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Objective To assess the value of a morphine Patient Controlled Intravenous Analgesia (PCIA) after Tonsillectomies (TE). Methods 30 adult patients were treated with oral analgesics (protocol group) and compared to 30 patients treated with a morphine PCIA for the first 3 Postoperative Days (PODs) after TE. Average and maximum pain severities (Numeric Rating Scale – NRS: 0–10) on PODs 1–3, analgesic score, quality of life, patient satisfaction and side effects were defined as outcome measures. Results Average pain severities of the protocol and the PCIA group were of similar magnitude (NRS) (POD1: 4.48 vs. 4.71 [p = 0.68], POD2: 4.75 vs. 4.22 [p = 0.32] and POD3: 4.44 vs. 4.25 [p = 0.71]). Maximum pain intensities on POD1 (p = 0.92), POD2 (p = 0.51) and POD3 (p = 0.36) were also comparable between both groups. Patients with a PCIA consumed significantly more opioids (p = 0.001) without significant more side-effects. Conclusion The PCIA did not provide a superior pain control compared to oral analgesics. In view of the considerable effort and the high opioid consumption, it cannot be recommended as a standardized application for pain control after TE.

Increased exposure to ultraviolet radiation (UVR) is associated with an increased risk of nonmelanoma skin cancer. Cutaneous surgery can be negatively influenced by UVR, causing delayed wound healing, hyperpigmentation of the scar, and an increased incidence of additional skin cancers. By changing sun protection behavior, these risks can be limited. Therefore, this study evaluates changes in patients' sun protective behavior after Mohs micrographic surgery (MMS). Patients undergoing MMS between December 2017 and November 2019 were included. Patients were asked to complete the FACE-Q Skin Cancer - Sun Protection Behavior checklist before and 3 months and 1 year post-surgery. A total of 125 patients completed the pre-operative and 3-months post-operative checklists, and 89 (71.2%) completed the 1-year post-operative checklist. Reported sun protective behaviors increased post-surgery at all time points (p < 0.001). Patients with a prior history of facial skin cancer demonstrated a larger increase in sun protection behaviors after surgery than patients without a history of facial skin cancer (p = 0.04). Patients with defects located on the ear or scalp demonstrated a lesser increase in sun protection behaviors than patients with defects located in more conspicuous areas as the face (p = 0.02). Our study demonstrates a change in sun protection behavior, with an increase in sun protection behavior over time in patients after MMS. However, more improvement is possible. Targeted counseling can increase sun protection behavior in patients without a history of facial skin cancer and patients with skin cancer located on the ears or scalp.

Objective Nonmelanoma skin cancer (NMSC) is one of the most diagnosed cancers in the world, with the number of new occurrences rising every year. Most patients with facial skin cancer experience cancer-related worry. Yet, little is known about their worry during the period after cancer treatment. This study aimed to assess the long-term change of cancer worry after surgical treatment in patients with NMSC. Methods Patients undergoing surgery for facial NMSC between December 2017 and March 2020 were asked to complete the FACE-Q Skin Cancer-Cancer Worry scale before (baseline), 3-month, and 1-year post-surgery. Results A total of 151 patients completed the baseline and 3-month, and 99 (65.6%) the 1-year post-operative survey. A significant decrease in cancer worry score was seen between baseline and 3-month post-surgery (p < 0.001). No difference was found between the 3-month and 1-year post-surgery scores (p = 0.78). Less improvement in cancer worry was seen for patients who had one facial skin cancer in their medical history (p = 0.001) and patients who had a history of facial surgery (p < 0.001). Conclusion Post-surgery patients still experience cancer worry. Therefore, targeted counseling might be of value when coping with cancer-related concerns. Patients with a history of facial NMSC and patients with a history of facial surgery might benefit from additional counseling.

Background There are three patient reported outcome measure instruments (PROMs) that have adequate content validity for breast reconstruction, BREAST-Q, BRECON-31 and EORTC QLQ-BRECON-23, and they all have been robustly validated. The aim of this study was to systematically review scores giving meaning to validated PROMs for breast reconstruction after mastectomy and discuss methods to enable interpretation of them. Methods A systematic review was performed according to the recommendations of PRISMA. Prospero CRD42021255874. Included articles had to meet criteria defined in a SPIDER (Sample, Phenomenon of Interest, Design, Evaluation, Research type). The included studies were critically appraised using the GRADE approach. Results Three articles were finally included in the review: two studies on scores for healthy controls and one on minimally important differences (MIDs), both of BREAST-Q. All of the studies were performed in North America. Only MIDs based on statistical characteristics, and not on what constitutes a relevant change for the patient, exist. The risk of bias was evaluated as very high and moderate, respectively, of inconsistencies as low, of indirectness as high, of imprecisions as low, and of publication bias as probably low. Conclusions The overall certainty of evidence for scores giving meaning to PROMs for breast reconstruction is low (GRADE ƟƟОО). More studies are needed to establish relevant healthy control scores and what constitutes a relevant clinical difference for patient-reported outcome measures for breast reconstruction after mastectomy. Clinical implications of the findings and suggestions for further research are suggested in the article., Highlights • Patient reported outcome measurements have to be clinically interpretable to make sense. • Three articles on scores giving meaning to PROMs for breast reconstruction post-mastectomy exist. • More studies are needed to establish relevant controls and clinical differences in scores. • Clinical implications and suggestions for further research are discussed.

