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Background Context: Lumbar spine fusion (LSF) surgery is a viable form of treatment for several spinal disorders. Treatment effects are preferably to be endorsed in real-life settings. Methods: This prospective study evaluated the 10-year outcomes of LSF. A population-based series of elective LSFs performed at 2 spine centers between January 2008 and June 2012 were enrolled. Surgeries for tumor, acute fracture, or infection, neuromuscular scoliosis, or postoperative conditions were excluded. The following patient-reported outcome measures (PROMs) were collected at baseline, and 1, 2, 5, and 10 years postsurgery: VAS for back and leg pain, ODI, SF-36. Longitudinal measures of PROMs were analyzed using mixed-effects models. Results: A total of 683 patients met the inclusion criteria, and 630 (92%) of them completed baseline and at least 1 follow-up PROMs, and they constituted the study population. Mean age was 61 (SD 12) years, 69% women. According to surgical indication, patients were stratified into degenerative spondylolisthesis (DS, n=332, 53%), spinal stenosis (SS, n=102, 16%), isthmic spondylolisthesis (IS, n=97, 15%), degenerative disc disease (DDD, n=52, 8%), and deformity (DF, n=47, 7%).All diagnostic cohorts demonstrated significant improvement at 1 year, followed by a partial loss of benefits by 10 years. ODI baselines and changes at 1 and 10 years were: (DS) 45, −21, and −14; (SS) 51, −24, and −13; (IS) 41, −24, and −20; (DDD) 50, −20, and −20; and (DF) 50, −21, and −16, respectively. Comparable patterns were seen in pain scores. Significant HRQoL achievements were recorded in all cohorts, greatest in physical domains, but also substantial in mental aspects of HRQoL. Conclusions: Benefits of LSF were partially lost but still meaningful at 10 years of surgery. Long-term benefits seemed milder with degenerative conditions, reflecting the progress of the ongoing spinal degeneration. Benefits were most overt in pain and physical function measures.

Background: There exist substantial patient barriers to accessing medications for opioid use disorder (MOUD), including travel distance, stigma, and availability of MOUD providers. Yet, despite these barriers, there exists a subset of patients who possess the requisite motivation to seek and remain adherent to treatment. Objective: To explore patient-derived goals in MOUD treatment-adherent patients. Methods: This study used in-depth interviews with patients receiving methadone who were enrolled in opioid treatment programs (OTPs) across Tennessee. Participants were recruited from 12 different OTPs to participate in telephonic semi-structured interviews to a point of saturation. Participants had to be adherent to treatment, in treatment for 6 months or greater, and English speaking. Analysis occurred inductively using a constructivist approach to Grounded Theory. Results: In total, 17 patient interviews were conducted in the spring of 2021. Participants described goal setting across three general stages of treatment: (1) addressing acute physical and emotional needs upon treatment entry, (2) development of supportive structure and routine to develop healthy skills facilitated by treatment team, and (3) identifying and pursuing future-focused goals not directly linked to treatment. A Proximal Goals in MOUD Framework is introduced. Conclusion: In this qualitative study on patient reported goals in MOUD it was found that goals are transitory and relative to the stage of treatment. Further research is needed to better understand goal evolution over the course of treatment and its impact on treatment retention.

Background: Reverse total shoulder arthroplasty (rTSA) is a relatively new procedure, only gaining approval in the United States in 2003. For these reasons, we sought to expand upon the breadth of current literature reporting on the long-term outcomes following this increasingly common orthopedic procedure. Case details: An institutional database was utilized to identify patients who underwent rTSA with a minimum postoperative follow-up of ten years. The five patients with the longest follow-up and complete clinical measures were ultimately included. Demographics, patient reported outcomes (Visual Analog Score (VAS), American Shoulder and Elbow Surgeon (ASES), Simple Shoulder Test (SST)), range of motion (Forward Elevation, Internal Rotation, External Rotation), and various radiographic measures were subsequently analyzed. The mean follow-up time was 11.6 ± 1.7 years. Patients saw improvements in VAS (pre-op.: 4.8 vs post-op: 3.2), ASES (pre-op.: 38.0 vs post-op: 58.0), SST (pre-op.: 2.6 vs post-op: 5.4), and Forward Elevation (pre-op.: 124° vs post-op: 142°) scores. However, internal and external rotation decreased by two vertebral levels and 14°, respectively. Only one shoulder failed during this follow-up time requiring revision surgery at 5.8 years. The overall survival was 100.0% and 80.0% at five- and ten-years follow-up, respectively, with a predicted implant survival time of 12.8 ± 1.6 years. Conclusion: Despite its relative novelty, rTSA is proving to be a sustainable procedure for patients with regards to pain, shoulder functionality, and implant survival, however, range of motion may not be completely restored. Overall, rTSA acts as a viable option for patients requiring shoulder replacement.

Background: Erythropoietic protoporphyria is a rare, inherited disorder presenting in early childhood with severe, painful phototoxicity. EPP has significant impacts on health-related quality of life, though there is variable disease severity. Accurately capturing how much time individuals with EPP can spend outdoors before they develop symptoms is critical to understanding HRQoL and measuring therapeutic response. Therefore, the goal of this study was to develop a comprehensive and content valid sun exposure diary to assess the efficacy of new therapies in individuals with EPP. Methods: Qualitative interviews were conducted with adult and adolescent EPP participants, as well as five clinical experts, to obtain their input on the content of an existing sun exposure diary. Revisions to the diary were made based on evidence generated in cognitive debriefing interviews analyzed in eight consecutive groups of EPP participant. Results: Interviews were conducted with 17 adults and 6 adolescents with EPP. The average age of adults was 40 years and of adolescents was 14 years. Clinical experts thought the original diary needed clarification on the description of symptoms, how time outdoors was captured, and the distinction between direct vs. indirect sunlight. Participants with EPP also noted these items needed revision, and that the distinction between prodromal symptoms and full reaction symptoms should be clarified. In the final diary version, participants with EPP found most items to be clear and easy to complete/think about. Seventy-six percent of participants (13/17) asked thought the diary was easy to complete. The remainder thought the majority of the diary was easy to complete with the exception of select questions. Conclusions: Evaluating a new treatment for EPP requires accurately capturing time in sunlight and symptoms in this unique disorder. The newly developed sun exposure diary is content valid and can be used to assess important aspects of symptoms and daily life and therefore evaluate clinically meaningful therapeutic response. [ABSTRACT FROM AUTHOR]

Background: This proof-of-concept retrospective case study investigated whether patient-reported outcomes (PRO) instruments, designed to capture symptomatic adverse event data, could identity a known exposure–response (ER) relationship for safety characterized in an original FDA analysis of an approved anti-cancer agent. PRO instruments have been designed to uniquely quantify the tolerability aspects of exposure-associated symptomatic adverse events. We explored whether standard ER analyses of clinician-reported safety data for symptomatic adverse events could be complemented by ER analysis using PRO data that capture and quantify the tolerability aspects of these same symptomatic adverse events. Methods: Exposure-associated adverse event data for diarrhea were analyzed in parallel in 120 patients enrolled in a clinical trial using physician reported Common Terminology Criteria for Adverse Events (CTCAE) and patient-reported symptomatic adverse event data captured by the National Cancer Institute's (NCI) PRO Common Terminology Criteria for Adverse Events (PRO-CTCAE) instrument. Comparative ER analyses of diarrhea were conducted using the same dataset. Results from the CTCAE and PRO-CTCAE ER analyses were assessed for consistency with the ER relationship for diarrhea established in the original NDA using a 750-patient dataset. The analysis was limited to the 120-patient subset with parallel CTCAE and PRO-CTCAE assessments. Results: Within the same 120-patient dataset, ER analysis using dense, longitudinal PRO-CTCAE-derived data was sensitive to identify the known ER relationship for diarrhea, whereas the standard CTCAE based ER analysis was not. Conclusions: ER analysis using PRO assessed symptomatic adverse event data may be a sensitive tool to complement traditional ER analysis. Improved identification of relationships for safety, by including quantification of the tolerability aspect of symptomatic adverse events using PRO instruments, may be useful to improve the sensitivity of exposure response analysis to support early clinical trial dosage optimization strategies, where decision making occurs within limited small patient datasets. [ABSTRACT FROM AUTHOR]

