains of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression were 0.21, 15.50, 1.50, 0.50, 0.50, 0.50, and 0.50, respectively; the corresponding distribution-based MCIDs were 0.17, 15, 99, 0.77, 0.80, 0.78, 0.60, and 0.70, respectively. Conclusion. We identified MCIDs for surgical treatment of spinal metastases, providing benchmarks for future clinical research. By retrospectively examining whether the MCIDs are achieved, factors favoring their achievement and risks affecting them can be explored. This could aid in decisions on surgical candidacy and patient counseling. Level of Evidence. II. [ABSTRACT FROM AUTHOR]

Background: With over 65,000 new cases per year in Germany, prostate cancer (PC) is the most common cancer in men in Germany. Localized PC is often treated by radical prostatectomy and has a very good prognosis. Postoperative quality of life (QoL) is significantly influenced by the side effects of surgery. One possible approach to improve QoL is postoperative symptom monitoring using ePROMs (electronic patient-reported outcome measures) to accurately identify any need for support. Methods: The PRO-P ("Influence of ePROMS in surgical therapy of PC on the postoperative course") study is a randomized controlled trial employing 1:1 randomization at 6 weeks postoperatively, involving 260 patients with incontinence (≥ 1 pad/day) at six participating centers. Recruitment is planned for 1 year with subsequent 1-year follow-up. PRO-monitoring using domains of EPIC-26, psychological burden, and QoL are assessed 6, 12, 18, 24, 36, and 52 weeks postoperatively. Exceeding predefined PRO-score cutoffs triggers an alert at the center, prompting patient contact, medical consultation, and potential interventions. The primary endpoint is urinary continence. Secondary endpoints refer to EPIC-26 domains, psychological distress, and QoL. Aspects of feasibility, effect, and implementation of the intervention will be investigated within the framework of a qualitative process evaluation. Discussion: PRO-P investigates the effect on postoperative symptom monitoring of a structured follow-up using ePROMs in the first year after prostatectomy. It is one of the first studies in cancer surgery investigating PRO-monitoring and its putative applicability to routine care. Patient experiences with intensified monitoring of postoperative symptoms and reflective counseling will be examined in order to improve primarily urinary continence, and secondly other burdens of physical and psychological symptoms, quality-of-life, and patient competence. The potential applicability of the intervention in clinical practice is facilitated by IT adaption to the certification standards of the German Cancer Society and the integration of the ePROMs survey via a joint patient portal. Positive outcomes could readily translate this complex intervention into routine clinical care. PRO-P might improve urinary incontinence and QoL in patients with radical prostatectomy through the structured use of ePROMs. Trial registration: ClinicalTrials.gov NCT05644821. Registered on 09 December 2022. [ABSTRACT FROM AUTHOR]

Background: Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients' day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipaglucosidase alfa plus miglustat (cipa+mig) with alglucosidase alfa plus placebo (alg+pbo) in adult patients with LOPD over 52 weeks and showed improved motor and respiratory function in patients switching treatment from standard-of-care enzyme replacement therapy (ERT) to cipa+mig at baseline. This study evaluated the impact of cipa+mig on patient-reported outcomes (PROs), including HRQoL in ERT-experienced patients, using data from PROPEL. Methods: PROs evaluated included the Subject's Global Impression of Change (SGIC), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 20a, PROMIS Fatigue Short Form 8a, Rasch-built Pompe-specific Activity (R-PAct), and European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L). The proportions of responders in the cipa+mig arm and the alg+pbo arm were compared via chi-squared or Fisher's exact test (patient-level responder analysis), and least squares (LS) mean differences were calculated for change from baseline at Week 52 of the PRO measures (group-level analysis). Results: At Week 52, patient-level SGIC responder and group-level SGIC analyses favored cipa+mig compared with alg+pbo across all SGIC domains (e.g. 90 vs. 59% responders in the cipa+mig vs. the alg+pbo group for SGIC ability to move around; P = 0.0005; and LS mean difference 0.385; P = 0.02). Similarly, PROMIS Physical Function and Fatigue domains numerically favored cipa+mig in both analyses (e.g. 50 vs. 40% responders in the cipa+mig vs. alg+pbo arm for PROMIS Physical Function; P = 0.37; and LS mean difference 3.1; P = 0.11). R-PAct for both treatment groups was similar in the patient-level responder analysis, but numerically favored alg+pbo in the group-level analysis (35% responders in both arms; P = 0.95; and LS mean difference −0.8; P = 0.48). Self-care, usual activities, and depression/anxiety domains of EQ-5D-5L numerically favored cipa+mig in both analyses (e.g. 20 vs. 12% responders in the cipa+mig vs. alg+pbo arm for EQ-5D-5L self-care; P = 0.54; and LS mean difference −0.108; P = 0.52). Conclusions: Overall, switching treatment from alglucosidase alfa to cipa+mig positively impacted PRO measurements during the double-blind period of PROPEL. Trial registration: NCT03729362; Registration date: November 1, 2018; https://clinicaltrials.gov/study/NCT03729362 Plain English summary: Late-onset Pompe disease (LOPD) is a rare, multisystemic inherited genetic disease that causes glycogen accumulation in muscles and other body organs, leading to muscle weakness and respiratory insufficiency. LOPD significantly impacts patients' day-to-day life. Enzyme replacement therapies (ERT) have greatly improved the lives of patients with LOPD. The first approved ERT for LOPD was alglucosidase alfa (alg). To evaluate the effects of a new treatment (cipaglucosidase alfa+miglustat [cipa+mig]) in adult patients with LOPD, two-thirds of patients were switched from alg to cipa+mig and the remaining patients continued receiving alg (alg+placebo [alg+pbo]). We used patient-reported outcome (PRO) questionnaires (asking patients how they feel) to assess changes in patient health. Groups were similar at baseline. Analyses showed that patients improved following cipa+mig treatment for all domains of the PROs Subject's Global Impression of Change (SGIC; overall physical well-being, effort of breathing, muscle strength, muscle function, ability to move around, activities of daily living, energy level, level of muscular pain) and the Patient-Reported Outcomes Measurement Information System (PROMIS; Physical Function, Fatigue) compared with when treated with alg+pbo. Rasch-built Pompe-specific Activity (R-PAct), a survey evaluating daily activities and social life of patients living with Pompe disease, showed that patients felt similar after cipa+mig and alg+pbo. European Quality of Life-5 Dimensions-5 Response Levels (EQ-5D-5L), a measure of health covering five dimensions, favored cipa+mig in the self-care, usual activities, pain/discomfort and depression/anxiety areas, and alg+pbo for mobility. Overall, changing treatment from alg to cipa+mig positively affects PROs and the patient's general well-being. [ABSTRACT FROM AUTHOR]

Background: Psoriatic arthritis (PsA) is a chronic inflammatory disease that causes pain and fatigue, reduces physical function, and negatively impacts health-related quality of life (HRQoL). In the phase III BE OPTIMAL and BE COMPLETE studies, bimekizumab demonstrated clinical efficacy and meaningful improvements in patient-reported outcome (PRO) measures in biologic disease-modifying antirheumatic drug (bDMARD)-naïve patients, and those who had prior inadequate response/intolerance to tumor necrosis factor inhibitors (TNFi-IR). Objectives: To examine the association between achieving increasingly stringent clinical disease control criteria and improvements in PRO measures in patients with active PsA receiving bimekizumab. Design: Post hoc analysis of two phase III studies. Methods: BE OPTIMAL and BE COMPLETE assessed subcutaneous bimekizumab 160 mg every 4 weeks in bDMARD-naïve and TNFi-IR patients with active PsA. Disease control was assessed using American College of Rheumatology (ACR) response criteria, Minimal Disease Activity, Disease Activity Index for Psoriatic Arthritis, and the composite outcome of ACR50 and 100% improvement in Psoriasis Area and Severity Index. Associations between clinical disease control criteria and PRO measures of pain, fatigue, physical function, and HRQoL were assessed at week 16 and week 52/40 (BE OPTIMAL/BE COMPLETE). Results: Achievement of increasingly stringent clinical disease control criteria was generally associated with sequentially greater improvements in all PRO measures, including pain. At week 52/40, 94.7% of bDMARD-naïve and 97.6% of TNFi-IR patients achieving ACR70 reported ⩾50% improvements in pain from baseline, and the greatest numerical improvements (−48.5 bDMARD-naïve; −54.7 TNFi-IR). This pattern was evident as early as week 16 and sustained when assessed at week 52/40 across the majority of clinical disease control criteria and PRO measures reported. Conclusion: The achievement of increasingly stringent thresholds of disease control was associated with corresponding greater improvements in PROs, for patients receiving bimekizumab treatment, irrespective of prior TNFi use. Trial registration ClinicalTrials.gov: NCT03895203, NCT03896581, and NCT04009499. [ABSTRACT FROM AUTHOR]