Purpose: The aim of this study was to evaluate the prevalence of bowel/bladder dysfunction in multiple sclerosis (MS) and its associations with cognitive impairment.Methods: We prospectively enrolled 150 MS patients. Patients were administered the Symbol Digit Modality Test (SDMT), the Neurogenic Bowel Dysfunction Score (NBDS), and the Actionable Bladder Symptom Screening Tool (ABSST). The associations between bowel/bladder dysfunction and cognitive function were assessed through hierarchical regression models using the SDMT and clinicodemographic features as independent variables and NBDS and ABSST scores as dependent variables.Results: The prevalence of bowel/bladder deficits was 44.7%, with 26 patients (17.3%) suffering from bowel deficits and 60 patients (40%) from bladder deficits. The total NBDS and ABSST scores were correlated with the SDMT (β=-0.10, P

Background Orthoplastic operations for lower limb osteomyelitis (LLOM) involving microvascular free tissue reconstructions (“free-flaps”) are usually performed under general anaesthesia (GA), with or without epidural anaesthesia (EA) due to concerns about the discomfort associated with prolonged surgery. However, our clinical experience supports “awake” epidural anaesthesia with sedation (EA + Sed) rather than EA + GA as a technique of choice for this type of surgery. Methods We used a standardised postoperative questionnaire to formally assess the experiences and outcomes for 50 patients who underwent free-flaps for LLOM under EA + Sed. Findings The mean duration of surgery was 522 min (8.7 h), range 240–875 min. There were no ITU admissions or flap failures. Postoperatively, fifty patients completed a standardised questionnaire about their experiences before the operation, in the anaesthetic room and theatre. 80% were aware of the procedure at least “some of the time”. 72.5% patients and 75% respectively, did not have any concerns in the anaesthetic room and theatre. Concerns expressed by the remaining patients were manageable. 97.5% of those patients who recalled their operation reported their overall experience as “comfortable” or “very comfortable”. 92% of respondents had undergone previous lower limb surgery under GA ± EA. In this subgroup, 91.3% reported the recovery after EA + Sed as “quicker” than GA, and 89.4% reported their experience with EA + Sed as “better”. All fifty patients (100%) were “satisfied” or “very satisfied” with their experience and all but one (98%) would recommend this technique to others. Conclusions Our study showed that despite prolonged duration, the patients' reported experiences and outcomes were excellent when EA + Sed was used for orthoplastic operations involving free-flaps for LLOM. We recommend EA + Sed as the anaesthetic technique of choice for such patients., Highlights • Orthoplastic operations for lower limb osteomyelitis (LLOM) involving microvascular free tissue reconstructions (“free-flaps”) are usually performed under general anaesthesia (GA) with or without epidural anaesthesia (EA), due to concerns about the discomfort associated with prolonged surgery. • EA without GA ensures reliable analgesia and provides stable physiology, thus optimising the perfusion of “free flaps”. EA alone avoids vasoconstriction due to inadequate pain relief, hypoxaemia, and surges of blood pressure. • Our study showed that despite prolonged duration, the patients' reported experiences and outcomes were excellent under EA with Sedation.

Background There are no any clear evidence to date has evaluating patients' and caregiver' preferences for r-hGH (recombinant-human growth hormone) injection in children in Japan. This study quantitatively evaluated the factors driving preferences for daily r-hGH injection among Japanese children with Growth Hormone Deficiency (GHD) or their caregivers and to determine the relative importance of treatment delivery attributes. Methods This study was performed among Japanese children with GHD or their caregivers who visited a specialized clinic in Japan as part of their routine care. The participants were asked to complete a web-based discrete choice experiment (DCE) questionnaire. Results Choice-based conjoint analysis was used to evaluate the relative importance of attributes as choice predictors and determine utilities for each attribute. Of the 47 respondents who participated in this study, 41 were caregivers who responded on behalf of the patients, and the remaining 6 were patients who completed the DCE themselves. The injection schedule was found to be the most important attribute for both patients and caregivers; a once-weekly injection schedule was preferred over a daily injection schedule. The storage and preparation attribute was deemed more important to patients than it was to caregivers, with patients preferring store in room temperature even if it needed an additional mixing(reconstitution) step. Both patients and caregivers showed a clear preference for devices that offered a dose setting memory. Conclusions A less frequent injection schedule may enhance adherence to r-hGH treatment, and expected improve quality of life in GHD patients over the long term.