Aims: To reassess the 6‐month efficacy and to assess the 12‐month sustained efficacy of the MiniMed™ 780G advanced hybrid closed‐loop automated insulin delivery (AID) system compared to multiple daily injections plus intermittently scanned glucose monitoring (MDI+isCGM) in people with type 1 diabetes not meeting glucose targets. Methods: The ADAPT study was a prospective, multicentre, open‐label, randomized control trial in people with type 1 diabetes, with a glycated haemoglobin (HbA1c) concentration of at least 8.0% (64 mmol/mol), on MDI+isCGM therapy. After a 6‐month study phase, participants randomized at baseline to MDI+isCGM switched to AID (SWITCH) while the others continued AID therapy (SUSTAIN) for an additional 6 months. The primary endpoint of this continuation phase was the within‐group change in mean HbA1c between 6 and 12 months, with superiority in the SWITCH group and noninferiority in the SUSTAIN group (ClinicalTrials.gov: NCT04235504). Results: A total of 39 SWITCH and 36 SUSTAIN participants entered the continuation phase. In the SWITCH group, HbA1c was significantly decreased by −1.4% (95% confidence interval [CI] −1.7% to −1.1%; P < 0.001) from a mean ± SD of 8.9% ± 0.8% (73.9 ± 8.6 mmol/mol) at 6 months to 7.5% ± 0.6% (58.5 ± 6.9 mmol/mol) at 12 months. Mean HbA1c increased by 0.1% (95% CI −0.05% to +0.25%), from 7.3% ± 0.6% (56.5 ± 6.7 mmol/mol) to 7.4% ± 0.8% (57.7 ± 9.1 mmol/mol) in the SUSTAIN group, meeting noninferiority criteria. Three severe hypoglycaemia events occurred in two SWITCH participants during the continuation phase. Conclusion: ADAPT study phase glycaemic improvements were reproduced and sustained in the continuation phase, supporting the early adoption of AID therapy in people with type 1 diabetes not meeting glucose targets on MDI therapy. [ABSTRACT FROM AUTHOR]

Objective: Assess quality of life and mental health implications of mastectomy for breast cancer on sub-Saharan African women. Background: Mortality rates amongst women diagnosed with breast cancer in sub-Saharan Africa (SSA) are high, with disparities in survival relative to women in high income countries partly attributed to advanced disease at presentation. Fears of the sequelae of mastectomy are a prominent reason for presentation delays. There is a critical need to better understand the effects of mastectomy on women in SSA to inform preoperative counseling and education for women with breast cancer. Methods: Women with breast cancer in Ghana and Ethiopia undergoing mastectomy were followed prospectively. Breast related quality-of-life and mental health measures were evaluated preoperatively, 3 and 6 months postoperatively, using BREAST-Q, PHQ-9, and GAD-7. Bivariate and logistic regression analyses evaluated changes in these measures for the total cohort and between sites. Results: A total of 133 women from Ghana and Ethiopia were recruited. The majority of women presented with unilateral disease (99%) and underwent unilateral mastectomy (98%) with axillary lymph node dissection. Radiation was more common in Ghana (P < 0.001). Across most BREAST-Q subscales, women from both countries reported significantly decreased scores at 3 months postoperative. At 6 months, the combined cohort reported decreased scores for breast satisfaction (mean difference, -3.4). Women in both countries reported similar improvements in anxiety and depression scores postoperatively. Conclusions: Women from Ghana and Ethiopia who underwent mastectomy experienced a decline in breast-related body image while also experiencing decreased levels of depression and anxiety. [ABSTRACT FROM AUTHOR]

Background: The goals of this study are to describe the reoperation rates in patients who underwent Wilson osteotomy compared with patients who underwent carpometacarpal (CMC) arthroplasty for early-stage arthritis and to evaluate the factors influencing the patient-reported outcomes. Methods: Retrospectively, 52 patients who underwent surgery for stage I/II osteoarthritis of the thumb carpometacarpal were identified, consisting of 17 (33%) patients who underwent Wilson osteotomy and 35 (67%) who underwent carpometacarpal arthroplasty. A total of 28 (55%) patients completed the outcome questionnaires, consisting of 11 (39%) patients who underwent Wilson osteotomy and 17 (61%) patients who underwent carpometacarpal arthroplasty. We performed a multivariable linear regression model to identify factors associated with the Numeric Rating Scale (NRS) pain intensity at final follow-up. Results: Among the patients who underwent CMC arthroplasty, 2 had a reoperation. Among the patients who underwent Wilson osteotomy, 3 had a reoperation. Among the patients who completed the outcome questionnaires, the median quick Disabilities of the Arm, Shoulder and Hand score was 10 and the median NRS Pain Intensity score was 0. In multivariable analysis, the postoperative Patient-Reported Outcomes Measurement Information System Pain Interference (PROMIS PI) was independently associated with higher postoperative NRS pain scores. Conclusion: In younger patients with stage I/II CMC osteoarthritis, Wilson osteotomy may be a reasonable alternative to CMC arthroplasty. Outcomes were similar between both groups at mid-term follow-up, with only a slightly higher pain score in the osteotomy group. In patients with stage I/II carpometacarpal osteoarthritis, the PROMIS PI is the main factor indicating successful outcomes. [ABSTRACT FROM AUTHOR]

The journey of metastatic colorectal cancer patients is complex and challenging, requiring coordination and collaboration between multiple healthcare providers. Understanding patients' needs, fears, feelings, concerns, and behaviors is essential for providing individualized patient-centered care. In recent years, mCRC patients have experienced improvements in clinical outcomes, from 16 months of overall survival to 32 months, thanks to research. However, there is still room for improvement, and integrating clinical and translational research into routine practice can help patients benefit from treatments and techniques that would not be an option. In the Journey of mCRC patients, living well with cancer and quality of life becomes a priority given the outcomes of the disease. Patient reported outcomes (PRO) and Patient Reported Outcome Measures (PROMs) are becoming therefore new estimands in Oncology. Patient advocates represent important figures in this process by prioritizing issues and research questions; evaluating research designs and the performance of the research; the analysis and interpretation of data; and how results are disseminated. Multidisciplinary Tumor Boards and shared decision-making is essential for designing treatment strategies for individual patients. Quality of Life is often prioritized only when it comes to refractory advanced disease and end-of-life care, but it has to be integrated from the beginning, as the emotional impact of diagnosis leads to a vulnerable situation where patients' needs and preferences can be easily overseen. First-line treatment will be chosen among more treatment options than subsequent lines, with longer progression-free survival and a bigger impact on the outcomes. Practicing patient-centered care and optimizing first-line treatment for colorectal cancer patients requires a comprehensive understanding of patient experience and treatment outcomes, which can guide clinical practice and inform regulatory decisions for the benefit of patients. [ABSTRACT FROM AUTHOR]

Global disease registries are critical to capturing common patient related information on rare illnesses, allowing patients and their families to provide information about their condition in a safe, accessible, and engaging manner that enables researchers to undertake critical research aimed at improving outcomes. Typically, English is the default language of choice for these global digital health platforms. Unfortunately, language barriers can significantly inhibit participation from non-English speaking participants. In addition, there is potential for compromises in data quality and completeness. In contrast, multinational commercial entities provide access to their websites in the local language of the country they are operating in, and often provide multiple options reflecting ethnic diversity. This paper presents a case study of how the Global Angelman Syndrome Registry (GASR) has used a novel approach to enable multiple language translations for its website. Using a "semi-automated language translation" approach, the GASR, which was originally launched in English in September 2016, is now available in several other languages. In 2020, the GASR adopted a novel approach using crowd-sourcing and machine translation tools leading to the availability of the GASR in Spanish, Traditional Chinese, Italian, and Hindi. As a result, enrolments increased by 124% percent for Spain, 67% percent for Latin America, 46% percent for Asia, 24% for Italy, and 43% for India. We describe our approach here, which we believe presents an opportunity for cost-effective and timely translations responsive to changes to the registry and helps build and maintain engagement with global disease communities. [ABSTRACT FROM AUTHOR]

Background: The COVID-19 pandemic forced otolaryngologists to seek new methods of providing patient care in a remote setting. The effect of this paradigm shift on patient satisfaction, however, remains unelucidated. This study compares patient satisfaction with telehealth visits during the COVID-19 pandemic to that with in-office visits during the same period in 2019. Methods: Press Ganey survey responses of patients seen by otolaryngologists within a large, academic, multicenter hospital system were gathered. Responses were included in analyses if they corresponded with a visit that occurred either in clinic March to December 2019 or via telehealth March to December 2020. Chi-Square Test of Independence and Fisher's Exact Test were employed to detect differences between years. Binary logistic regressions were performed to detect the factors most predictive of positive telehealth experiences. Results: Patient overall satisfaction with in-office and telehealth visits did not differ significantly (76.4% in 2019 vs 78.0% in 2020 rated visit overall as "very good," P =.09). Patients seen by a Head and Neck (odds ratio 4.13, 95% confidence interval 1.52-11.26, P =.005), Laryngology (OR 5.96, 95% CI 1.51-23.50, P =.01), or Rhinology (OR 4.02, 95% CI 1.55-10.43, P =.004) provider were significantly more likely to report a positive telehealth experience. Conclusions: Patients seen via telehealth during COVID-19 reported levels of satisfaction similar to those seen in-office the year prior. These telehealth satisfaction levels, however, are contextualized within the expected confines of a pandemic. Further research is required to determine whether satisfaction remains consistent as telemedicine becomes a ubiquitous component of medical practice. [ABSTRACT FROM AUTHOR]