AbstractObjectiveMethodsResultsConclusion\nPLAIN LANGUAGE SUMMARYTo evaluate quality of life, migraine disability, and work productivity and activity impairment in patients with migraine who received preventive treatment by comparing standard of care preventive medications and calcitonin gene-related monoclonal antibodies (CGRP mAbs), including galcanezumab alone.This cross-sectional study conducted across the United States (US) and Europe used data from the Adelphi Migraine Disease Specific Programme. Physicians completed record forms for consecutive patients, who then completed self-report forms assessing patient-reported outcomes (PROs) such as quality of life, migraine disability, and work productivity and activity impairment. T-tests, Fisher’s exact test, and Mann–Whitney U test were used for analysis.From May 2022 to June 2023, 557 physicians submitted data for 6723 patients. A total of 4036 patients (US 956; Europe 3080) with a history of preventive treatment were included (>60% female, >80% White, mean [standard deviation] age range, 38.7 [12.8] to 46.3 [12.1]). Patients who received 3+ lines of preventive therapy and were receiving CGRP mAbs (including galcanezumab alone) had enhanced health-related quality of life (HRQoL) compared to those who received standard of care. Similar findings were observed across Europe; however, in the US, there was no significant difference in any PROs.Patients with migraine in the overall population and Europe who received 3+ lines of preventive migraine therapy and were receiving CGRP mAbs/galcanezumab demonstrated enhanced HRQoL compared to those who received standard of care.This study evaluated quality of life, migraine disability, and work productivity in patients with migraine who were treated with standard of care preventive medications or calcitonin gene-related monoclonal antibodies (CGRP mAbs), including galcanezumab alone. The study was conducted across the United States and Europe and included data from 557 physicians and 6723 patients. Results showed that patients with migraine who received 3+ lines of preventive therapy and were receiving CGRP mAbs in overall population and Europe demonstrated enhanced health-related quality of life (HRQoL) compared with those who received standard of care. However, in the US, there was no significant difference in HRQoL and migraine disability. The study concludes that CGRP mAb treatments, especially in patients requiring multiple lines of preventive therapy, are a viable approach for optimal migraine management. [ABSTRACT FROM AUTHOR]

This study compares baseline clinical characteristics, physical function testing, and patient-reported outcomes for patients undergoing primary cytoreductive surgery versus neoadjuvant chemotherapy, with the goal of better understanding unique patient needs at diagnosis. Patients with suspected advanced stage (IIIC/IV) epithelial ovarian cancer undergoing either primary cytoreductive surgery or neoadjuvant chemotherapy were enrolled in a single-institution, non-randomized prospective behavioral intervention trial of prehabilitation. Baseline clinical characteristics were abstracted. Physical function was evaluated using the Short Physical Performance Battery, Fried Frailty Index, gait speed, and grip strength. Patient-reported outcomes were evaluated using Patient-Reported Outcomes Measurement Information System metrics and the Perceived Stress Scale. There were no significant differences in demographics or clinical characteristics between cohorts at enrollment, with the exception of performance status, clinical stage, and albumin. While gait speed and grip strength were lower amongst neoadjuvant chemotherapy patients, there were no significant differences in physical function using the Short Physical Performance Battery and Fried Frailty Index. Patients in the neoadjuvant chemotherapy cohort reported decreased perception of physical function and increased fatigue on Patient-Reported Outcomes Measurement Information System metrics. A larger proportion of patients in the neoadjuvant cohort reported severe levels of emotional distress and anxiety, as well as greater perceived stress at diagnosis. Our findings suggest that patients undergoing neoadjuvant chemotherapy for advanced ovarian cancer present with increased psychosocial distress and decreased perception of physical function at diagnosis and may benefit most from early introduction of supportive care. • Ovarian cancer patients undergoing NACT report decreased perception of physical function compared to those undergoing PCS. • NACT patients report increased emotional distress/anxiety and perceived stress when compared to those undergoing PCS. • NACT patients may benefit most from early initiation of supportive care services. [ABSTRACT FROM AUTHOR]

Aims Methods Results Conclusions The SURMOUNT‐1 trial investigated effects of tirzepatide, a glucose‐dependent insulinotropic polypeptide and glucagon‐like peptide‐1 receptor agonist, on body weight in participants with obesity or overweight. This analysis evaluated changes in patient‐reported outcomes (PROs) assessing physical function, psychosocial well‐being, and overall health aspects of participants' health‐related quality of life (HRQoL) in SURMOUNT‐1.PRO instruments included the Impact of Weight on Quality of Life‐Lite Clinical Trials version (IWQOL‐Lite‐CT), Short Form Survey‐36 version 2 (SF‐36v2) and EQ‐5D‐5L. Scores were analysed by treatment group and by categorical degree of weight reduction group: >0 to <5%, ≥5 to <10%, ≥10 to <20% and ≥20%. Relevant PROs were evaluated for participants with or without physical or psychosocial limitations at baseline, as measured by Patient Global Impression of Status for physical activity (PGIS) and Patient Health Questionnaire‐2 (PHQ‐2), respectively.All tirzepatide groups demonstrated significant improvements in PRO scores versus placebo. There was a consistent trend of incremental PRO improvement with greater degrees of weight reduction, starting from ≥5% weight reduction. Participants achieving ≥20% weight reduction demonstrated the greatest changes from baseline to week 72 (SF‐36v2 Physical Component Summary, 4.60; SF‐36v2 Mental Component Summary, 0.80; IWQOL‐Lite CT Total score, 24.7). Those with baseline physical and psychosocial limitations experienced greater improvements than those without.Tirzepatide treatment was associated with improved HRQoL compared to placebo in people with overweight or obesity. Higher percentages of weight reduction were associated with greater improvements.Clinical trial registration number for SURMOUNT‐1: NCT04184622. [ABSTRACT FROM AUTHOR]

A primary goal in treating obstructive hypertrophic cardiomyopathy (oHCM) is to improve patients' health status: their symptoms, function, and quality of life. The health status benefits of aficamten, a novel cardiac myosin inhibitor, have not been comprehensively described. This study sought to determine the effect of aficamten on patient-reported health status, including symptoms of fatigue, shortness of breath, chest pain, physical and social limitations, and quality of life. SEQUOIA-HCM (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM) randomized symptomatic adults with oHCM to 24 weeks of aficamten (n = 142) or placebo (n = 140), followed by a 4-week washout. The Kansas City Cardiomyopathy Questionnaire (KCCQ) and Seattle Angina Questionnaire 7-item (SAQ7) were serially administered. Changes in mean KCCQ—Overall Summary Score (KCCQ-OSS) and SAQ7—Summary Score (SAQ7-SS) from baseline to 24 weeks and following treatment withdrawal were compared using linear regression adjusted for baseline scores and randomization strata. Proportions of patients with clinically important changes were compared. Among 282 participants, the mean age was 59 ± 13 years, 115 (41%) were female, and 223 (79%) were White. Baseline KCCQ-OSS (69.3 ± 20.1 vs 67.3 ± 18.8) and SAQ7-SS (72.0 ± 21.0 vs 72.4 ± 18.3) were similar between aficamten and placebo groups. Treatment with aficamten, compared with placebo, improved both the mean KCCQ-OSS (13.3 ± 16.3 vs 6.1 ± 12.6; mean difference: 7.9; 95% CI: 4.8-11.0; P < 0.001) and SAQ7-SS (11.6 ± 17.4 vs 3.8 ± 14.4; mean difference: 7.8; 95% CI: 4.7-11.0; P < 0.001) at 24 weeks, with benefits emerging within 4 weeks. No heterogeneity in treatment effect was found across subgroups. A much larger proportion of participants experienced a very large health status improvement (≥20 points) with aficamten vs placebo (KCCQ-OSS: 29.7% vs 12.4%, number needed to treat: 5.8; SAQ7-SS: 31.2% vs 13.9%, number needed to treat: 5.8). Participants' health status worsened significantly more after withdrawal from aficamten than placebo (KCCQ-OSS: −16.2 ± 19.0 vs −3.0 ± 9.6; P < 0.001; SAQ7-SS: −17.4 ± 21.4 vs −2.5 ± 13.3), further confirming a causal effect of aficamten. In patients with symptomatic oHCM, treatment with aficamten resulted in markedly improved health status, including significant improvement in chest pain-related health status, than placebo. (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM [SEQUOIA-HCM]; NCT05186818) [Display omitted] [ABSTRACT FROM AUTHOR]