Hyun-Il Gil,1,* Sungmin Zo,2,* Paul W Jones,3,4 Bo-Guen Kim,2 Noeul Kang,2 Yeonseok Choi,5 Hyun Kyu Cho,6 Danbee Kang,7 Juhee Cho,7 Hye Yun Park,2 Sun Hye Shin2 1Division of Pulmonary and Critical Care Medicine, Department of Internal Medicine, Kangbuk Samsung Hospital, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea; 2Division of Pulmonary and Critical Care Medicine, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea; 3Institute For Infection and Immunity, St George’s University of London, London, UK; 4Value Evidence and Outcomes, Global Medical R&D, GlaxoSmithKline, Uxbridge, UK; 5Division of Pulmonary, Allergy and Critical Care Medicine, Department of Internal Medicine, Kyung Hee University School of Medicine, Seoul, Republic of Korea; 6Division of Pulmonary and Critical Care Medicine, Department of Medicine, Samsung Changwon Hospital, Sungkyunkwan University School of Medicine, Changwon, Republic of Korea; 7Samsung Advanced Institute for Health Sciences & Technology (SAIHST), Sungkyunkwan University, Seoul, Republic of Korea*These authors contributed equally to this workCorrespondence: Sun Hye Shin; Hye Yun ParkDivision of Pulmonary and Critical Care Medicine, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of KoreaTel +82-2-3410-3429Fax +82-2-3410-3849Email freshsunhye@gmail.com; hyeyunpark@skku.eduPurpose: The chronic obstructive pulmonary disease (COPD) assessment test (CAT) is widely used to assess the impact of COPD symptoms on health status. Whilst the CAT consists of eight different items, details on the distribution of each item are limited. This study aimed to investigate the distribution and clinical implication of each CAT item, stratified by CAT severity group, in stable COPD patients.Patients and Methods: This was a cross-sectional study at a single referral hospital in South Korea. Spirometry confirmed COPD patients with CAT measured at the first clinical visit were retrospectively identified. Patients were categorized into three groups: low (0 ≤ CAT < 10), medium (10 ≤ CAT < 20), and high (20 ≤ CAT ≤ 40) impact group. For the purpose of this analysis, the first four items (cough, sputum, chest tightness, and dyspnea) and the remaining four items (activities, confidence, sleep and energy) were also grouped as “pulmonary” and “extra-pulmonary”, respectively.Results: A total of 815 patients were included, and mean (SD) forced expiratory volume in 1 s (FEV1) was 62.8 (17.4) % pred. Among them, 300 patients (36.8%) were in the high impact group and had a greater exacerbation history and lower lung function. The proportion of “extra-pulmonary” items score was greater in patients with higher total CAT scores, with the activity and confidence items showing higher scores.Conclusion: In our study, in addition to dyspnea, activity limitation is a particular problem in individual patients with higher CAT total scores, for which physicians need to pay more attention. Our study suggests that whilst CAT total score captures the overall impact of COPD, each item of the CAT contains potentially useful information in understanding the patient’s symptom burden.Keywords: COPD, COPD assessment test, patient reported outcome, symptom

Objectives GCA is a large vessel vasculitis (LVV) presenting with headache, jaw claudication, musculoskeletal and visual involvement. Current treatment is glucocorticoids and anti-IL-6 tocilizumab in refractory disease. The objective of this study was to explore the impact of GCA and its treatment on people’s health-related quality of life (HRQoL), to inform the development of a disease-specific patient-reported outcome measure (PROM) for use in clinical trials and practice. Methods Participants from the UK and Australia, with biopsy- or imaging-confirmed GCA, were interviewed to identify salient aspects of HRQoL in relation to GCA and its treatment. Purposive sampling included a range of demographic and disease features (cranial, LVV-GCA and visual involvement). Inductive analysis identified individual themes of importance, then domains. Candidate questionnaire items were developed from the individual themes, refined by piloting, cognitive interviews and a linguistic translatability assessment. Results Thirty-six interviews were conducted to saturation with participants with GCA from the UK (25) and Australia (11). Mean age was 74 years, 23 (63.9%) were female, 13 (36.1%) had visual loss and 5 (13.9%) had LVV-GCA. Thirty-nine individual themes within five domains were identified: physical symptoms; activity of daily living and function; participation; psychological impact; and impact on sense of self and perception of health. Sixty-nine candidate items were developed from individual themes; piloting and refinement resulted in a 40-item draft questionnaire. Conclusion This international qualitative study underpins the development of candidate items for a disease-specific PROM for GCA. The draft questionnaire is now ready for psychometric testing.

Background This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective. Methods A 54‐item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA. Results Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients. Conclusions Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.