Introduction: Ofatumumab (Kesimpta™) is a s.c. applicable anti-CD20 antibody, which has been used in Germany since 2021 for the treatment of relapsing multiple sclerosis (RMS). The self-application offers a high degree of independence from intravenous forms of application with highly effective immunotherapy. In this study we recorded the patient-centered experience in 99 out of 127 patients who were adjusted to the drug by us. The aim was to investigate the tolerability and acceptance from the patient's perspective. Methods: Data collection was carried out using doctor documentation, questionnaires and telephone interviews. Results: The cohort consists of 127 patients. The patients received 2.8 (± SD 1.7) pre-therapies. The mean duration of therapy with Ofatumumab was 9.8 months (± SD 3.5). Structured data were collected from 99 patients. 23% of patients had no side effects during initial application. 19% rated the side effects as "very mild" and 18% as "mild". In addition to chills/fever (48%), headache (46%), limb pain (45%) and "other symptoms" (19%) also occurred. For subsequent injections, 72% of patients reported no side effects. 87% of patients found handling the medication "very easy". There was one relapse event during therapy. Conclusion: Our study shows that Ofatumumab is well accepted and tolerated by patients. There was one relapse event during the observation period. The side effects are mild and occur during initial application. No increased tendency to infection could be observed. The data suggest that Ofatumumab is also an effective and safe treatment option for patients with relapsing remitting multiple sclerosis in real-world use. [ABSTRACT FROM AUTHOR]

Several minimally invasive lumbar interbody fusion techniques may be used as a treatment for spondylolisthesis to alleviate back and leg pain, improve function and provide stability to the spine. Surgeons may choose an anterolateral or posterior approach for the surgery however, there remains a lack of real-world evidence from comparative, prospective studies on effectiveness and safety with relatively large, geographically diverse samples and involving multiple surgical approaches. To test the hypothesis that anterolateral and posterior minimally invasive approaches are equally effective in treating patients with spondylolisthesis affecting one or two segments at 3-months follow-up and to report and compare patient reported outcomes and safety profiles between patients at 12-months post-surgery. Prospective, multicenter, international, observational cohort study. Patients with degenerative or isthmic spondylolisthesis who underwent 1- or 2-level minimally invasive lumbar interbody fusion. Patient reported outcomes assessing disability (ODI), back pain (VAS), leg pain (VAS) and quality of life (EuroQol 5D-3L) at 4-weeks, 3-months and 12-months follow-up; adverse events up to 12-months; and fusion status at 12-months post-surgery using X-ray and/or CT-scan. The primary study outcome is improvement in ODI score at 3-months. Eligible patients from 26 sites across Europe, Latin America and Asia were consecutively enrolled. Surgeons with experience in minimally invasive lumbar interbody fusion procedures used, according to clinical judgement, either an anterolateral (ie, ALIF, DLIF, OLIF) or posterior (MIDLF, PLIF, TLIF) approach. Mean improvement in disability (ODI) was compared between groups using ANCOVA with baseline ODI score used as a covariate. Paired t -tests were used to examine change from baseline in PRO for both surgical approaches at each timepoint after surgery. A secondary ANCOVA using a propensity score as a covariate was used to test the robustness of conclusions drawn from the between group comparison. Participants receiving an anterolateral approach (n=114) compared to those receiving a posterior approach (n=112) were younger (56.9 vs 62.0 years, p <.001), more likely to be employed (49.1% vs 25.0%, p<.001), have isthmic spondylolisthesis (38.6% vs 16.1%, p<.001) and less likely to only have central or lateral recess stenosis (44.9% vs 68.4%, p=.004). There were no statistically significant differences between the groups for gender, BMI, tobacco use, duration of conservative care, grade of spondylolisthesis, or the presence of stenosis. At 3-months follow-up there was no difference in the amount of improvement in ODI between the anterolateral and posterior groups (23.2 ± 21.3 vs 25.8 ± 19.5, p=.521). There were no clinically meaningful differences between the groups on mean improvement for back- and leg-pain, disability, or quality of life until the 12-months follow-up. Fusion rates of those assessed (n=158; 70% of the sample), were equivalent between groups (anterolateral, 72/88 [81.8%] fused vs posterior, 61/70 [87.1%] fused; p=.390). Patients with degenerative lumbar disease and spondylolisthesis who underwent minimally invasive lumbar interbody fusion presented statistically significant and clinically meaningful improvements from baseline up to 12-months follow-up. There were no clinically relevant differences between patients operated on using an anterolateral or posterior approach. • Real world data from a large global sample of patients with spondylolisthesis. • Clinically significant improvement up to 12-months after minimally invasive fusion. • No clinically relevant differences between anterolateral and posterior approaches. [ABSTRACT FROM AUTHOR]

Although incorporating patient reported outcomes (PROMs) into practice allows healthcare systems to evaluate the value of care provided, research and policy reflecting PROMs can only be valid if they represent all patients. Few studies have evaluated socioeconomic barriers to PROM completion, and none have done so in a spine patient population. To identify patient barriers to PROM completion one year following lumbar spine fusion. Retrospective single-institution cohort study. A total of 2,984 patients undergoing lumbar fusion between 2014 and 2020 Completion of Mental Component Score (MCS-12) and Physical Component Score (PCS-12) of Short Form-12 questionnaire 1 year postoperatively. A retrospective review was conducted of all patients undergoing 1-3-level lumbar fusion at a single urban tertiary center. PROMs were queried from our prospectively managed electronic outcomes database. Patients were considered to have complete PROMs if 1-year outcomes were available. Community-level characteristics were collected from patients' zip codes using the Economic Innovation Group Distressed Communities Index. Bivariate analyses were performed to assess factors associated with PROM incompletion along with multivariate logistic regression to control for confounders. A total of 1,968 (66.0%) had incomplete 1-year PROMs. Patients with incomplete PROMs were more likely to be Black (14.5% vs 9.3%, p<.001), Hispanic (2.9% vs 1.6%, p=.027), reside in a distressed community (14.7% vs 8.5%, p<.001), and be active smokers (22.4% vs 15.5%, p<.001). On multivariate regression, Black race (OR: 1.46, p=.014, Hispanic ethnicity (OR: 2.19, p=.027), distressed community status (OR: 1.47, p=.024), workers' compensation status (OR: 2.82, p=.001), and active smoking (OR:1.31, p=.034) all were independently associated with PROM incompletion. Surgical characteristics, including primary surgeon, revision status, approach, and levels fused were not associated with PROM incompletion. Social determinants of health impact completion of PROMs. Patients completing PROMs are overwhelmingly White, non-Hispanic, and reside in wealthier communities. Efforts should be taken to provide better education regarding PROMs and ensure closer follow-up of certain subgroups of patients to avoid furthering disparities in PROM research. [ABSTRACT FROM AUTHOR]

Introduction: Poorly developed patient-reported outcome measures (PROs) risk type-II errors (i.e. false negatives) in clinical trials, resulting in erroneous failure to achieve trial endpoints. Validity is a fundamental requirement of fit-for-purpose PROs, with the main determinant of validity being the PROs items, i.e. content validity. Here, we sought to identify fatigue PRO instruments used in multiple sclerosis (MS) studies and to assess the extent to which their development satisfied current content validity standards. Methods: We searched Embase® and Medline® for MS studies using fatigue-based PROs. Abstracts were screened, PROs identified, and their relevant development papers assessed against seven Consensus Standards for Measurement Instruments (COSMIN) criteria for content development. Results: From 3814 abstracts, 18 fatigue PROs met our inclusion criteria. Most PROs did not satisfy at least one COSMIN content validity standard. Frequent omissions during PRO development include: clearly defined constructs; conceptual frameworks; qualitative research in representative samples; and literature reviews. PRO development quality has improved significantly since FDA guidance was published (U = 10.0, p = 0.02). However, scatterplots and correlations between PRO COSMIN scores and citation frequency (rho = − 0.62) and clinical trials usage (rho = + 0.18) implied that PRO quality is unrelated to choice. COSMIN scores implied that the Fatigue Symptoms and Impact Questionnaire—Relapsing Multiple Sclerosis (FSIQ-RMS) and Neurological Fatigue Index—Multiple Sclerosis (NFI-MS) had the strongest evidence for adequate content validity. Conclusion: Most existing fatigue PROs do not meet COSMIN content validity requirements. Although two PROs scored well on aggregate (NFI-MS and FSIQ-RMS), our subsequent evaluation of the item sets that generated their scores implied that both PROs have weaker content validity than COSMIN suggests. This indicates that COSMIN criteria require further development, and raises significant concerns about how we have measured one of the most common and burdensome MS symptoms. A detailed head-to-head psychometric evaluation is needed to determine the impact of different PRO development qualities and the implications of the problems implied by our analyses, on measurement performance. Plain Language Summary: In MS clinical trials, impacts such as fatigue, walking ability, and quality of life, are measured using questionnaires—called patient-reported outcome measures—completed by people living with MS. The quality of these measures is fundamentally important. If poor quality patient-reported outcome measures are used, treatment benefits are easily missed or underestimated. We studied the quality of 18 fatigue patient-reported outcome measures previously used in MS studies. Specifically, we studied how the questionnaire questions were developed and scored them against recognised quality control standards. In general, the patient-reported outcome measures were poor. Only two scored reasonably well. One common weakness was that people living with MS were not involved during patient-reported outcome measure development. We also conducted novel examinations that went beyond the quality control standards. These test how well the questions relate back to the MS impacts they claim to measure. We found even the two best patient-reported outcome measures were poor. Our study had two findings. First, patient-reported outcome measures of MS fatigue are poor. Second, current standards for testing patient-reported outcome measure development are too easy to satisfy, overestimate patient-reported outcome measure quality, and need updating. Therefore, the ways we measure MS fatigue, one of the most common and burdensome MS symptoms, are scientifically weak. 3u_RvEza8RqxoERL84ceCF Measuring fatigue in multiple sclerosis: there may be trouble ahead—a video abstract (MP4 125165 KB) [ABSTRACT FROM AUTHOR]