Purpose: To critically examine the current state of machine learning (ML) models including patient-reported outcome measure (PROM) scores in cancer research, by investigating the reporting quality of currently available studies and proposing areas of improvement for future use of ML in the field. Methods: PubMed and Web of Science were systematically searched for publications of studies on patients with cancer applying ML models with PROM scores as either predictors or outcomes. The reporting quality of applied ML models was assessed utilizing an adapted version of the MI-CLAIM (Minimum Information about CLinical Artificial Intelligence Modelling) checklist. The key variables of the checklist are study design, data preparation, model development, optimization, performance, and examination. Reproducibility and transparency complement the reporting quality criteria. Results: The literature search yielded 1634 hits, of which 52 (3.2%) were eligible. Thirty-six (69.2%) publications included PROM scores as a predictor and 32 (61.5%) as an outcome. Results of the reporting quality appraisal indicate a potential for improvement, especially in the areas of model examination. According to the standards of the MI-CLAIM checklist, the reporting quality of ML models in included studies proved to be low. Only nine (17.3%) publications present a discussion about the clinical applicability of the developed model and reproducibility and only three (5.8%) provide a code to reproduce the model and the results. Conclusion: The herein performed critical examination of the status quo of the application of ML models including PROM scores in published oncological studies allowed the identification of areas of improvement for reporting and future use of ML in the field. [ABSTRACT FROM AUTHOR]

Introduction: Seborrheic dermatitis (SD) is a common, chronic inflammatory skin disease, but the physical and emotional burden of patients with SD experience has not been well characterized. Methods: The Harris Poll conducted online surveys of US patients and healthcare providers (HCPs) from December 2021 to January 2022. Results: Almost half of patients reported that SD negatively impacts their emotional and physical well-being "a lot/a great deal"; HCPs underestimate the level of impact on patients. Most patients with SD reported a significant mental health impact, including anxiety, depression, anxiety about interacting with other people, and isolation. Two-thirds of patients said they did not know anyone else who had been diagnosed with SD, and even after diagnosis, less than half of patients still said they did not know anyone else with SD. Nearly all patients and HCPs agreed that it was challenging to hide SD symptoms, and most patients felt embarrassed when people commented on their SD symptoms. Most patients agreed that they would be further along in their career if they did not have SD, and SD symptoms made them less confident at work and less likely to want to interact with people at work. Almost half of patients reported ever missing work as a result of SD symptoms. Conclusion: These insights emphasize the physical and emotional patient burden associated with SD, impacting all aspects of patients' lives. Graphical abstract available for this article. [ABSTRACT FROM AUTHOR]

Objective: The aim of this observational retrospective study was to report quality of life (QoL) in patients with postoperative facial nerve (FN) palsy after vestibular schwannoma (VS) surgery, investigating clinical factors related to functional outcomes. Methods: Forty-eight consecutive patients (M:F 25:23; median age: 52.5 years) with facial palsy following surgery for sporadic VS were considered retrospectively. FN palsy was graded by using the Sunnybrook facial grading system (SBFGS), while postoperative QoL and subjective functional aspects were assessed by using the Penn Acoustic Neuroma Quality of Life (PANQOL) Scale, the Synkinesis Assessment Questionnaire, and questions on eating and drinking. Results: A significant correlation emerged between all Sunnybrook scores and median PANQOL domain regarding facial function. Increasing overall SBFGS scores were associated with reduced risk of slow chewing on the affected side (p = 0.004), lack of masticatory strength (p = 0.025), masticatory fatigue (p < 0.001), accumulation of food in the oral vestibule (p < 0.001), difficulty in drinking from a glass (p = 0.019), and fluid spillage while drinking (p = 0.016). Conclusions: This study suggests that the clinical evaluation of patients with FN palsy after VS surgery should be integrated with patient reports about functional outcomes and perceived QoL to help clinicians guide rehabilitation choices. [ABSTRACT FROM AUTHOR]

Purpose: We applied a previously established common T-score metric for patient-reported and performance-based physical function (PF), offering the unique opportunity to directly compare measurement type-specific patterns of associations with potential laboratory-based, psychosocial, sociodemographic, and health-related determinants in hemodialysis patients. Methods: We analyzed baseline data from the CONVINCE trial (N = 1,360), a multinational randomized controlled trial comparing high-flux hemodialysis with high-dose hemodiafiltration. To explore the associations of potential determinants with performance-based versus patient-reported PF, we conducted multiple linear regression (backward elimination with cross-validation and Lasso regression). We used standardized T-scores as estimated from the PROMIS PF short-form 4a (patient-reported PF) and the Physical Performance Test (performance-based PF) as dependent variables. Results: Performance-based and patient-reported PF were both significantly associated with a laboratory marker-based indicator of muscle mass (simplified creatinine index), although the effects were relatively small (partial f2 = 0.04). Age was negatively associated with PF; the effect size was larger for performance-based (partial f2 = 0.12) than for patient-reported PF (partial f2 = 0.08). Compared to performance-based PF, patient-reported PF showed a stronger association with self-reported health domains, particularly pain interference and fatigue. When using the individual difference between patient-reported and performance-based T-scores as outcome, we found that younger age and more fatigue were associated with lower patient-reported PF compared to performance-based PF (small effect size). Conclusion: Patient-reported and performance-based assessments were similarly associated with an objective marker of physical impairment in hemodialysis patients. Age and fatigue may result in discrepancies when comparing performance-based and patient-reported scores on the common PF scale. Trial Registration CONVINCE is registered in the Dutch Trial Register (Register ID: NL64750.041.18). The registration can be accessed at: https://onderzoekmetmensen.nl/en/trial/52958. [ABSTRACT FROM AUTHOR]

Purpose: Quality of life (QoL) is identified as a clinical and research priority by the autistic community. Researchers have the responsibility to ensure that instruments used to measure QoL do so reliably and accurately among autistic participants. Methods: Our study evaluated measurement invariance of Emotional Distress (Depression, Anxiety, Anger, Psychological Stress) and Subjective Well-Being (Life Satisfaction, Positive Affect, and Meaning & Purpose) scales of the Patient-Reported Outcomes Measurement Information System (PROMIS) among groups of autistic (N=140, n per scale=132–140) and general population (N=1,224, n per scale=406–411) teenagers (14–17 years). These scales were included in the PROMIS Autism Battery-Lifespan, which uses PROMIS scales to measure QoL domains most relevant for autistic people. Results: Multi-group confirmatory factor analyses using permutation tests demonstrated that Depression and Positive Affect scales exhibited scalar invariance between groups, indicating that scores can be meaningfully compared across autistic and general population teens. Anger and Psychological Stress scales demonstrated metric invariance between groups, indicating that these scales measure the same latent trait in both groups, but group comparisons are not supported. Conclusion: We provide guidance as to how these scales can be used in psychometrically supported ways to capture constructs relevant for understanding QoL among autistic teens. [ABSTRACT FROM AUTHOR]

Background: Appropriate selection of surgical approach for associated fractures of the acetabulum that involves both columns is still elusive. Therefore, present study aimed to assess the quality of life of complex acetabulum fracture managed with combined anterior and posterior approach (dual approach) as well as the association of postoperative reduction and patient reported outcomes. Material and methods: This retrospective study was performed on 42 associated acetabular fracture patients with involvement of both columns, who were treated with a dual approach including combined anterior modified ilio-inguinal approach in combination with posterior Kocher-Langenbeck (K-L) approach in a single anaesthetic session having minimum two years follow-up. Postoperative reduction and radiological outcomes were assessed using Matta criteria. Functional outcome was assessed by a score system developed by Merle d'Aubigné-Postel. Patient-reported outcomes were assessed using the 36-Item Short Form Survey (SF-36). Results: The mean age of participants was 47.9 ± 11.95 years and mean follow-up period was 2.8 ± 0.49 years. Anatomical reduction was achieved in 32 (76.19%) cases in postoperative period. 8 (19.04%) cases had congruent and 2 (4.76%) had incongruent reduction. Radiological and functional outcome was excellent/good n = 35 (83.33%) cases. Patient-reported outcome on domains physical function and role physical showed a significant improvement between 6 to 24 months period (p < 0.001). Conclusions: The dual approach for complex acetabulum fracture with mid-term follow-up showed good to excellent radiological and functional outcomes. A significant improvement in patient-reported outcomes on Physical function and Role physical domains of SF-36 was also seen over the follow-up period. [ABSTRACT FROM AUTHOR]