Backgroud The purpose of this study was to examine the between-mode equivalence and the relative efficiency of the 2 available modes of patient-reported outcome (PRO) data collection: a web-enabled touch screen tablet and a smartphone in a sample of patients who underwent foot and ankle orthopedic surgery. Methods A total of 136 patients who visited the clinic after foot/ankle surgery participated in the study. All patients completed the PRO questionnaire set using tablets at the hospital. After 24 hours of completing the first PRO questionnaire, the patients completed the same PRO questionnaire at home using their personal smartphones. The outcomes were statistically compared, and the patients' preferences were surveyed. Results The intraclass correlation coefficients for comparing the results of PRO measurements between the 2 modes were 0.970 for the visual analog scale, 0.952 for the Foot Function Index, 0.959 for the foot and ankle outcome scale, and 0.957 for the patient's satisfaction. Sixty-eight participants (58.6%) responded that they were able to answer the questionnaires with more honesty at home using their smartphones. Regarding the mode, 60 participants (48.1%) responded that they have no preference between the devices. Conclusions The results of this study showed the equivalence of the 2 modes of PRO data collection: web-enabled touch screen tablets and smartphones. Smartphones may be the preferred mode of PRO measurement, due to their easy accessibility, increased privacy, and the patients' increased honesty in answering questionnaires.

Technical improvements in head and neck cancer radiotherapy over the last decade have resulted in substantial reductions in dose to organs-at-risk. For a mix of tumors, we saw less xerostomia moving from 3D-conformal to more advanced techniques. For oropharynx-only there were additional improvements, including in global quality-of-life and sticky saliva.

Background A few previous studies have investigated patient satisfaction after total hip arthroplasty (THA) according to the degree of pelvic deformity. This study compared patient-reported outcomes after primary THA for Crowe types III, IV and I dysplasia. Methods This retrospective, single-center, single-surgeon case-control study included patients who underwent primary THA between 2008 and 2016. We sent postal questionnaires to 38 patients with Crowe type III and IV dysplasia. Among the questionnaire respondents, 23 patients, excluding those with a follow-up period of Results The H and control groups were not significantly different in the postoperative JOA scores and SF-36. In the H group, VAS at the final follow-up was significantly higher, and significantly more patients felt that postoperative rehabilitation was serious, expressing that they underwent THA for LLD correction. In addition, the VAS scores in the FO group was higher than those in the N-FO group. Postoperative LLD was significantly greater in the H group than in the control group. Each group had an NPS of > 50. Conclusion The postoperative VAS score was higher in Crowe type III and IV dysplasia than in Crowe type I dysplasia, but no significant differences were detected in the postoperative satisfaction, JOA score, and SF-36 score. These findings may help explain the effects of THA preoperatively to patients with Crowe type III and IV dysplasia. Level of evidence Therapeutic Level 3b.

Background An arthroscopic Bankart operation is the most common operative procedure to treat shoulder instability. In case of recurrence, both Bankart and Latarjet procedures are used as revision procedures. The purpose of this study was to compare the re-recurrence rate of instability and clinical results after arthroscopic revision Bankart and open revision Latarjet procedures following failed primary arthroscopic Bankart operations. Methods Consecutive patients operatively treated for shoulder instability at Turku University Hospital between 2002 and 2013 were analyzed. Patients who underwent a primary arthroscopic Bankart operation followed by a recurrence of instability and underwent a subsequent arthroscopic Bankart or open Latarjet revision operation with a minimum of 1 year of follow-up were called in for a follow-up evaluation. The re-recurrence of instability, Subjective Shoulder Value, and Western Ontario Shoulder Instability index were assessed. Results Of 69 patients, 48 (dropout rate, 30%) were available for follow-up. Recurrent instability symptoms occurred in 13 patients (43%) after the revision Bankart procedure and none after the revision Latarjet procedure. A statistically and clinically significant difference in the Western Ontario Shoulder Instability index was found between the patients after the revision Bankart and revision Latarjet operations (68% and 88%, respectively; P = .0166). Conclusions The redislocation rate after an arthroscopic revision Bankart operation is high. Furthermore, patient-reported outcomes remain poor after a revision Bankart procedure compared with a revision Latarjet operation. We propose that in cases of recurring instability after a failed primary Bankart operation, an open Latarjet revision should be considered.

Background and purpose — Patient-reported outcomes (PROMs) after primary total hip arthroplasty (THA) and revision THA are important information in the preoperative shared decision-making process. We present 1-year results on pain, function, and quality of life following primary and revision THA. Patients and methods — From 2010 to 2018, 3,559 primary THA and 406 revision THAs were included in our institutional quality registry. PROMs were registered preoperatively, 3 months, and 1 year after surgery, numeric rating scale (0–10) for pain during mobilization and at rest, healthrelated quality of life (EQ-5D), and a hip-specific physical function score (HOOS-PS). 2 anchor questions were asked 1 year after surgery concerning joint function and willingness to go through surgery again. Results — There were statistically significant improvements in all PROMs at the 3-month follow-up in both groups. All PROMs improved more in the primary group relative to the revision group. 1 year after surgery, pain during mobilization was reduced with a mean change of 5.1 (SD 2.6) for primary THA and 2.9 (SD 3.0) for revision THA. 93% of primary THA patients reported both better function 1 year after surgery and that they would have gone through surgery again, compared with 78% and 79% in the revision THA group. Interpretation — Primary THA patients reported better function and more pain relief than the revision THA group 1 year after surgery. Pain during mobilization shows the most marked improvement in both groups, which is important preoperative information for patients.