This study aimed to evaluate feasibility, acceptability, reliability, and validity of the existing four‐item Shared Decision Making (SDM) Process Scale for use in evaluating genetic testing decisions. Patients from a large hereditary cancer genetics practice were invited to participate in a two‐part survey after completing pre‐test genetic counseling. The online survey included the SDM Process Scale and the SURE scale, a measure of decisional conflict. SDM Process scores were compared to SURE scores to test convergent validity, and respondents were sent a second survey 1 week later to assess retest reliability. The response rate was 65% (n = 259/398) and missing data was low (<1%). SDM scores ranged from zero to four with a mean of 2.3 (SD = 1.1). Retest reliability was good, with intraclass correlation of 0.84, 95% confidence interval (0.79, 0.88). No relationship was found between SDM Process scores and decisional conflict (p = 0.46), likely because 85% of participants reported no decisional conflict. The four‐item SDM Process Scale demonstrated feasibility, acceptability, and retest reliability, but not convergent validity with decisional conflict. These findings provide initial evidence for use of this scale to measure patient perceptions of SDM in pre‐test counseling for hereditary cancer genetic testing. [ABSTRACT FROM AUTHOR]

(1) Objective: We explore the predictive power of a novel stream of patient data, combining wearable devices and patient reported outcomes (PROs), using an AI-first approach to classify the health status of Parkinson's disease (PD), multiple sclerosis (MS) and stroke patients (collectively named PMSS). (2) Background: Recent studies acknowledge the burden of neurological disorders on patients and on the healthcare systems managing them. To address this, effort is invested in the digital transformation of health provisioning for PMSS patients. (3) Methods: We introduce the data collection journey within the ALAMEDA project, which continuously collects PRO data for a year through mobile applications and supplements them with data from minimally intrusive wearable devices (accelerometer bracelet, IMU sensor belt, ground force measuring insoles, and sleep mattress) worn for 1–2 weeks at each milestone. We present the data collection schedule and its feasibility, the mapping of medical predictor variables to wearable device capabilities and mobile application functionality. (4) Results: A novel combination of wearable devices and smartphone applications required for the desired analysis of motor, sleep, emotional and quality-of-life outcomes is introduced. AI-first analysis methods are presented that aim to uncover the prediction capability of diverse longitudinal and cross-sectional setups (in terms of standard medical test targets). Mobile application development and usage schedule facilitates the retention of patient engagement and compliance with the study protocol. [ABSTRACT FROM AUTHOR]

Background: Goal-oriented patientcare is a key element in qualityhealthcare. Medical-caregiver's (MC) are expected to generate a shared decision-making process with patients regarding goals and expected health-outcomes. Hip-fracture patients (HFP) are usually older-adults with multiple health-conditions, necessitating that agreed-upon goals regarding the rehabilitation process, take these conditions into consideration. This topic has yet to be investigated by pairing and comparing the perception of expected outcomes and therapeutic goals of multidisciplinary MCs and their HF patient's. Our aim was to assess in a quantitative method whether HFPs and their multidisciplinary MCs agree upon target health-outcomes and their most important goals as they are reflected in the SF12 questionnaire. Methods: This was a cross-sectional, multi-center, study of HFPs and their MCs. Patients and MCs were asked to rate their top three most important goals for rehabilitation from the SF12 eight subscales: physical functioning, physical role limitation, bodily pain, general health, vitality, social functioning, emotional role limitation and mental health, and indicate their expected outcome. Descriptive statistics and mixed effect logistic-regression were used to compare concordance of the ratings. Agreement between patients and MCs was assessed using interclass coefficients (ICCs). Results: A total of 378 ratings were collected from 52 patients, 12 nurses, 12 physicians and 6 paramedical personnel. Each patient had between 3 and 9 raters. Patients considered physical functioning and physical role limitation more important than did MCs. Physicians and nurses emphasized the importance of bodily pain while patients referred to it as relatively less significant. The total ICC was low (2%) indicating poor agreement between MCs and patients. With the exception of physical-functioning, MCs predicted a less optimistic outcome in all of the SF12's subscales in comparison to HFPs. Conclusion: Effective intervention in HFPs requires constructive communication between MCs and patients. The study suggests that caregivers have an insufficient understanding of the expectations of HFPs. More effective communication channels are required in order to better understand HFPs' needs and expectations. [ABSTRACT FROM AUTHOR]

Background: Hemodialysis (HD) patients commonly receive polypharmacy leading to increased likelihood of drug related problems (DRPs) and poor quality of life. Medication Therapy Management (MTM) services discover and resolve DRPs and may specifically improve Medication-burden Quality of life (MBQoL) in HD patients. We aimed to assess the effect of MTM services on DRPs and MBQoL among HD patients. Methods: A prospective pre-post study was conducted on 104 patients in an HD unit in Alexandria, Egypt. MBQoL was assessed at baseline and after three months of MTM sessions, using the Arabic, validated version of the Patient Reported Outcomes Measure of Pharmaceutical Therapy (PROMPT) questionnaire. Cohen's d test and multiple linear regression were used to assess the effect size of MTM and the factors affecting MBQoL, respectively. DRPs, adverse events and adherence were also monitored. Results: MBQoL improved significantly after the implementation of MTM (Cohen's d=0.88, p < 0.01) with the largest effect size in the "medicine information and relation with healthcare providers" domain. DRPs decreased significantly after MTM implementation (11.97 ± 4.65 versus 7.63 ± 3.85 per patient, p<0.001). The mean adverse events per patient were also reduced (9.69 ± 4.12 versus 6.56 ± 3.77, p < 0.001). Conclusion: Applying MTM services presents an opportunity to improve care for HD patients by improving MBQoL, decreasing DRPs and adverse events. [ABSTRACT FROM AUTHOR]

Purpose: The aims of this study were to evaluate long-term multidimensional fatigue in patients with brain metastases (BM) up to 21 months after Gamma Knife radiosurgery (GKRS) and (change in) fatigue as predictor of survival. Methods: Patients with 1 to 10 BM, expected survival > 3 months, and Karnofsky Performance Status ≥ 70, and Dutch non-cancer controls were included. Fatigue was measured with the Multidimensional Fatigue Inventory. Levels of fatigue between patients and controls were compared using independent-samples t-tests. Linear mixed models were used to evaluate fatigue within the patient group up to 21 months after GKRS. Pre-GKRS fatigue and minimal clinically important (MCI) changes in fatigue in the first three months (defined as a 2-point difference) after GKRS were evaluated as predictors of survival time. Results: Prior to GKRS, patients with BM (n = 92) experienced significantly higher fatigue on all subscales than controls (n = 104). Over 21 months, physical fatigue increased, and mental fatigue decreased significantly. More specifically, general, and physical fatigue increased significantly between pre-GKRS and 3 months, followed by stable scores between 3 (n = 67) and 6 (n = 53), 6 and 12 (n = 34) and 12 and 21 (n = 21) months. An MCI increase in general or physical fatigue over the first 3 months after GKRS was a significant predictor of shorter survival time. Conclusion: Except for mental fatigue, all aspects of fatigue remained elevated or further increased up to 21 months after treatment. Furthermore, an increase in general or physical fatigue within three months after GKRS may be a prognostic indicator for poorer survival. ClinicalTrials.gov identifier: NCT02953756, November 3, 2016. [ABSTRACT FROM AUTHOR]

Background: Verbal rating scales (VRS) are widely used in patient-reported outcome (PRO) measures. At our institution, patients complete an online instrument using VRSs with a five-point brief response scale to assess symptoms as part of routine follow-up after ambulatory cancer surgery. We received feedback from patients that the brief VRS descriptors such as "mild" or "somewhat" were vague. We added explicit descriptors to our VRSs, for instance, "Mild: I can generally ignore my pain" for pain severity or "Somewhat: I can do some things okay, but most of my daily activities are harder because of fatigue" for fatigue interference. We then compared responses before and after this change was made. Methods: The symptoms investigated were pain, fatigue and nausea. Our hypothesis was that the explicit descriptors would reduce overall variance. We therefore compared the coefficient of variation of scores and tested the association between symptoms scores and known predictors thereof. We also compared time to completion between questionnaires with and without the additional descriptors. Results: A total of 17,500 patients undergoing 21,497 operations were assigned questionnaires in the period before the descriptors were added; allowing for a short transition period, 1,417 patients having 1436 operations were assigned questionnaires with the additional descriptors. Symptom scores were about 10% lower with the additional descriptors but the coefficient of variation was slightly higher. Moreover, the only statistically significant difference between groups for association with a known predictor favored the item without the additional language for nausea severity (p = 0.004). Total completion time was longer when the instrument included the additional descriptors, particularly the first and second time that the questionnaire was completed. Conclusions: Adding descriptors to a VRS of post-operative symptoms did not improve scale properties in patients undergoing ambulatory cancer surgery. We have removed the additional descriptors from our tool. We recommend further comparative psychometric research using data from PROs collected as part of routine clinical care. [ABSTRACT FROM AUTHOR]