Objectives: Racial and ethnic disparities in patient-reported outcomes (PROs) among gynecologic cancer survivors are not well studied. We evaluated whether individual-level characteristics were associated with PROs in diverse gynecologic cancer survivors. Methods: Gynecologic cancer patients in an ambulatory oncology clinic completed a psychosocial and practical needs assessment before their appointments through the electronic medical record (EMR) patient portal. Assessments were available in English and Spanish. Fatigue, pain, physical function, depression, and anxiety were assessed with Patient-Reported Outcomes Measurement Information System (PROMIS®) computer adaptive tests, and health-related quality of life was assessed by FACT-G7. PROs were dichotomized based on severity (normal/mild vs moderate/severe). Demographic and clinical information was collected. Analyses were performed using Chi-square, t-tests, and Kruskal–Wallis tests. Results: A total of 582 women completed the assessment; 20% (n = 116) were racial minorities, and 54.5% (n = 310) were Hispanic. A total of 192 (32.8%) completed the assessments in Spanish. Hispanic patients had lower mean fatigue scores (49.31 vs 51.74, p = 0.01), and patients whose preferred language was Spanish had lower mean depression (47.63 vs 48.97, p = 0.05) and fatigue scores (48.27 vs 51.27, p < 0.01). There were no significant differences in the severity of PROs by race, ethnicity, or preferred language. QOL scores were worse in patients with high symptom severity for anxiety (p = 0.04) and physical functioning (p < 0.01). Current smokers had worse physical functioning (13.4% vs 6.5%, p = 0.03). Conclusions: We found no significant differences in severity of PROs by race, ethnicity, or preferred language. Quality of life scores were worse for patients with high symptom severity for physical functioning and anxiety. [ABSTRACT FROM AUTHOR]

Purpose: Meniscal allograft transplantation (MAT) is a valuable option for postmeniscectomy syndrome in young and active patients, which can successfully improve symptoms, function and quality of life. This study aimed to report outcomes and complications in patients treated with isolated MAT or MAT combined with revision anterior cruciate ligament reconstruction (ACLR) and lateral extra‐articular tenodesis (LET). Methods: In total, 18 patients who were treated with MAT using a soft tissue fixation technique were retrospectively analysed. Patients were allocated into two groups, the isolated MAT group and the MAT plus revision ACLR + LET group (MAT+ group). Both groups were assessed using the following Patient‐Reported Outcome Measures (PROMs): Knee Injury and Osteoarthritis Outcome Score (KOOS), International Knee Documentation Committee (IKDC), Tegner Activity Scale, Lysholm score, EuroQol 5‐Dimension 5‐level (EQ‐5D‐5L) questionnaire and a patient acceptable symptom state (PASS) statement. Results: The mean follow‐up was 3.2 years and the mean age of patients was 29 (±7.6) years. Ten patients were treated with isolated MAT and eight patients were treated with MAT combined with revision ACLR and LET. No complications and no reoperations were reported. In both groups, all PROMs significantly improved postoperatively (p < 0.05), except for the Tegner score in the MAT+ group, which did not reach statistical significance (not significant [n.s.]). No significant difference was found postoperatively in PROMs between the MAT and the MAT+ group; however, differences in the KOOS Symptoms, Sports and Quality‐of‐life subscales reached the minimal clinically important threshold. Mean values were: IKDC 61.2 (±13.9) versus 64.7 (±23.2), KOOS Total 72.8 (±14.9) versus 68.0 (±16.7), Lysholm score 83.2 (±11) versus 84.2 (±8.7) and EQ‐5D‐5L 71.8 (±14.9) versus 72.2 (±22.4). Median values for the Tegner Activity Scale were 4 versus 3. PASS statement was negative in 37.5% of the MAT+ group in comparison to 20% of the MAT group (n.s.). Conclusion: In terms of patient‐reported outcomes, MAT is an effective and safe procedure, even when combined with revision ACLR + LET. Compared to the preoperative assessment, a significant improvement of the postoperative PROMs can be expected either when MAT is performed isolated or in combination with ACLR + LET. However, clinically important differences between the two groups can be noted in favour of the isolated MAT procedure, especially regarding symptoms and sporting activity. Level of Evidence: Level III. [ABSTRACT FROM AUTHOR]

Purpose: To evaluate long‐term outcomes of patients treated with posterior medial meniscal root tear (PMMRT) repair through assessment of functional outcome scores and to identify patient surgical and magnetic resonance imaging (MRI) characteristics associated with improved outcomes. Methods: This was a single‐centre, retrospective study evaluating patients who had undergone a PMMR repair using a transtibial suture pullout technique with two locking cinch sutures. This was performed as a follow‐up to previously published 2‐year and 5‐year outcome studies, using the same cohort. All patients from the prior short‐term and midterm studies were invited to participate. Patient‐reported outcome (PROs) scores, including the International Knee Documentation Committee (IKDC) and Lysholm scores, were collected. Previously collected demographic data were updated based on review of the electronic medical record. Patient outcomes were assessed preoperatively, as well as at 2‐year, 5‐year and 8‐year postoperatively. MRI outcome measurements were assessed at 2‐year and 5‐year follow‐ups. All statistical analysis was performed using SPSS version 26. Results: Seventeen patients of the original 18 patients (94.4%) were included in the final analysis. Additionally, three patients who had additional ipsilateral surgery were excluded from the analysis of PROs. The IKDC score significantly increased from 44.7 ± 11.6 at preoperative baseline to 71.2 ± 21.3 at 8‐year post‐operation (p = 0.001). There were no significant differences in IKDC score between 2‐year and 8‐year follow‐ups (p = n.s.) or 5‐year and 8‐year follow‐ups (p = n.s.). The Lysholm score significantly increased from 49.6 ± 7.3 at preoperative baseline to 76.4 ± 17.2 at 8‐year follow‐up (p < 0.001). There was no significant difference in Lysholm scores between 2‐year and 8‐year follow‐ups (p = n.s.) or 5‐year and 8‐year follow‐ups (p = n.s.). A linear regression analysis found that 5‐year IKDC scores were significantly correlated with 8‐year IKDC scores (β = 0.681, p = 0.038). At 8‐year follow‐up, four (23.5%) patients required additional procedures on their operative knee (one total knee arthroplasty conversion). Conclusion: Patients treated with repair of PMMRT had maintenance of clinical outcome improvements at long‐term follow‐up despite worsening MRI outcomes at short‐term and medium‐term follow‐ups. While a high proportion of patients required additional procedures on their operative knee at 8‐year follow‐up, few of these patient's additional procedures were related to failure of their primary surgery. Providers and patients may expect durable clinical outcomes following the repair of PMMRT, irrespective of radiographic appearance. Level of Evidence: Level IV. [ABSTRACT FROM AUTHOR]

Background: Barbed sutures (BS) have been increasingly used in the last two decades across surgical disciplines but little is known about how widespread their adoption has been in ventral hernia repair (VHR). The aim of this study was to document the use of barbed sutures in VHR in a multicenter database with associated clinical and patient-reported outcomes. Method: Prospectively collected data from the Abdominal Core Health Quality Collaborative database was retrospectively reviewed, including all adult patients who underwent VHR with fascial closure from 2020 to 2022. A univariate analysis compared patients with BS against non-barbed sutures (NBS) across the preoperative, intraoperative, and postoperative timeframes including patient-reported outcomes concerning quality of life and pain scores. Results: A total of 4054 patients that underwent ventral hernia repair with BS were compared with 6473 patients with non-barbed sutures (NBS). Overall, BS were used in 86.2% of minimally invasive ventral hernia repairs and about 92.2% of robotic surgery compared to only 9.6% of open procedures. Notable differences existed in patient selection, including a higher BMI (32 vs 30.5; p < 0.001), more incisional hernias (63.3% vs 51.1%; p < 0.001), wider hernias (4 cm vs 3 cm; p < 0.001), and higher ASA score (p < 0.001) in patients with BS. Outcomes in patients with BS included a shorter length of stay (mean days; 1.4 vs 2.4; p < 0.001), less SSI (1.5% vs 3.6%; p < 0.001), while having similar SSO (7.6% vs 7.3%; p = 0.657), readmission (3.0 vs 3.2; p = 0.691), and reoperation (1.5% vs 1.45%; p = 0.855), at a longer operative time (p < 0.001). Hernia-specific questionnaires for quality of life (HerQLes) and pain in patients with BS had a worse preoperative score that was later matched and favorable compared to NBS (p = 0.048). PRO concerning hernia recurrence suggest around 10% at two years of follow-up (p = 0.532). Conclusion: Use of barbed sutures in VHR is widespread and highly related to MIS. Outcomes from this multicenter database cannot be reported as superior but suggest that barbed sutures do not have a negative impact on outcomes. [ABSTRACT FROM AUTHOR]