Background Kidney failure with replacement therapy and hemodialysis are associated with a decrease in quality of life (QOL). Self-reported QOL symptoms are not always prioritized by the medical team, potentially leading to conflicting priorities with patients. Electronic patient-reported outcome measures (ePROMs) allow physicians to better identify these symptoms. The objective was to describe the prevalence of symptoms self-reported by hemodialysis (HD) patients. Methods A multicenter cross-sectional study was conducted in three HD centers. Patients were included if they were 18 years old or over treated with HD for at least 3 months in a center. Data were collected by the patient via a self-administered ePROMs questionnaire. Data included patient characteristics, post-dialysis fatigue and intensity, recovery time after a session, perceived stress, impaired sleep the day before the dialysis session, current state of health and the change from the past year. A multivariate analysis was conducted to identify relations between symptoms. Results In total, we included 173 patients with a mean age of 66.2 years, a mean ± SD hemodialysis duration of 48.9 ± 58.02 months. The prevalence of fatigue was 72%. 66% had a high level of stress (level B or C). Recovery time was more than 6 h after a HD session for 25% of patients and 78% declared they had a better or unchanged health status than the previous year. Sleep disturbance was associated with cardiovascular comorbidities (OR 5.08 [95% CI, 1.56 to 16.59], p = 0.007). Conclusions Fatigue and stress were the main symptoms reported by HD patients. The patient’s care teams should better consider these symptoms.

Pain is the most common symptom in chronic pancreatitis (CP) with a major impact on quality of life. Few validated questionnaires to assess pain in CP exist, and the lack of consensus negatively impacts clinical management, research and meta-analysis. This guideline aims to review generic pain questionnaires for their usability in CP, to outline how pain assessment can be modified by confounding factors and pain types, to assess the value of additional measures such as quality of life, mental health and quantitative sensory testing, and finally to review pain assessment questionnaires used specifically in CP. A systematic review was done to answer 27 questions that followed the PICO (Population; Intervention; Comparator; Outcome) template. Quality of evidence of the statements was judged by Grades of Recommendation, Assessment, Development and Evaluation (GRADE) criteria. The manuscript was sent for review to 36 experts from various disciplines and continents in a multi-stage Delphi process, and finally reviewed by patient representatives. Main findings were that generic pain instruments are valid in most settings, but aspects of pain are specific for CP (including in children), and instruments have to account for the wide phenotypic variability and development of sensitization of the central nervous system. Side effects to treatment and placebo effects shall also be considered. Some multidimensional questionnaires are validated for CP and are recommended together with assessment of quality of life and psychiatric co-morbidities. This guideline will result in more homogeneous and comprehensive pain assessment to potentially improve management of painful CP. (c) 2021 The Authors. Published by Elsevier B.V. on behalf of IAP and EPC. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

Background Intense and aggressive treatment regimens for most children’s cancer have achieved vast improvements in survival but are also responsible for both a high number and burden of symptoms. The use of Patient Reported Outcome Measures (PROMs) demonstrates a range of benefits for improved symptom management in adults with cancer. There are, however, multiple barriers to integrating PROMs into routine care in children and adolescents with cancer. This study aims to evaluate: (1) the effectiveness of electronic PROMs to generate stratified alerts, symptom management recommendations and graphical summaries (the RESPONSE system) to improve health outcomes and (2) the implementation of the RESPONSE system by assessing feasibility, acceptability, satisfaction, and sustainability. Methods A pragmatic hybrid II effectiveness-implementation controlled trial, using mixed methods, will be undertaken, advancing both knowledge of the effectiveness of the intervention and implementation factors. One-hundred and sixty children with cancer receiving active treatment will be recruited 1:1 to a non-randomised study involving two groups with an equal number of participants in each group. The intervention group (n = 80) will be prospectively recruited to receive the RESPONSE system intervention over eight weeks, versus the historical matched control group (n = 80) who will complete the ePROMs without access to the RESPONSE system. The primary outcome of the effectiveness trial is change between groups in total symptom burden. Secondary outcomes include child health-related quality-of-life and implementation outcomes. Trial data will be analysed using linear mixed-effects models. Formative implementation evaluation is informed by CFIR and ERIC frameworks and implementation outcomes will be mapped to the RE-AIM framework and include interviews, field notes, as well as administrative data to evaluate feasibility, acceptability, satisfaction and sustainability. Trial registration number ACTRN12621001084875. Retrospectively Registered 16 August 2021.