Introduction: Sarcomas are rare cancers and very heterogeneous in their location, histological subtype, and treatment. Health-Related Quality of Life (HRQoL) of sarcoma patients has rarely been investigated in longitudinal studies. Methods: Here, we assessed adult sarcoma patients and survivors between September 2017 and February 2020, and followed-up for one year in 39 study centers in Germany. Follow-up time points were 6 (t1) and 12 months (t2) after inclusion. We used a standardized, validated questionnaire (the European Organisation for Research and Treatment of Cancer Quality of Life Core Instrument (EORTC QLQ-C30) and explored predictors of HRQoL in two populations (all patients (Analysis 1), patients in ongoing complete remission (Analysis 2)) using generalized linear mixed models. Results: In total we included up to 1111 patients at baseline (915 at t1, and 847 at t2), thereof 387 participants were in complete remission at baseline (334 at t1, and 200 at t2). When analyzing all patients, HRQoL differed with regard to tumor locations: patients with sarcoma in lower extremities reported lower HRQoL values than patients with sarcomas in the upper extremities. Treatment which included radiotherapy and/or systemic therapy was associated with lower HRQoL. For patients in complete remission, smoking was associated with worse HRQoL-outcomes. In both analyses, bone sarcomas were associated with the worst HRQoL values. Being female, in the age group 55- <65 years, having lower socioeconomic status, and comorbidities were all associated with a lower HRQoL, in both analyses. Discussion: HRQoL increased partially over time since treatment and with sporting activities. HRQoL improved with time since treatment, although not in all domains, and was associated with lifestyle and socioeconomic factors. Bone sarcomas were the most affected subgroup. Methods to preserve and improve HRQoL should be developed for sarcoma patients. [ABSTRACT FROM AUTHOR]

Cancer-related fatigue (CRF) is a common side effect and remains under-diagnosed. Screening of CRF by physical therapists (PTs) and patient perspectives of their experiences has not been comprehensively examined. To survey PTs to understand the frequency of CRF screening, and to assess the knowledge and experiences of survivors as it relates to CRF. Two separate electronic surveys developed by the authors were distributed. One targeted oncology PTs, the other for adult survivors of cancer. Of the 199 PT respondents, 36% reported screening for CRF at every encounter. Screening included interviews (46%) and/or standardized questionnaires (37%). The most common barriers to receiving treatment for CRF was lack of physician referrals and time constraints. Of patient responses (n = 61), 84% reported CRF as an important ongoing issue; 77% reported that they initiated the discussion about CRF with their provider, and 23% reported being told there were treatment options for CRF. CRF is common among cancer survivors. However, consistent screening by PTs is lacking. Patients with CRF frequently initiated the conversation with their providers because of symptoms and many patients were not told of treatment options. These findings represent a substantial gap in clinical practice regarding CRF screening and management. [ABSTRACT FROM AUTHOR]

Background: When assessing the efficacy of a treatment in any clinical trial, it is recommended by the International Conference on Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multifaceted diseases, which is often the case in rare diseases. Therefore, the use of a combination of several clinically meaningful outcomes is preferred. Many methodologies that allow for combining outcomes in a so-called composite endpoint are however limited in a number of ways, not in the least in the number and type of outcomes that can be combined and in the poor small-sample properties. Moreover, patient reported outcomes, such as quality of life, often cannot be integrated in a composite analysis, in spite of their intrinsic value. Results: Recently, a class of non-parametric generalized pairwise comparisons tests have been proposed, which members do allow for any number and type of outcomes, including patient reported outcomes. The class enjoys good small-sample properties. Moreover, this very flexible class of methods allows for prioritizing the outcomes by clinical severity, allows for matched designs and for adding a threshold of clinical relevance. Our aim is to introduce the generalized pairwise comparison ideas and concepts for rare disease clinical trial analysis, and demonstrate their benefit in a post-hoc analysis of a small-sample trial in epidermolysis bullosa. More precisely, we will include a patient relevant outcome (Quality of life), in a composite endpoint. This publication is part of the European Joint Programme on Rare Diseases (EJP RD) series on innovative methodologies for rare diseases clinical trials, which is based on the webinars presented within the educational activity of EJP RD. This publication covers the webinar topic on composite endpoints in rare diseases and includes participants' response to a questionnaire on this topic. Conclusions: Generalized pairwise comparisons is a promising statistical methodology for evaluating any type of composite endpoints in rare disease trials and may allow a better evaluation of therapy efficacy including patients reported outcomes in addition to outcomes related to the diseases signs and symptoms. [ABSTRACT FROM AUTHOR]

Objectives: To compare post‐treatment neck and shoulder function between human papillomavirus‐associated oropharynx squamous cell carcinoma (HPV + OPSCC) treatments. Design: Prospective, repeated‐measures study. Setting: Tertiary care center. Participants: Treatment‐naïve patients with American Joint Committee on Cancer eighth edition stage T0‐3/N0‐2 HPV+OPSCC. Main Outcome Measures: Patients completed the Neck Dissection Impairment Index (NDII) pre‐treatment and 3‐months and 1‐year post‐treatment. The NDII assesses 10 neck and shoulder functions scored 0–5 (total score 0–100), with higher scores suggesting better function. Results: A total of 106 patients underwent: surgery alone (SA, n = 46, 43%), surgery with adjuvant radiation ± chemotherapy (S + a[C]XRT, n = 18, 17%), or definitive radiation ± chemotherapy (d[C]XRT, n = 42, 40%). cTN classification and pre‐treatment NDII scores did not differ between groups. SA patients reported worsened 3‐month post‐treatment versus pre‐treatment self‐care (4.6 vs. 5.0), lifting light (4.6 vs. 5.0) and heavy (4.2 vs. 4.8) objects, overhead reach (4.5 vs. 4.9), activity (4.5 vs. 4.9), socialization (4.7 vs. 4.9), recreation (4.6 vs. 4.9), and overall score (86.8 vs. 95.3) (all p < 0.05). One‐year post‐treatment scores (n = 34) were no different than pre‐treatment in all domains. S + a[C]XRT patients reported worsened 3‐month versus pre‐treatment stiffness (4.0 vs. 4.8), lifting heavy objects (3.8 vs. 4.9), overhead reach (4.2 vs. 4.9), socialization (4.6 vs. 5.0), recreation (4.4 vs. 4.9) and overall score (82.4 vs. 96.0) (all p < 0.05). One‐year post‐treatment scores (n = 13) were no different than pre‐treatment in all domains. d[C]XRT patients reported worsened 3‐month versus pre‐treatment difficulty lifting heavy objects (4.3 vs. 4.7) and recreation (4.3 vs. 4.7). One‐year posttreatment scores (n = 21) were no different than pre‐treatment in all domains. Conclusion: HPV + OPSCC patients may experience mild shoulder/neck dysfunction 3 months after treatment that usually resolves by 1 year, independent of treatment modality. [ABSTRACT FROM AUTHOR]

Introduction: Lichen planus (LP) is an inflammatory skin disorder that can present in various forms across the body, including lesions on the skin (cutaneous LP [CLP]), scalp (lichen planopilaris [LPP]), and mucosal regions (mucosal LP [MLP]). Qualitative exploration of the patient experience of LP, notably symptoms and impacts on health-related quality of life (HRQoL), is limited. A scarcity of research was also identified relating to emotional wellbeing impacts of CLP patients. Two qualitative studies were conducted with LP patients to address these gaps. Methods: Study 1 consisted of exit interviews conducted with a subset of adult patients with MLP (n = 5), CLP (n = 4), and LPP (n = 4) enrolled in an LP clinical study in the United States (US) to explore the patient experience. Study 2 consisted of independent qualitative interviews conducted with adult CLP patients (n = 13) from the US and Germany to further explore impacts on emotional wellbeing. Results: Exit interviews found that itch , pain, and skin lesions were most frequently reported as signs/symptoms of LP. Itch and skin lesions were experienced across all LP subtypes, while pain was only reported by CLP and MLP patients. These signs/symptoms impacted HRQoL including emotional wellbeing (frustration, embarrassment), daily activities (oral hygiene, clothing options), social functioning (intimacy, social activities), and physical functioning (chewing/swallowing, opening/moving mouth). Impacts on activities of daily living (ADL) and physical functioning were mostly experienced by MLP patients. Independent qualitative interviews, which further explored impacts of CLP on patients' emotional wellbeing, identified frustration, worry, sadness, embarrassment, and depression as the most frequently experienced. Conclusion: The findings contribute to the literature by providing qualitative insights into signs/symptoms and HRQoL impacts of LP, from the adult patient perspective. The findings also highlight the importance of considering assessment of HRQoL impacts in future clinical LP research, particularly impacts on emotional wellbeing when selecting instruments for assessment of HRQoL in the CLP population. Trial Registration: NCT04300296. [ABSTRACT FROM AUTHOR]