Objectives: This umbrella review aims to synthesise and summarise the role of teledentistry in improving oral health outcomes and access to dental care. Methods: We searched the databases PubMed (Medline), Scopus, CINHAL, OVID, ScienceDirect, JSTOR, JBI Database of Systematic Reviews & Implementation Reports and the Cochrane Database of Systematic Reviews through March 2024. Systematic reviews and meta‐analyses on teledentistry were eligible for inclusion. No publication time or language restrictions were applied. Our search retrieved 24 studies for which we conducted quality assessments using the Joanna Biggs Institute (JBI) for Systematic Reviews and Research Synthesis Critical Appraisal Tool. Results were reported according to the Preferred Reporting Items for Systematic Reviews and Meta‐Analyses (PRISMA) guidelines. Results: Studies addressed clinical oral health outcomes, health‐related quality of life and patient experience, access to dental care and cost‐effectiveness of teledentistry compared to conventional, face‐to‐face dental consultations. We found that there was consensus that teledentistry enhanced oral health through the early detection of oral lesions and increased access to dental care in remote areas and was time‐ and cost‐saving. Conclusion: Teledentistry can improve oral health outcomes and access to dental care. Future research on its impact on oral health equity is warranted. [ABSTRACT FROM AUTHOR]

Novel surgical treatments for painful neuromas are increasingly used, but determining which provides the greatest benefit has been difficult due to the inconsistent use of outcome measures. We mapped the current literature of outcome measures used to evaluate peripheral nerve surgery for the management of symptomatic neuromas in patients who underwent an adult‐acquired upper extremity amputation (UEA). Medline, Embase, Cochrane, and CINAHL were searched for primary research written in the English language from inception to February 2023. The search yielded 1137 articles, of which 35 were included for final analysis. Studies varied in their assessment of pain, health‐related quality of life (HRQOL), neurotrophic measures, psychological and sensorimotor function, highlighting a consensus on crucial domains but also revealing significant heterogeneity in the use and application of outcome measures among primary studies. Our findings highlight the need to establish common standards that reflect the best evidence and unique needs of the UEA population. This includes developing a core outcome set, utilizing multi‐center trials, and maintaining flexibility to adapt to ongoing advancements in patient‐reported outcome measures (PROMs) research. [ABSTRACT FROM AUTHOR]

Because young children cannot self-report symptoms, there is a need for parent surrogate reports. Although early work suggested parent-child alignment for eosinophil esophagitis (EoE) patient-reported outcomes (PROs), the longitudinal alignment is unclear. We sought to assess the agreement and longitudinal stability of PROs between children with EoE and their parents. A total of 292 parent-child respondents completed 723 questionnaires over 5 years in an observational trial in the Consortium of Eosinophilic Gastrointestinal Disease Researchers. The change in and agreement between parent and child Pediatric Eosinophilic Esophagitis Symptom Score version 2 (PEESSv2.0) and Pediatric Quality of Life Eosinophilic Esophagitis Module (PedsQL-EoE) PROs over time were assessed using Pearson correlation and Bland-Altman analyses. Clinical factors influencing PROs and their agreement were evaluated using linear mixed models. The cohort had a median disease duration equaling 3.7 years and was predominantly male (73.6%) and White (85.3%). Child and parent PEESSv2.0 response groups were identified and were stable over time. There was strong correlation between child and parent reports (PEESSv2.0, 0.83;PedsQL-EoE, 0.74), with minimal pairwise differences for symptoms. Longitudinally, parent-reported PedsQL-EoE scores were stable (P ≥.32), whereas child-reported PedsQL-EoE scores improved (P =.026). A larger difference in parent and child PedsQL-EoE reports was associated with younger age (P <.001), and differences were driven by psychosocial PRO domains. There is strong longitudinal alignment between child and parent reports using EoE PROs. These data provide evidence that parent report is a stable proxy for objective EoE symptoms in their children. [ABSTRACT FROM AUTHOR]

Post-traumatic critical-sized bone defects pose a reconstructive challenge for reconstructive surgeons. The vascularized fibula graft is a well-described treatment for osseous defects of the femur and tibia. This study aimed to assess long-term patient-reported quality of life, the success-, and complication rates in lower extremity reconstruction with vascularized fibula grafts. A retrospective cohort of 29 patients who underwent fibula graft reconstruction for critical-sized bone defects after post-traumatic tibial and femoral bone loss between 1990 and 2021 was included. To assess the health-related quality of life and return to work and satisfaction, a cross-sectional survey was performed using the short-form-36, lower extremity functional scale, and a self-made questionnaire including the DN4, satisfaction, and subjective ankle function. The median bone defect size was 8 cm (IQR 9–7 cm). The mental component scores were comparable to the Dutch population norm, whereas the impaired physical function scores were associated with pain (r 0.849, p < 0.001). Neuropathic symptoms were reported in 7 out of 19 patients, and 11 out of 19 patients returned to normal daily activity. All respondents reported positive or neutral scores on overall satisfaction with the recovery. Bone healing was uneventful in 19 out of 29 patients. Union was achieved in 25 out of 29 patients. Persistent nonunion was observed in 4 patients, leading to amputation in 2 patients. Vascularized fibula graft use led to high union rates and limb salvage in patients with post-traumatic segmental bone loss of the tibia and femur. Patient satisfaction with the overall recovery was positive; however, functional outcomes remained impaired. [ABSTRACT FROM AUTHOR]

Background: This study characterized the impact of baseline symptom burden on long‐term quality‐of‐life in patients receiving head and neck radiation therapy (RT). Methods: The Vanderbilt Head and Neck Symptom Survey was collected prior to head and neck RT and at follow‐up visits. Responses were divided into symptom clusters of toxicities and scored from 0 (asymptomatic) to 10 (severe). Patients with responses at baseline and 1‐year or 2‐year follow‐up were stratified by scores ≤1 or >1 and compared using the Mann–Whitney U‐test. Results: At 1‐year follow‐up (n = 75), patients with higher baseline scores had greater symptom burden for every cluster except in taste/smell. At 2‐year follow‐up (n = 47), patients with higher baseline scores had greater symptom burden for every cluster except in nutrition, dry mouth, trismus, neck tightness, and hearing. Conclusion: The Vanderbilt Head and Neck Symptom Survey demonstrated a relationship between baseline symptom burden and long‐term quality‐of‐life and might be useful as a screening tool. [ABSTRACT FROM AUTHOR]

Background: Pelvic exenteration (PE) is often the only curative treatment option for selected locally advanced and locally recurrent colorectal cancer associated with significant morbidity. Open and laparoscopic approaches were accepted for this procedure. Objective: This study aimed to examine the Chinese patient-reported outcomes (PROs) and health-related quality of life (HRQoL) after PE. Methods: A total of 122 enrolled participants were asked to complete PROs at baseline and 1, 3, 6, 9 and 12 months after PE. PROs included seven symptoms from the National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). The HRQoL was assessed using the Functional Assessment of Cancer Therapy-Colorectal (FACT-C). Results: The overall postoperative complication rate was 41.0%. Patients experienced lower physical and functional well-being and FACT-C 1 month after surgery, then gradually recovered. The FACT-C score returned to baseline 9 months after surgery. Social and emotional well-being did not show signs of recovery until 6 months after the surgical procedure, and did not fully return to baseline until 12 months post-surgery. Symptom rates of insomnia, anxiety, discouragement, and sadness (composite score >0) did not improve significantly from baseline until 12 months after surgery. Conclusions: PE is a feasible treatment choice for locally advanced primary and recurrent colorectal cancer. Social, psychological, and emotional recovery in the Chinese population after PE tends to be slower compared with the physical condition. [ABSTRACT FROM AUTHOR]

Background: Although studies have compared patient-reported outcomes (PROs) after breast conserving-therapy (BCT) and postmastectomy breast reconstruction (PMBR), they often have been confounded by treatment or other factors that complicate a direct comparison. This study aimed to compare PROs after BCT and PMBR by using propensity score-matching analysis. Methods: Patients who underwent BCT or PMBR between 2010 and 2022 and completed the BREAST-Q were identified. Each BCT patient was matched to a PMBR patient using nearest-neighbor 1:1 matching with replacement for each BREAST-Q time point. Outcomes included all prospectively collected BREAST-Q domains preoperatively, at 6 months, and at 1, 2, and 3 years postoperatively. A 4-point difference was considered clinically meaningful. Results: For this study, 6215 patients (2501 BCT [40.2%] and 3714 PMBR [59.8%] patients) were eligible, and 2616 unique patients were matched. Preoperatively, 463 BCT and 463 PMBR patients were matched for analysis (6 months [443 matched pairs], 1 year [639 matched pairs], 2 years [421 matched pairs], 3 years [254 matched pairs]). At 6 months postoperatively, the BCT patients scored higher on all BREAST-Q domains than the PMBR patients (p < 0.05; differences > 4 points). At 1, 2, and 3 years, the patients who underwent BCT consistently had superior Satisfaction With Breasts, Psychosocial Well-Being, and Sexual Well-Being (p < 0.05), and the differences were clinically meaningful. Conclusion: In this statistically powered study, the BCT patients reported higher quality of life than the PMBR patients in early assessment and also through 3 years of follow-up evaluation. Given the equivalency in survival and recurrence outcomes between BCT and PMBR, patients eligible for either surgery should be counseled regarding the superiority of BCT in terms of PROs. [ABSTRACT FROM AUTHOR]