BackgroundRehabilitation following anterior cruciate ligament (ACL) reconstructions is based mainly on comprehensive progressive exercise programmes using a multi-dimensional approach. Elastic knee sleeves may be useful adjuncts to rehabilitation. The aim of this study was to determine the immediate and 6-week effects of wearing a knee sleeve on person-reported outcomes and function in participants who had undergone an ACL reconstruction and who had residual self-reported functional limitations.MethodsIndividuals with ACL reconstruction in the previous 6 months to 5 years were recruited. Immediate effects of a commercially-available elastic knee sleeve on single-leg horizontal hop distance were explored using a cross-over design. Following this first session, participants were randomised into a Control Group and a Sleeve Group who wore the sleeve for 6 weeks, at least 1 h daily. Outcome measures for the randomised clinical trial (RCT) were the International Knee Documentation Classification Subjective Knee Form (IKDC-SKF) score, the single-leg horizontal hop distance, and isokinetic quadriceps and hamstring peak torque. Linear mixed models were used to determine random effects. Where both limbs were measured at multiple time points, a random measurement occasion effect nested within participant was used.ResultsThirty-four individuals (16 women) with ACL reconstruction completed the cross-over trial. Hop distance for the injured side during the sleeve condition increased by 3.6 % (95 % CI 0.4–6.8 %,p = 0.025). There was no evidence of differential changes between groups for the IKDC-SKF (Sleeve Groupn = 15; Control Groupn = 16;p = 0.327), or relative improvement in the injured side compared to the uninjured side for the physical performance measures (Sleeve Groupn = 12, Control Groupn = 12; three-way interactionp = 0.533 [hop distance], 0.381 [quadriceps isokinetic peak torque], and 0.592 [hamstring isokinetic peak torque]).ConclusionsSingle-leg hop distance of the ACL reconstructed side improved when wearing a knee sleeve. Wearing the knee sleeve over 6 weeks did not lead to enhanced improvements in self-reported knee function, hop distance and thigh muscle strength compared to the control group.Trial registrationThe trial was prospectively registered with the Australia New Zealand Clinical Trials Registry No:ACTRN12618001083280, 28 June 2018.

Aims The aim of this study was to identify factors associated with poor outcome following coccygectomy on patients with chronic coccydynia and instability of the coccyx. Methods From the Danish National Spine Registry, DaneSpine, 134 consecutive patients were identified from a single centre who had coccygectomy from 2011 to 2019. Patient demographic data and patient-reported outcomes, including pain measured on a visual analogue scale (VAS), Oswestry Disability Index (ODI), EuroQol five-dimension five-level questionnaire, and 36-Item Short-Form Health Survey questionnaire (SF-36) were obtained at baseline and at one-year follow-up. Patient satisfaction was obtained at follow-up. Regression analysis, including age, sex, smoking status, BMI, duration of symptoms, work status, welfare payment, preoperative VAS, ODI, and SF-36 was performed to identify factors associated with dissatisfaction with results at one-year follow-up. Results A minimum of one year follow-up was available in 112 patients (84%). Mean age was 41.9 years (15 to 78) and 97 of the patients were female (87%). Regression showed no statistically significant association between the investigated prognostic factors and a poor outcome following coccygectomy. The satisfied group showed a statistically significant improvement in patient-reported outcomes at one-year follow-up from baseline, whereas the dissatisfied group did not show a significant improvement. Conclusion We did not identify factors associated with poor outcome following coccygectomy. This suggests that neither of the included parameters should be considered contraindications for coccygectomy in patients with chronic coccydynia and instability of the coccyx. Cite this article: Bone Jt Open 2021;2(7):540–544.

Introduction: Spleen Deficiency Syndrome (SDS) is recognized as one of the most common Traditional Chinse Medicine (TCM) syndromes in patients with colorectal cancer (CRC). However so far there is no existing patient-reported outcome (PRO) to evaluate SDS. Our study aimed to develop and validate a PRO TCM-SDS scale for CRC in China. Methods: We developed an initial 8-item TCM-SDS scale for CRC based on literature review and consultation with experts. We then pilot tested the scale ( n = 40) and performed item revision. We conducted a survey study among CRC patients from oncology clinics at a TCM Hospital to further determine the reliability and validity of the scale. Results: Among 100 patients finally enrolled and analyzed in the survey study, 46% were female with median age of 60 years old, 77% had left side tumors and 23% had stage IV disease. Factor loading indicated that there were three domains within TCM-SDS scale. The final TCM-SDS scale involves 5 items including “I feel loss of appetite,” “I feel abdomen fullness,” “I feel my arms and legs lack strength,” “I feel short of breath when talking,” and “My stool is formless” (Cronbach’s alpha coefficient 0.76). We calculated the total score of the scale by summing the 5 individual items and normalizing them to a value maximum of 10, with higher scores indicating greater burden of spleen deficiency syndrome. The average spleen deficiency score for all patients was 3.55 ± 1.54. Among them, those who had stage IV disease had higher scores than stage I-III patients (4.30 vs 3.38, P = .015). Test-retest reliability after 2 weeks showed Pearson coefficient of 0.67 and all items were highly related ( P Conclusion: The patient-reported TCM-SDS scale for CRC showed adequate initial reliability and validity. The development of the scale provided an outcome measurement tool, which could facilitate future studies to better evaluate the role of TCM in treating CRC.