Cannabis use in patients undergoing arthroplasty has increased with ongoing legalization throughout the United States. The purpose of this study was to report total hip arthroplasty (THA) outcomes in patients self-reporting cannabis use. There were 74 patients who underwent primary THA from January 2014 to December 2019 at a single institution with minimum 1-year follow-up who had their self-reported cannabis use retrospectively reviewed. Patients who had a history of alcohol or illicit drug abuse were excluded. A match control was conducted based on age; body mass index; sex; Charlson Comorbidity Index; insurance status; and use of nicotine, narcotics, antidepressants, or benzodiazepines to patients undergoing THA who did not self-report cannabis use. Outcomes included Harris Hip Score (HHS), Hip Disability and Osteoarthritis Outcome Score for Joint Reconstruction (HOOS JR), in hospital morphine milligram equivalents (MMEs) consumed, outpatient MMEs prescribed, in hospital lengths of stay (LOS), postoperative complications, and readmissions. There was no difference in the preoperative, postoperative, or change in Harris Hip Score or HOOS JR between cohorts. There was also no difference in hospital MMEs consumed (102.4 versus 101, P =.92), outpatient MMEs prescribed (119 versus 156, P =.11) or lengths of stay (1.4 versus 1.5 days, P =.32). Also, readmissions (4 versus 4, P = 1.0) and reoperations (2 versus 1, P =.56) were not different between groups. Self-reported cannabis use does not influence 1-year outcomes after THA. Further studies are warranted to determine the efficacy and safety of perioperative cannabis use after THA to help guide orthopaedic surgeons in counseling patients. [ABSTRACT FROM AUTHOR]

To systematically identify all patient reported outcome measures (PROMs) (quality of life [QOL] instruments or other instrument/methodology) that have been used to date in aortic dissection (AD) and to explore how well these instruments evaluate QOL according to the Consensus based Standards for the selection of health Measurement Instruments (COSMIN) methodology or guideline. Embase, MEDLINE, PsycINFO, CINAHL, and Cochrane Library were search on 1st July 2022. This scoping review was undertaken according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for scoping reviews (PRISMA-ScR) and the COSMIN guidelines for performing systematic reviews of validated PROMs. Studies that reported on any aspect or domain of QOL using a PROM or other instrument or methodology on AD were included. Data synthesis, including psychometric property analysis and risk of bias assessment were performed according to COSMIN guidelines. Forty-five studies, published between 1994 and 2021 reporting on 5 874 patients (mean age 63 years, 70.6% male), were included. A total of 39 PROMs were used, and three studies used semi-structured interviews. The majority (69%) of studies were in patients with type A aortic dissection (TAAD). The most common PROM used was the SF-36 (51%). Six studies evaluated one or more psychometric properties of a PROM. Only one of these studies was specifically designed as a validation study. No study reported on content validity. Internal consistency was the most evaluated psychometric property. No study evaluated all the psychometric properties according to COSMIN methodology. The methodological quality used to assess these PROMs was judged to be adequate or very good. This review highlights the heterogeneity of PROMs or methods used to determine QOL in AD patients. The lack of research regarding a comprehensive evaluation of the psychometric properties of a PROM used in AD highlights the need for the development and validation of a dissection specific PROM. [PROSPERO registration no. CRD42022310477]. [ABSTRACT FROM AUTHOR]

To assess the effect of cosmetic rhinoplasty on PROMS using the FACE-Q™ tool. Between July 2020 and February 2022 all patients undergoing rhinoplasty by a single surgeon were approached pre-operatively and 6 months post-operatively to complete the Face-Q™ "Satisfaction with Nose" module. Post-operative patients were asked to complete the FACE-Q™ "Satisfaction with Outcome" module. One hundred and sixty-five patients underwent rhinoplasty (147 primary, 18 revisions). Eighty two percent (n = 135) completed a pre-operative "Satisfaction with Nose" module. Thirty three percent (n = 54) completed the full pre and post- operative dataset. The mean pre-operative "Satisfaction with Nose" score was 32.88 (± 8.40). The mean post-operative "Satisfaction with Nose" score was 77.45 (SD17.26) and "Satisfaction with Outcome" score was 75.27(SD 21.88). The mean change in score 133% (SD 63%). Seventy-seven percent of patients were "very satisfied" or "somewhat satisfied" across all 10 aspects of the nose. The tip had the lowest post-operative satisfaction with 19% of patients somewhat or very dissatisfied. Rhinoplasty generates significant improvements in PROMS and satisfaction is high, an important positive finding for patients considering surgery. Routine collection of PROMS for rhinoplasty can inform practice and guide expectations as to its psychological impact. [ABSTRACT FROM AUTHOR]

Objective: We recently described the development of the Neck Fibrosis Scale (NFS). In this submission, we confirm domain structure and validate a scoring system for the NFS. Study Design: Prospective cross‐sectional study. Methods: Between January 2020 and December 2021, 127 head and neck cancer patients with varying degrees of cutaneous neck fibrosis completed the original 15 item NFS. Exploratory factor analysis was used to identify optimal groupings with similar underlying factors. The association between the domains of the NFS and various measures of neck morbidity (i.e., construct validity) were assessed using gamma regression. Results: Exploratory factor analysis confirmed 13 of the 15 items from the NFS mapped onto two factors, which were labelled 'physical' and 'emotional' domains. Of the remaining two items, 'energy' did not load uniquely onto one factor and was removed. 'Neck‐swelling' did not load on either factor (loadings <0.3) but was retained within the physical domain based on clinical importance. This resulted in a revised 14‐item questionnaire. Internal consistency for these two domains was high (>0.8, p < 0.01). Both the physical and emotional domains of the revised NFS show strong correlation with the neck dissection impairment index and neck range of motion. The physical domain strongly correlated with neck elasticity (0.902 [95%CI 0.839–0.972], p < 0.01). Patients receiving multimodal therapy had physical domain scores that were 31.6% [95% 13.9–51.8] higher (worse) than unimodal therapy patients. Conclusions: A domain structure and scoring strategy have been developed for the NFS. Future efforts should be directed toward an evaluation of responsiveness. Level of Evidence: NA Laryngoscope, 133:2198–2202, 2023 [ABSTRACT FROM AUTHOR]

IntroductionInfants with SCID are treated with hematopoietic cell transplantation (HCT) or gene therapy (GT). Caregiver perceptions of pre-treatment counseling and understanding of durability of HCT/GT are poorly understood.MethodsA survey was designed and distributed to families of patients with SCID. Topics in the questionnaire included SCID genotype and treatment, family recollections of pre-treatment counseling and present clinical status.Results151 surveys were analyzed. 132 were treated with HCT, 19 with GT. From counseling received, 37% expected HCT/GT would lead to "cure"; 43% expected HCT/GT would last a lifetime. Of 136 living patients, 59% reported overall good health but 65% reported some persistent health challenges.ConclusionsFor some, interpretation of the word "cure" varied, leading to misunderstanding regarding need for continued medical evaluations and additional therapies. Clear communication regarding the importance of lifelong follow-up, no matter the treatment outcome, will help to optimize good health and quality of life.

Abstract Background Cancer and its treatment can have significant impacts on health status, quality of life and functioning of patients. Direct information from patients regarding these aspects can be collected via electronic platforms in the form of electronic Patient Reported Outcome Measures (ePROMs). Research has shown that the use of ePROMS in cancer care leads to improved communication, better symptom control, prolonged survival and a reduction in hospital admissions and emergency department attendance. Acceptability and feasibility of routine ePROM collection has been reported by both patients and clinicians but to date their use has predominantly been limited to clinical trials. MyChristie-MyHealth is an initiative from a UK comprehensive cancer centre The Christie NHS Foundation Trust which incorporates the regular collection of ePROMs into routine cancer care. This study, carried out as part of a service evaluation, explores patient and clinician experiences of using the MyChristie-MyHealth ePROMs service. Results 100 patients with lung and head and neck cancers completed a Patient Reported Experience questionnaire. All patients reported that MyChristie-MyHealth was easy to understand and, almost all found it timely to complete and easy to follow. Most patients (82%) reported it improved their communication with their oncology team and helped them to feel more involved with their care (88%). A large proportion of clinicians (8/11) felt ePROMs helped communication with their patients and over half (6/10) felt they led to consultations being more patient focused. Clinicians also felt that the use of ePROMs resulted in patients being more engaged in consultations (7/11) and their cancer care in general (5/11). Five clinicians reported that the use of ePROMs altered their clinical decision making. Conclusions Regular ePROMs collection as part of routine cancer care is acceptable to both patients and clinicians. Both patients and clinicians feel their use improved communication and increased the feeling of patient involvement with their care. Further work is needed to explore the experiences of patients that did not complete the ePROMs as part of the initiative and to continue to optimize the service for both patients and clinicians.