Background: Transfusion-dependent β-thalassemia (TDT) is a severe inherited disorder. Without regular treatment, patients with TDT experience complications that can significantly shorten life expectancy and severely impact both their quality of life and that of their families. The condition has attracted significant attention in global health discussions. Due to the challenges of blood supply shortages, the high costs of iron chelation therapy, and hematopoietic stem cell transplantation (HSCT), TDT presents a serious health risk to patients and imposes a substantial burden on families and society. However, research on the health-related quality of life (HRQoL) of thalassemia patients in China remains limited. This study evaluated the factors affecting the HRQoL of these patients, with the goal of developing strategies to improve their quality of life. Methods: In this cross-sectional study, children with TDT were recruited from five treatment centers in Guangxi, a province with a high prevalence of thalassemia in China. Structured questionnaires were employed to gather relevant data on sociodemographic variables, disease characteristics, treatments, and associated costs. The HRQoL was assessed using the Transfusion-Dependent Quality of Life (TranQoL) questionnaire, with a proxy version for patients aged 0–11 years and a child version for those aged 12–18 years. Results: The study included 418 participants, yielding an overall TranQoL score of 60.6 ± 16.3 among thalassemia patients. Multiple linear regression analysis revealed a negative correlation (P < 0.05) between overall TranQoL scores and several factors: increasing patient age, the presence of multiple thalassemia patients within a family, and undergoing HSCT. Conversely, adherence to regular treatment was positively correlated with higher TranQoL scores (P < 0.05). Conclusion: The study demonstrates that HRQoL among Chinese patients with TDT is at a low level. Age, treatment adherence, family support, and socioeconomic status were identified as key determinants influencing HRQoL. It is essential to further enhance and optimize health insurance policies and medical services to support comprehensive treatment strategies for these patients. [ABSTRACT FROM AUTHOR]

Background: In clinical oncology practice, patient-reported outcomes (PROs) are essential for assessing the symptom burden, quality of life, and psychological status of patients. However, there remains a gap between the use of PROs in an oncologic setting and its implementation. Furthermore, numerous reviews in PRO implementation are often based on one particular technology, setting, or health condition, making it difficult to obtain a comprehensive and coherent summary of available evidence to help plan and undertake implementation. This systematic review aims to identify and integrate enablers and barriers to PRO implementation through the comprehensive framework for implementation research (CFIR) to provide a reference for implementing patient-reported outcomes management in oncology settings. Methods: This review strictly observed the preferred reporting items for systematic reviews and meta-analyses (PRISMA) guidelines. PubMed, Web of Science, CINAHL, Embase, and PsycINFO databases were systematically searched using a three-step search strategy. The search was limited from the inception of each database to April 2024. Articles describing facilitators and barriers to implementing PROs in clinical oncology practice were included. Two researchers screened the literature independently, and the quality assessment of cross-sectional, qualitative, and mixed studies was completed using the critical appraisal tools recommended by Joanna Briggs Institute (JBI) and the mixed methods assessment tool, respectively. Basic information about the included studies and determinants affecting PRO implementation was extracted, and coding categorization of facilitators and barriers was completed based on the 48 constructs provided by the CFIR framework. Results: We included 30 studies from 5,649 search results, including 25 original and 5 review studies. The quality of the literature for qualitative studies was generally good, and the quality for quantitative and mixed studies was assessed as fair. We identified 52 facilitators and 50 barriers in the included literature, covering the domains used in the CFIR framework and 39 constructs, mainly including "Innovation Evidence-Base", "Innovation Complexity", "Innovation Design", "Structural Characteristics", "Compatibility", "Incentive Systems", "Access to Knowledge & Information", "Innovation Deliverers", "Innovation Recipients", and "Planning". Conclusions: This systematic review integrated facilitators and barriers affecting PRO implementation in routine oncology clinical practice settings and categorized them through the CFIR framework. These influencing factors should be fully considered in future clinical practice to ensure the successful implementation of PROs. Trial registration: It has been registered prospectively in PROSPERO under the registration number 42024532983. [ABSTRACT FROM AUTHOR]

Background: To assess the results after elbow arthroplasty it is essential to gather patient-reported outcome measures (PROMs). However, the acquisition of PROMs poses a challenge because of potential low literacy, lengthiness and diversity of questionnaires, and questionnaire fatigue. Instead of a questionnaire, patient-reported outcomes can be collected using a single assessment numeric evaluation (SANE), the subjective elbow value (SEV). The aim of this pilot study is to assess the correlation between the SEV and conventionally used patient reported outcome measures (PROMs) after elbow arthroplasty. Materials and methods: The SEV was added to our follow-up system in 2021, consisting of a scale from 0 to 10 in which the patients are asked to rate the overall functionality of their elbow, 0 corresponds to very poor functionality and 10 to a perfectly functional or healthy elbow. All patients who underwent elbow arthroplasty (total or radial head) and responded to the SEV question were retrospectively identified and included. The correlation between the SEV at the final follow-up and the Oxford Elbow Score (OES), and between the SEV and the Quick Disbailities of the Arm, Shoulder, and Hand (quickDASH) score was assessed using Pearson's r. Results: In total, 82 patients responded to the SEV question and were included in the study, with a median follow-up of 5 years [interquartile range (IQR) 3–7]. Of these patients, 17 (21%) underwent radial head arthroplasty and 65 (79%) total elbow arthroplasty. The Pearson's r for the correlation between SEV and OES was 0.502 (p < 0.001) and between the SEV and the QuickDASH −0.537 (p < 0.001), which correspond to a moderate correlation. Conclusions: The SEV shows a moderate correlation with conventional PROMs, demonstrating its potential in simplifying the follow-up of elbow arthroplasty, possibly decreasing time, costs, and patients' questionnaire fatigue compared with conventional PROM questionnaires. Evidence level: III. [ABSTRACT FROM AUTHOR]

Objectives Subjects and Methods Results Conclusion To characterise the long‐term success rate of ventral onlay buccal mucosa graft urethroplasty (vBMG) in the management of bulbar urethral stricture disease (USD), assess patient‐reported postoperative satisfaction and decision regret, and delineate clinical factors impacting patient‐reported metrics.Patients with prior vBMG for bulbar USD, performed at Cleveland Clinic between 2003 and 2022, were contacted and brief structured interviews were performed. Stricture recurrence and need for secondary procedures, baseline demographics, and patient‐reported outcome surveys were collected. The surveys included the Decision Regret Scale (DRS), the Urethral Stricture Symptom Impact Measure (USSIM) and the 10‐item Patient‐Reported Outcomes Measurement Information System Short Form, version 1.2 (PROMIS‐10). Descriptive, univariate and multivariable analyses were performed for clinical outcomes and survey responses.A total of 104 patients recorded responses. The median patient age was 49 years and the median follow‐up was 7.4 years at time of survey. The median graft length was 5 cm and 38% of patients underwent partial thickness augmented anastomotic urethroplasty. At time of follow‐up, 10 patients underwent a secondary procedure. Moderate to severe regret on the DRS was found in 12% of patients, and greater regret was associated with recurrence. The mean physical and mental health PROMIS‐10 Global Health T‐scores were 52 and 53. The mean total USSIM score was 56. A significant correlation was found between USSIM and DRS scores, with higher DRS score and recurrence negatively impacting USSIM score. USSIM scoring across all domains was significantly worse in the moderate to severe DRS group.This study showed that vBMG for bulbar USD confers both high success rates and patient‐reported satisfaction at extended follow‐up, based on emerging and validated patient‐reported outcome measures. [ABSTRACT FROM AUTHOR]