Healing after dermal injury is a complex but imperfect process that results in a wide range of visible scars. The degree of disfigurement is not the sole determinant of a scar's effect on patient well-being, with a number of other factors being critical to outcome. These include cosmetic appearance, symptoms such as itch and pain, functional loss, psychological or social problems, and quality of life. An accurate assessment of these domains can help clinicians measure outcomes, develop, and evaluate treatment strategies. A PubMed literature search was performed up to 31st March 2020. Ten objective scar measurements, four Clinician-Reported Outcome Measures (CROMs), six Patient-Reported Outcome Measures (PROMs), and one combined measure were evaluated for their reliability, clinical relevance, responsiveness to clinical change, and feasibility. Many quantitative tools were limited in their clinical relevance and feasibility, whereas few qualitative CROMs and PROMs have undergone rigorous assessment. This review examines currently available assessment tools, focusing primarily on subjective scar measurements (CROMs, PROMs), and offers a perspective on future directions in the field.

Summary Background Surgery is performed in many individuals with disorders/differences of sex development (DSD). Irreversibility of some surgical procedures, lack of information about the procedures, and lack of follow-up care for physical and psychological outcomes, lead to wish for more knowledge from both surgeons and patients. After the consensus conference in 2006, multidisciplinary care is provided to a higher degree with psychological support and more restricted surgical procedures. Outcome studies after genital surgery often lack of patient's perspective. Objective To describe surgical procedures in relation to diagnosis, to evaluate the outcomes of surgery through genital examination, and through patient's and observer's satisfaction with the anatomical and functional result after genital surgery. Study design In a cross-sectional clinical study performed in six European countries in 2014/15, we have included 500 participants where surgery was performed, from a total of 1040 adolescents (≥16years) and adults with a DSD. Diagnoses included Turner syndrome (n = 301), mixed gonadal dysgenesis (45,XO/46,XY; n = 45), Klinefelter syndrome (n = 218), XYY (n = 1), 46, XY DSD (n = 222) and 46, XX DSD (n = 253). Study protocol included clinical report files, an optional gynecological or urological examination, patient reported outcomes including received surgical interventions, satisfaction with appearance and function after surgery, and impact of the surgical procedure on life. Results Five hundred participants had received genital or breast surgery, with the highest rate in 46, XY DSD and the lowest in Turner syndrome. Altogether; 240 participants had feminizing surgery, 112 had masculinizing surgery, and 217 underwent gonadectomy. Physicians evaluated anatomical appearance at genital examination as poor in less than 10%. Dissatisfaction with anatomical appearance was reported by 22% of the participants, dissatisfaction with function by 20%. Being (very) dissatisfied with anatomical appearance and function was reported by 13% of the study participants. Most participants reported no impact, or positive impact, of the surgical procedures on their lives, but 29% experienced a negative effect of gonadectomy on their life. Discussion There might be a selection bias and/or a recall bias for participating in our studies. Due to poor data quality about surgical procedures performed in the past, we also relied on participants memory about surgical procedures in their past. Ideally, patient reported outcomes should be evaluated both before and after surgical procedures. Conclusion A vast majority are satisfied with appearance and function, but still genital or breast surgery have a long-lasting effect on patient's life. Self-reported satisfaction is usually lower than the observer's evaluation regarding both appearance and function. Download : Download high-res image (217KB) Download : Download full-size image Summary Figure . Satisfaction with anatomy and function after genital surgery in individuals with DSD (appearance: n = 244; function: n = 257) (n = total number of participants).