Abstract Background In-office vocal fold injections (VFI) are an effective treatment for glottic insufficiency. The primary objective of this study was to assess if patients reported decisional regret after VFI. Secondary objectives included determining if variables were associated with lower decisional regret. Methods Case–control study of patients who underwent in-office VFIs for glottic insufficiency from August 2017 to December 2019 at a tertiary laryngology clinic. Participants completed the validated Decision Regret Scale (DRS). Demographic data, clinician’s perceptual analysis with GRBAS (Grade, Roughness, Breathiness, Asthenia, Strain), and patient’s self-reported Voice Handicap Index-10 (VHI-10) were analyzed. Nonparametric tests as well as univariate and multiple logistics regression were performed. Results Of patients eligible, 75% (136/182) completed the DRS (mean age 65.4 years (SD 13.9), 58.1% male). Eighty-three (61.0%) reported no decisional regret, thirty-three (24.3%) reported mild decisional regret, and twenty (14.7%) reported moderate to strong decisional regret. Improvement in most recent VHI-10 (Kendall correlation coefficient tau = 0.156, p = 0.029), Grade of voice (tau = 0.236, p value = 0.002) and Breathiness of voice (tau = 0.150, p = 0.044) were associated with lower DRS. Multivariate logistics regression results showed that the change in Grade of voice (OR 9.9, p

Cataract surgery has moved from the era of anti-blindness surgery to the era of refractive surgery, and vision-related quality of life has become an important prognostic indicator for cataract surgery. Since indicators like visual acuity are difficult to describe the visual quality of patients in a panoramic view, the evaluation of visual function based on subjective experience has received attention in clinical and scientific research. The visual functioning index(VF-14)is the first scale applied to assess the visual function in cataract surgery, and is widely used internationally so far. This review presented the research history of VF-14, scrutinized its clinical applicability, and summarized the research and application of VF-14 in the assessment of visual function, evaluation of surgical efficacy, and postoperative follow-up from three groups of people with cataract, refractive discomforts, and other common ophthalmic diseases, aiming to provide a reference for the clinical application of the VF-14.

Background: Ocrelizumab is an effective medication for multiple sclerosis. However, infusion-related reactions (IRRs) are a concern for patients and may lead to discontinuation of ocrelizumab. To minimize IRRs, pre-medications are administered. However, from our experience, these medications, especially diphenhydramine, can cause marked drowsiness. The primary objective of this study was to evaluate whether cetirizine is non-inferior to diphenhydramine in limiting the proportion and severity of reactions from ocrelizumab infusions. Methods: Twenty participants were serially randomized in a 1:1 ratio to receive 10 mg of cetirizine or 25 mg of diphenhydramine orally prior to their first three ocrelizumab infusions. Results: The rate of IRRs in this study was similar across both treatment groups with no increase in the risk of severity, and no grade 3 IRRs. Further, patients receiving cetirizine experienced a reduction in fatigue. While there was not a significant difference in global satisfaction, this score increased over time in the cetirizine arm while it remained unchanged in the diphenhydramine arm. Conclusions: Overall, our results suggest that cetirizine does not increase the risk of infusion-related reactions compared to diphenhydramine.

Clinical Scenario: Patients with chronic ankle instability (CAI) commonly display lower levels of self-reported function and health-related quality of life. Several rehabilitation interventions, including manual therapy, have been investigated to help CAI patients overcome these deficits. However, it is unclear if the addition of manual therapy to exercise-based rehabilitation is more effective than exercise-based rehabilitation alone. Clinical Question: Does incorporating manual therapy with exercise-based rehabilitation improve patient-reported outcomes when compared with exercise-based rehabilitation alone? Summary of Key Findings: The literature was searched for articles that examined the difference in outcomes for patients with CAI between manual therapy with exercise-based rehabilitation and exercise-based rehabilitation alone. A total of 3 peer-reviewed randomized controlled trials were identified. Two articles demonstrated improved patient-reported outcome scores following the incorporation of manual therapy with exercise-based rehabilitation, whereas one study found no statistically significant differences between interventions. Clinical Bottom Line: The current evidence suggests that incorporating manual therapy in addition to exercised-based rehabilitation may improve patient-reported outcome scores in patients with CAI. Strength of Recommendation: In accordance with the Strength of Recommendation Taxonomy, the grade of A is recommended due to consistent evidence from high-quality studies. [ABSTRACT FROM AUTHOR]

Context: Lateral ankle sprains (LAS) have one of the highest recurrence rates of all musculoskeletal injuries. An emphasis on rapid return to sport (RTS) following LAS likely increases reinjury risk. Unfortunately, no set of objective RTS criteria exist for LAS, forcing practitioners to rely on their own opinion of when a patient is ready to RTS. Purpose: To determine if there was consensus among published expert opinions that could help inform an initial set of RTS criteria for LAS that could be investigated in future research. Evidence Acquisition: PubMed, CINHL, and SPORTDiscus databases were searched from inception until October 2018 using a combination of keywords. Studies were included if they listed specific RTS criteria for LAS. No assessment of methodological quality was conducted because all included papers were expert opinion papers (level 5 evidence). Extracted data included the recommended domains (eg, range of motion, balance, sport-specific movement, etc) to be assessed, specific assessments for each listed domain, and thresholds (eg, 80% of the uninjured limb) to be used to determine RTS. Consensus and partial agreement were defined, a priori, as ≥75% and 50% to 75% agreement, respectively. Evidence Synthesis: Eight domains were identified within 11 included studies. Consensus was reached regarding the need to assess sport-specific movement (n = 9, 90.9%). Partial agreement was reached for the need to assess static balance (n = 7, 63.6%). The domains of pain and swelling, patient reported outcomes, range of motion, and strength were also partially agreed on (n = 6, 54.5%). No agreement was reached on specific assessments of cutoff thresholds. Conclusions: Given consensus and partial agreement results, RTS decisions following LAS should be based on sport-specific movement, static balance, patient reported outcomes, range of motion, and strength. Future research needs to determine assessments and cutoff thresholds within these domains to minimize recurrent LAS risk. [ABSTRACT FROM AUTHOR]

Resiliency is a psychological construct that describes an individual's ability to recover from adversity and stress. Resiliency has been studied in the health care setting. In the past decade multiple studies have investigated the relationship between resiliency and patient-reported outcomes following orthopaedic surgery. The aim of this article is to review the most frequently used resiliency measures in orthopaedic surgery research and their implementation in outcome studies. The most commonly utilized resiliency measures in orthopaedic surgery are the Brief Resilience Score (BRS), Connor-Davidson Resilience Scale (CD-RISC), and the Resilience Scale (RS). These resiliency measures are each designed for use with adults and considered to be high quality, reliable, accurate, and consistent measurements of resilience.

The journey of metastatic colorectal cancer patients is complex and challenging, requiring coordination and collaboration between multiple healthcare providers. Understanding patients’ needs, fears, feelings, concerns, and behaviors is essential for providing individualized patient-centered care. In recent years, mCRC patients have experienced improvements in clinical outcomes, from 16 months of overall survival to 32 months, thanks to research. However, there is still room for improvement, and integrating clinical and translational research into routine practice can help patients benefit from treatments and techniques that would not be an option. In the Journey of mCRC patients, living well with cancer and quality of life becomes a priority given the outcomes of the disease. Patient reported outcomes (PRO) and Patient Reported Outcome Measures (PROMs) are becoming therefore new estimands in Oncology. Patient advocates represent important figures in this process by prioritizing issues and research questions; evaluating research designs and the performance of the research; the analysis and interpretation of data; and how results are disseminated. Multidisciplinary Tumor Boards and shared decision-making is essential for designing treatment strategies for individual patients. Quality of Life is often prioritized only when it comes to refractory advanced disease and end-of-life care, but it has to be integrated from the beginning, as the emotional impact of diagnosis leads to a vulnerable situation where patients’ needs and preferences can be easily overseen. First-line treatment will be chosen among more treatment options than subsequent lines, with longer progression-free survival and a bigger impact on the outcomes. Practicing patient-centered care and optimizing first-line treatment for colorectal cancer patients requires a comprehensive understanding of patient experience and treatment outcomes, which can guide clinical practice and inform regulatory decisions for the benefit of patients.