Obesity is a key factor in the development and progression of both heart failure with preserved ejection fraction (HFpEF) and atrial fibrillation (AF). In the STEP-HFpEF Program (comprising the STEP-HFpEF [Research Study to Investigate How Well Semaglutide Works in People Living With Heart Failure and Obesity] and STEP-HFpEF DM [Research Study to Look at How Well Semaglutide Works in People Living With Heart Failure, Obesity and Type 2 Diabetes] trials), once-weekly semaglutide 2.4 mg improved HF-related symptoms, physical limitations, and exercise function and reduced body weight in patients with obesity-related HFpEF. Whether the effects of semaglutide in this patient group differ in participants with and without AF (and across various AF types) has not been fully examined. The goals of this study were: 1) to evaluate baseline characteristics and clinical features of patients with obesity-related HFpEF with and without a history of AF; and 2) to determine if the efficacy of semaglutide across all key trial outcomes are influenced by baseline history of AF (and AF types) in the STEP-HFpEF Program. This was a secondary analysis of pooled data from the STEP-HFpEF and STEP-HFpEF DM trials. Patients with heart failure, left ventricular ejection fraction ≥45%, body mass index ≥30 kg/m2, and Kansas City Cardiomyopathy Questionnaire–Clinical Summary Score (KCCQ-CSS) <90 points were randomized 1:1 to receive once-weekly semaglutide 2.4 mg or matching placebo for 52 weeks. Dual primary endpoints (change in KCCQ-CSS and percent change in body weight), confirmatory secondary endpoints (change in 6-minute walk distance; hierarchical composite endpoint comprising all-cause death, HF events, thresholds of change in KCCQ-CSS, and 6-minute walk distance; and C-reactive protein [CRP]), and exploratory endpoint (change in N-terminal pro–B-type natriuretic peptide [NT-proBNP]) were examined according to investigator-reported history of AF (yes/no). Responder analyses examined the proportions of patients who experienced a ≥5-, ≥10, ≥15, and ≥20-point improvement in KCCQ-CSS per history of AF. Of the 1,145 participants, 518 (45%) had a history of AF (40% paroxysmal, 24% persistent AF, and 35% permanent AF) and 627 (55%) did not. Participants with (vs without) AF were older, more often male, had higher NT-proBNP levels, included a higher proportion of those with NYHA functional class III symptoms, and used more antithrombotic therapies, beta-blockers, and diuretics. Semaglutide led to larger improvements in KCCQ-CSS (11.5 points [95% CI: 8.3-14.8] vs 4.3 points [95% CI: 1.3-7.2]; P interaction = 0.001) and the hierarchal composite endpoint (win ratio of 2.25 [95% CI: 1.79-2.83] vs 1.30 [95% CI: 1.06-1.59]; P interaction < 0.001) in participants with AF vs without AF, respectively. The proportions of patients receiving semaglutide vs those receiving placebo experiencing ≥5-, ≥10-, ≥15-, and ≥20-point improvement in KCCQ-CSS were also higher in those with (vs without) AF (all P interaction values <0.05). Semaglutide consistently reduced CRP, NT-proBNP, and body weight regardless of AF status (all P interaction values not significant). There were fewer serious adverse events and serious cardiac disorders in participants treated with semaglutide vs placebo irrespective of AF history. In the STEP-HFpEF Program, AF was observed in nearly one-half of patients with obesity-related HFpEF and was associated with several features of more advanced HF. Treatment with semaglutide led to significant improvements in HF-related symptoms, physical limitations, and exercise function, as well as reductions in weight, CRP, and NT-proBNP in people with and without AF and across AF types. The magnitude of semaglutide-mediated improvements in HF-related symptoms and physical limitations was more pronounced in those with AF vs without AF at baseline. (Research Study to Investigate How Well Semaglutide Works in People Living With Heart Failure and Obesity [STEP-HFpEF; NCT04788511 ]; Research Study to Look at How Well Semaglutide Works in People Living With Heart Failure, Obesity and Type 2 Diabetes [STEP-HFpEF DM; NCT04916470 ]) [ABSTRACT FROM AUTHOR]

Background: Chronic obstructive pulmonary disease (COPD) is marked by severe physical symptoms, impaired quality of life, and high psychological distress. Despite its impact, the identification of not only physical but also psychosocial and existential issues in the clinic lags behind that of other patient groups. Methods: This study aimed to assess physical, psychosocial, and existential issues among patients with COPD using a patient-reported outcome measure for general palliative care (the 'PRO-Pall') in a Danish outpatient clinic. We included 115 adults with COPD who completed the PRO-Pall either electronically or in the clinic. Sociodemographic and illness-related data were retrieved from their electronic health records. Results: We found that shortness of breath, tiredness, and difficulty walking were predominant physical issues. Worry about change in social roles was the most frequently reported psychosocial issue, while existential issues were reported by approximately one in ten patients. Most patients (44.5%) felt able to share their feelings with family or friends, and a majority (62.2%) felt their illness-related issues were addressed satisfactorily. Females expressed a greater need for rest and males more frequently reported intimacy issues. Higher COPD-impact on life measured by the COPD Assessment Test was associated with lower ratings on the quality-of-life item of the PRO-Pall independent of age, gender, lung function, and smoking status. Conclusions: Patients reported issues in physical, psychosocial, and existential dimensions of the PRO-Pall. The PRO-Pall shows potential as a broader alternative to measures that focus mainly on physical issues. [ABSTRACT FROM AUTHOR]

Background: As with many other chronic diseases, systemic lupus erythematosus (SLE) and lupus nephritis (LN) have significant impacts on the health-related quality of life (HRQoL). Medication non-adherence is a significant challenge in the management of SLE, with consistently up to 75% of patients being non-adherent with their SLE medications. There is a need to assess the patient's perspective using patient-reported outcomes (PROs) to better understand the current impact of LN on HRQoL and treatment adherence in our region. The aim of this study was to explore the relationship between HRQoL and treatment adherence in patients with LN from the Colombian Caribbean. Methods: A cross-sectional study was conducted from June to December 2022, including patients with biopsy-proven LN. HRQoL and treatment adherence were assessed using the Lupus Quality of Life (LupusQoL) and the Compliance Questionnaire in Rheumatology 19 (CQR19) instruments, respectively. Patients were categorized as adherent or non-adherent based on medication intake (defined as >80% correct dosage). Principal component analysis (PCA) was employed to identify principal components between adherent and non-adherent patients. Results: A total of 42 patients with LN were included. Of these, 38 (90%) were female, and the mean age was 31 ± 10 years. Proliferative class IV was the predominant histopathological profile (90%). Twenty-five (60%) patients were categorized as non-adherent. Across all LupusQoL domains, a comprehensive range of responses was observed. Pain, planning, and intimate relationships domains remained unaffected, while burden to others domain had the lowest score. Poorer planning score correlated with older age (r = −0.72; p <.05) and longer disease duration (r = −0.74; p <.05). SLEDAI-2 K correlated with the pain domain (r = −0.78; p <.05). Non-adherent patients exhibited significantly worse pain domain scores compared to adherent counterparts (p <.05). PCA showed strong interactions between planning and pain, as well as between physical health and body image domains. Conclusions: LupusQoL pain domain scores were significantly worse in non-adherent patients compared to adherent patients. Effective pain management could be a determinant in HRQoL and treatment adherence rates in our population. [ABSTRACT FROM AUTHOR]

Aim ASPEN is a randomized, open-label, Phase III study comparing zanubrutinib and ibrutinib in patients with Waldenström macroglobulinemia (WM). Materials & methods: Patient-reported outcomes were exploratory end points assessed using the EORTC QLQ-C30 and EQ-5D-5L VAS scores. Results: Overall, 201 patients (102 zanubrutinib; 99 ibrutinib) were enrolled. Clinically meaningful differences were observed in diarrhea and nausea/vomiting in both the intent-to-treat population and in patients attaining very good partial response (VGPR) in earlier cycles of treatment, as well as in long-term physical functioning and fatigue in patients achieving VGPR. Conclusion: Treatment with zanubrutinib was associated with greater improvements in health-related quality of life compared with ibrutinib in patients with WM and MYD88 mutations. Clinical Trial Registration:NCT03053440 (ClinicalTrials.gov) Plain Language Summary Patient quality of life is important What is this article about? This article talks about a study called the ASPEN trial, which compares two medicines used for treating a rare blood cancer that doctors call Waldenström macroglobulinemia. The medicines are called zanubrutinib (ZAN) and ibrutinib (IBR). They work in the same way, by blocking a protein called Bruton tyrosine kinase. When patients take medicines for an illness, it is important to learn about their physical, social, emotional and mental well-being (quality of life). In this study, we asked patients to fill out questionnaires about their well-being before starting the study treatment for their blood cancer, and again a few times while taking the medication, to see if there were any changes. What were the results of the study? There were two groups of patients. One group took ZAN and the other took IBR. The patients could not choose which medicine they were going to take. Results from both groups of patients were compared. Patients taking ZAN did not feel worse or better about their diarrhea and sickness, but those taking IBR said these symptoms had become worse. Both medicines improved how patients were feeling. However, improvement in tiredness and physical ability was larger in patients taking ZAN than those on IBR, especially for the patients whose cancer was getting better. What do the results mean? For patients with a rare blood cancer in this study, those taking ZAN had a better quality of life than those taking IBR. Tweetable Abstract In a head-to-head comparison in the Phase III ASPEN study, patients with Waldenström macroglobulinemia treated with zanubrutinib had better quality of life than patients treated with ibrutinib. Article highlights Waldenström macroglobulinemia (WM) is a rare B-cell lymphoma marked by an excess of monoclonal immunoglobulin-M. Patients with WM experience symptoms including fatigue, muscle weakness, neuropathy and anemia, significantly affecting their health-related quality of life (HRQoL). Bruton tyrosine kinase (BTK) inhibitors, particularly ibrutinib, have shown efficacy in treating WM; however, they are associated with adverse events that can lead to early treatment discontinuation, negatively affecting HRQoL. Zanubrutinib, a next-generation BTK inhibitor, was designed with improved selectivity for BTK to minimize off-target effects and toxicities and thereby potentially improve HRQoL. Materials & methods ASPEN is a randomized, open-label, Phase III study comparing zanubrutinib and ibrutinib in patients with relapsed/refractory or treatment-naive WM. In this study, HRQoL was measured with two validated patient-reported instruments: the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire – Core 30 and the EQ-5D-5L. Results Clinically meaningful improvements in diarrhea and nausea/vomiting symptoms were observed in patients receiving zanubrutinib compared with ibrutinib. Patients with a very good partial response to zanubrutinib had significantly increased HRQoL, particularly in physical functioning and fatigue symptoms. Both treatment arms had consistently improved EQ-5D-5L scores through Cycle 13. Discussion This is the first head-to-head study comparing HRQoL in patients with WM treated with different BTK inhibitors. The results reported here are consistent with the primary results of the ASPEN study reported by Tam et al. [10]. Conclusions In addition to the better efficacy observed with zanubrutinib vs. ibrutinib, zanubrutinib provides a higher quality of life to patients with WM. [ABSTRACT FROM AUTHOR]