La salud mental es fundamental para el bienestar y para tener una vida plena; de hecho, el bienestar es un aspecto nuclear para definir la salud según la Organización Mundial de la Salud (OMS), en la que el bienestar parece que se presenta como una suerte de estado ideal, de objetivo a alcanzar para que una persona pueda ser considerada sana. No obstante, cabría preguntarse: ¿existe un solo bienestar?, ¿podemos considerar que existe una situación meta, denominada bienestar, que sea universal?, incluso podríamos plantearnos: ¿son universales los aspectos que definen aquello que denominamos calidad de vida? La importancia de la definición de salud de la OMS radica en la inclusión del término bienestar y, consecuentemente, enfatizar la subjetividad. Fue probablemente este mayor protagonismo del bienestar y lo que significa lo que ha motivado la evolución hacia una atención sanitaria centrada en el paciente y en la atención sanitaria basada en valor, donde la medición de resultados en salud reportados por el paciente y de la experiencia del paciente son métricas clave para evaluar la calidad asistencial. Ambas mediciones recogen información sobre la perspectiva de los pacientes y ambas permiten valorar no solo la calidad de vida sino el impacto que la enfermedad y los tratamientos en la vida diaria de nuestros pacientes. Nos permiten “oír la voz de nuestros pacientes” a través de datos clínicos. ABSTRACT Mental health is essential for wellbeing and for having a full life; actually, wellbeing is a nuclear element to define health according to the World Health Organization (WHO), where wellbeing seems to be a kind of ideal state, a sort of objective to achieve so that a person could be considered as healthy. Nevertheless, we could wonder: Is there a unique wellbeing?, could we consider there exist a universal goal situation, called wellbeing? The importance of the WHO definition of health lies on the inclussion of the term wellbeing and, therefore, to emphasize. It was probably this higher protagonism of wellbeing and what it means what has motivated the evolution towards a patient-centered health attention and value-based attention, where the patient-reported outcomes measures and the patient-reported experience measurement are key metrics to assess quality of care. Both measurements gather information about patient views and both of them allow us to asses not only quality of care but also the impact of illness and treatments in our patient’s daily life. They provide us “to hear our patient’s voice” throught clinical data.

Background: Paclitaxel-induced peripheral neuropathy (PN) is a serious clinical problem with no approved drug for prevention. This study aimed to examine the neuroprotective effect of metformin against paclitaxel-induced PN in breast cancer patients. Methods: Patients with confirmed breast cancer diagnosis who were planned to receive paclitaxel were randomized to receive either metformin or placebo. Both groups received the standard chemotherapy protocol for breast cancer. Patients started metformin/placebo 1 week before paclitaxel initiation and continued study interventions thereafter for nine consecutive weeks. The primary outcome was the incidence of development of grade two or more paclitaxel-induced sensory PN. The PN was graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE). Patients' quality of life (QoL)was assessed by the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACTGOG-Ntx) subscale. Pain severity was measured by the Brief Pain Inventory Short Form (BPI-SF). Serum levels of nerve growth factor (NGF) and neurotensin (NT) were measured at baseline and at the end paclitaxel treatment. Results: A total of 73 patients (36 in the metformin arm and 37 in the control arm)were evaluated. The cumulative incidence of development of grade two or more PN was significantly lower in the metformin arm (14 (38.9%) than the control arm (28 (75.7%); p= 0.001). At the end of paclitaxel treatment, patients' QoL was significantly better in the metformin arm [median (IQR) FACTGOG-Ntx subscale of (24.0 (20.5-26.5)] compared to the control arm (21.0 (18.0-24.0); p = 0.003). The metformin arm showed lower "average" and "worst" pain scores than those detected in the control arm. At the end of the paclitaxel treatment, there was a significant difference in the median serum NGF levels between the two arms, favoringmetformin (p < 0.05), whileNT serum levels were deemed comparable between the two study arms (p = 0.09). Conclusion: The use of metformin in breast cancer patients offered a marked protection against paclitaxel-induced PN, which translated to better patient QoL. [ABSTRACT FROM AUTHOR]

Introduction: Obesity is a well‐known risk factor for atrial fibrillation (AF). We aim to evaluate the effect of baseline obesity on procedural complications, AF recurrence, and symptoms following catheter ablation (CA). Methods: All consecutive patients undergoing AF ablation (2013–2021) at our center were enrolled in a prospective registry. The study included all consecutive patients with available data on body mass index (BMI). Primary endpoint was AF recurrence based on electrocardiographic documentation. Patients were categorized into five groups according to their baseline BMI. Patients survey at baseline and at follow‐up were used to calculate AF symptom severity score (AFSS) as well as AF burden (mean of AF duration score and AF frequency score; scale 0: no AF to 10: continuous and 9 frequencies/durations in between). Patients were scheduled for follow‐up visits with 12‐lead electrocardiogram at 3, 6, and 12 months after ablation, and every 6 months thereafter. Results: A total of 5841 patients were included (17% normal weight, 34% overweight, 27% Class I, 13% Class II, and 9% Class III obesity). Major procedural complications were low (1.5%) among all BMI subgroups. At 3 years AF recurrence was the highest in Class III obesity patients (48%) followed by Class II (43%), whereas Class I, normal, and overweight had similar results with lower recurrence (35%). In multivariable analyses, Class III obesity (BMI ≥ 40) was independently associated with increased risk for AF recurrence (hazard ratio, 1.30; confidence interval, 1.06–1.60; p =.01), whereas other groups had similar risk in comparison to normal weight. Baseline AFSS was lowest in normal weight, and highest in Obesity‐III, median (interquartile range) 10 (5–16) versus 15 (10–21). In all groups, CA resulted in a significant improvement in their AFSS with a similar magnitude among the groups. At follow‐up, AF burden was minimal and did not differ significantly between the groups. Conclusion: AF ablation is safe with a low complication rate across all BMI groups. Morbid obesity (BMI ≥ 40) was significantly associated with reduced AF ablation success. However, ablation resulted in improvement in QoL including reduction of the AFSS, and AF burden regardless of BMI. [ABSTRACT FROM AUTHOR]

Purpose: The animated activity questionnaire (AAQ) is a computer-based measure of activity limitations. To answer a question, patients choose the animation of a person performing an activity that matches their own level of limitation. The AAQ has not yet been tested for suitability to be applied as computer-adaptive test (CAT). Thus, the objective of this study was to develop and evaluate an AAQ-based CAT to facilitate the application of the AAQ in daily clinical care. Methods: Patients (n = 1408) with hip/knee osteoarthritis from Brazil, Denmark, France, The Netherlands, Norway, Spain, and the UK responded to all 17 AAQ items. Assumptions of item-response theory (IRT) modelling were investigated. To establish item parameters for the CAT, a graded response model was estimated. To evaluate the performance of post-hoc simulated AAQ-based CATs, precision, test length, and construct validity (correlations with well-established measures of activity limitations) were evaluated. Results: Unidimensionality (CFI = 0.95), measurement invariance (R2-change < 2%), and IRT item fit (S-X2p >.003) of the AAQ were supported. Performing simulated CATs, the mean test length was more than halved (≤ 8 items), while the range of precise measurement (standard error ≤ 0.3) was comparable to the full AAQ. The correlations between original AAQ scores and three AAQ-CAT versions were ≥ 0.95. Correlations of AAQ-CAT scores with patient-reported and performance measures of activity limitations were ≥ 0.60. Conclusion: The almost non-verbal AAQ-CAT is an innovative and efficient tool in patients with hip/knee osteoarthritis from various countries, measuring activity limitations with lower respondent burden, but similar precision and construct validity compared to the full AAQ. [ABSTRACT FROM AUTHOR]

NRG/RTOG 1203 compared 3-D conformal radiotherapy (3D CRT) to intensity-modulated radiotherapy (IMRT) in patients with endometrial or cervical cancer requiring post-operative radiotherapy after hysterectomy. The purpose of this study was to report the first quality-adjusted survival analysis comparing the two treatments. NRG/RTOG 1203 randomized patients having undergone hysterectomy to either 3DCRT or IMRT. Stratification factors included RT dose, chemotherapy, and disease site. The EQ-5D, both index and visual analog scale (VAS), were obtained at baseline, 5 weeks after the start of RT, 4–6 weeks post RT and 1 and 3-years post RT. EQ-5D index and VAS scores along with quality-adjusted survival (QAS) were compared between treatment arms using the t -test at a two-sided significance level of 0.05. NRG/RTOG 1203 enrolled 289 patients of which 236 consented to participate in the patient reported outcome (PRO) assessments. QAS was higher in women treated with IMRT, 1374 vs 1333 days (p = 0.5) compared to patients treated with 3DCRT, but this difference was not statistically different. Patients treated with IMRT had less of a decline in VAS score 5 weeks post RT, −5.04, compared to patients treated with 3DCRT, −7.48, although not statistically significant (p = 0.38). This is the first report of the use of the EQ-5D comparing two radiotherapy techniques in the treatment of gynecologic malignancies after surgery. While there were no significant differences in QAS and VAS scores between patients who received IMRT vs. 3DCRT, RTOG 1203 was not powered to show statistical differences in these secondary endpoints. • The first randomized clinical trial comparing two types of radiotherapy in gynecologic malignancies. • Utilities were measured a priori to calculate quality-adjusted survival between the two treatment arms. • Although not powered for these endpoints, non-statistically significant improvements were seen with the use of IMRT. [ABSTRACT FROM AUTHOR]