Background: It is unclear how patient-reported outcomes (PROs) change longitudinally after breast cancer surgery. We sought to compare trends in PROs among patients who underwent lumpectomy versus mastectomy over the first year after surgery. Patients and Methods: Newly diagnosed stage 0–III female patients with breast cancer who underwent lumpectomy or mastectomy at an academic breast center between June 2019 and March 2023 were invited to participate in a longitudinal PRO study. Enrolled patients received the BREAST-Q™ module, a validated tool measuring domains, such as satisfaction with breasts, psychosocial well-being, physical well-being, and sexual well-being. Scores for each domain were compared between the lumpectomy and mastectomy groups over the first year after surgery. Linear mixed models were used to estimate the change in PRO scores over time. Results: The cohort included 203 who underwent lumpectomy and 144 who underwent mastectomy. Patients who underwent lumpectomy were older, more likely to receive adjuvant radiation and endocrine therapy, and less likely to receive adjuvant chemotherapy. Patients who underwent lumpectomy demonstrated greater increases in scores over time for satisfaction with breasts, psychosocial well-being, and sexual well-being compared with patients who underwent mastectomy, after adjusting for the abovementioned covariates and receipt of reconstruction. The lumpectomy group had a larger decline in physical well-being over time compared with the mastectomy group. Conclusions: Patients who underwent lumpectomy demonstrated greater satisfaction with their breasts, psychosocial well-being, and sexual well-being but worse physical well-being over the first year after surgery compared with patients who underwent mastectomy. These results may help inform early-stage breast cancer patients making decisions about their surgical care. [ABSTRACT FROM AUTHOR]

Background and Aims Methods Results Discussion The Charcot–Marie–Tooth Disease Health Index (CMT‐HI) is a disease‐specific, patient‐reported disease burden measure. As part of an international clinical trial readiness study, individuals with CMT1A (ages 18–75 years) underwent clinical outcome assessments (COAs), including the CMT‐HI, to capture their longitudinal perspective on the disease burden.Two hundred and fifteen participants underwent serial COAs including the CMT‐HI, CMT Functional Outcome Measure (CMT‐FOM), CMT Neuropathy Score (CMTNSv2R), and CMT Exam Score (CMTES/CMTES‐R). Correlations between the total and subscale scores for the CMT‐HI and other COAs were determined. Changes in the CMT‐HI scores over 12 months were assessed using paired t‐tests. The minimum clinically important difference (MCID) for the CMT‐HI and its subscales were calculated by anchoring to a participant global impression of change scale.At baseline, CMT1A participants were 44.5 ± 15 years old (range: 18–75) and 58% were women. The mean CMT‐HI was 25.7 ± 18.8 (range: 0–91.9; 100 reflecting maximal disease burden). The CMT‐HI correlated with the CMT‐FOM (r = .54, p < .0001), CMTNSv2R (r = .48, p < .0001), and CMTES/CMTES‐R (r = .52/r = .54, p < .0001). Disease burden was greater in women than in men (CMT‐HI 29.1 ± 19.1 vs. 21.2 ± 17.3, p = .001). Over 12 months, there was a nonsignificant mean increase in CMT‐HI of 0.40 ± 10.0 (n = 189, p = .89). The MCID for the CMT‐HI total score was 3.8 points (95% CI: 1.7–5.9).Patient‐reported disease burden in CMT1A as measured by the CMT‐HI is associated with measures of neurologic impairment and physical functioning. Women reported a higher disease burden than men. These data will inform the design of clinical trials in CMT1A. [ABSTRACT FROM AUTHOR]

Aim Materials and Methods Results Conclusions To analyse the potential drivers (glucose level, complications, diabetes type, gender, age and mental health) of diabetes symptoms using continuous glucose monitoring (CGM) and ecological momentary assessment.Participants used a smartphone application to rate 25 diabetes symptoms in their daily lives over 8 days. These symptoms were grouped into four blocks so that each symptom was rated six times on 2 days (noon, afternoon and evening). The symptom ratings were associated with the glucose levels for the previous 2 hours, measured with CGM. Linear mixed‐effects models were used, allowing for nested random effects and the conduct of N = 1 analysis of individual associations.In total, 192 individuals with type 1 diabetes and 179 with type 2 diabetes completed 6380 app check‐ins. Four symptoms showed a significant negative association with glucose values, indicating higher ratings at lower glucose (speech difficulties, P = .003; coordination problems, P = .00005; confusion, P = .049; and food cravings, P = .0003). Four symptoms showed a significant positive association with glucose values, indicating higher scores at higher glucose (thirst, P = .0001; urination, P = .0003; taste disturbances, P = .021; and itching, P = .0120). There were also significant positive associations between microangiopathy and eight symptoms. Elevated depression and diabetes distress were associated with higher symptom scores. N = 1 analysis showed highly idiosyncratic associations between symptom reports and glucose levels.The N = 1 analysis facilitated the creation of personalized symptom profiles related to glucose levels with consideration of factors such as complications, gender, body mass index, depression and diabetes distress. This approach can enhance precision monitoring for diabetes symptoms in precision medicine. [ABSTRACT FROM AUTHOR]

Background The associations between self-reported chronic kidney disease–associated pruritus (CKD-aP) and patient-reported outcomes (PROs) have been reported using various instruments to assess itch. Data collection via multiple CKD-aP instruments allows the evaluation of different domains and measurements of CKD-aP burden and may help tailor data capture for future research or clinical care. Methods An electronic PRO (ePRO) survey was distributed to European hemodialysis (HD) patients enrolled in the Dialysis Outcomes and Practice Patterns Study (DOPPS) in 2021–23. The DOPPS is an international cohort study that aims to investigate practice patterns and outcomes in HD patients. The ePRO survey included multiple CKD-aP instruments: Average Itch and Worst Itching Intensity Numerical Rating Scales (AI-NRS, WI-NRS) and a Kidney Disease Quality of Life (KDQOL)-36 single question. Linear and logistic regression were used to estimate adjusted associations between CKD-aP instruments and various PROs. Results This analysis included 769 patients who completed the WI-NRS from HD facilities in France, Germany, Italy, Spain, Sweden and the UK. The correlation between WI-NRS and the KDQOL-36 itch question was 0.88 overall and 0.46 among patients at least somewhat bothered by itch. Mean WI-NRS scores stratified by response to the KDQOL-36 itch question were 8.1, 6.4, 4.1 and 3.1 for extremely, very much, moderately and somewhat bothered, respectively. Patients with worse WI-NRS scores reported worse sleep quality, greater fatigue, more depressive symptoms, and lower mental and physical quality of life; these associations were similar to those observed for the KDQOL-36 itch question. Conclusion Correlation between CKD-aP instruments was high overall, but moderate among the subgroup of patients bothered by itch; differences can be partially attributed to the recall period for the KDQOL-36 (4 weeks) vs the AI- and WI-NRS (24 h). The consistent associations of these instruments with poor outcomes underscores the importance to identify and effectively treat HD patients suffering from pruritus. [ABSTRACT FROM AUTHOR]