Objective: The Constant-Murley score (CMS) is widely used to evaluate disabilities associated with shoulder injuries, but it has been criticized for relying on imprecise terminology and a lack of standardized methodology. A modified guideline, therefore, was published in 2008 with several recommendations. This new version has not yet been translated or culturally adapted for Turkish-speaking populations. The purpose of this study was to translate and cross-culturally adapt the modified CMS and its test protocol, as well as define and measure its reliability and validity. Methods: The modified CMS was translated into Turkish, consistent with published methodological guidelines. The measurement properties of the Turkish version of the modified CMS were tested in 30 patients (12 males, 18 females; mean age: 59.5±13.5 years) with a variety of shoulder pathologies. Intraclass correlation coefficients (ICC) were used to estimate test-retest reliability. Construct validity was analyzed with the Turkish version of the American Shoulder and Elbow Surgeons (ASES) Standardized Shoulder Assessment Form and Short-Form Health Survey (SF-12). Results: No difficulties were found in the translation process. The Turkish version of the modified CMS showed excellent test-retest reliability (ICC=0.86). The correlation coefficients between the Turkish version of the modified CMS and the ASES, SF-12-physical component score, and SF-12 mental component scores were found to be 0.48, 0.35, and 0.05, respectively. No floor or ceiling effects were found. Conclusion: The translation and cultural adaptation of the modified CMS and its standardized test protocol into Turkish were successful. The Turkish version of the modified CMS has sufficient reliability and validity to measure a variety of shoulder disorders for Turkish-speaking individuals.

Background Hyperhidrosis is estimated to affect ~ 4.8% of the US population, and most patients experience a negative psychological impact. Here, we describe development and psychometric evaluation of a patient-reported outcome (PRO) measure to assess severity of axillary hyperhidrosis in clinical trials that meets current U.S. regulatory standards to support product approvals. Methods Three rounds of hybrid concept-elicitation/cognitive-debriefing qualitative interviews were conducted in adults with clinician-diagnosed primary axillary hyperhidrosis, followed by similar interviews in children/adolescents. The draft measure included diary items for presence, severity, impact and bothersomeness (basis of the Axillary Sweating Daily Diary [ASDD]), exploratory weekly impact items, and a single-item Patient Global Impression of Change (PGIC). Phase 2 (adults only) and phase 3 (adults and children ≥9 years) clinical trial data were utilized to evaluate measurement properties of the resulting draft measure: floor/ceiling effects, nonresponse bias, test-retest reliability, construct validity, and responsiveness were assessed. The primary concept of interest was axillary sweating severity (ASDD Item 2); however, additional supportive concepts were explored to allow for development of a comprehensive hyperhidrosis measure. Results Twenty-nine patient interviews were conducted (N = 21 adult and N = 8 children/adolescents), resulting in the ASDD (4 items, patients ≥16y) and child-specific ASDD-C (2 items ≥9y to

Background Haemorrhoidal disease (HD) is a common colorectal condition that often requires surgical treatment. Less invasive procedures are usually more acceptable to patients. The aim of this study was to report the outcome of a novel and minimally invasive technique employing a radiofrequency ablation (RFA) energy (Rafaelo®) to treat HD. Methods A total number of 27 patients who had RFA for the treatment of HD were recruited to this study. The procedure was performed under deep sedation and local anaesthesia. Patients’ demographics; haemorrhoid severity score (HSS); quality of life; pain and satisfaction scores; and recurrence rate were recorded. Results The mean age of the patients was 46 (SD 14) years, 18 (67%) males and 9 (33%) females. The mean body mass index was 25 (SD 4) kg/m2. The predominant symptom of all patients was per-rectal bleeding. HSS improved from 7.2 (SD 1.9) before the procedure to 1.6 (SD 1) after the procedure (p

Contains fulltext : 205187.pdf (Publisher’s version ) (Closed access) BACKGROUND & AIMS: Home parenteral nutrition-quality of life (HPN-QOL((c))) is a self-assessment tool for the measurement of QOL in patients on HPN. The aims of this study were: to re-assess the basic psychometric properties of the HPN-QOL((c)) in a multinational sample of adult patients; to provide a description of QOL dimensions by short and long HPN treatment duration; to explore clinical factors potentially associated to QOL scores. METHODS: Patients (n = 699) from 14 countries completed the HPN-QOL((c)). The questionnaires were analysed to evaluate data completeness, convergent/discriminant validity and internal-consistency reliability. The association of overall QOL and HPN treatment duration as well as other clinical factors were investigated using multivariable linear regression models. RESULTS: The analysis of the multitrait-scaling and internal consistency indicates a good fit with the questionnaire structure for most items. Item discriminant validity correlation was satisfactory and psychometric evaluation of the HPN-QOL((c)) in the different English, French and Italian language patient sub-groups confirmed psychometric equivalence of the three questionnaire versions. The results of the multivariable linear regression showed that QOL scores were significantly associated with HPN duration (better in long-term), underlying disease (better in Crohn's disease and mesenteric ischaemia) and living status (worse in living alone) and, after adjusting for the other factors, with the number of days of HPN infusion per week. CONCLUSIONS: The HPN-QOL((c)), is a valid tool for measurement of QOL in patients on HPN, to be used in the clinical practice as well as in research.

Introduction The use of patient reported outcome measures (PROMs) has increased during the past decade, and the focus on how to use them has resulted in a more proactive application. Studies have shown that proactive use of PROMs during treatment improves patient-clinician communication, leads to better symptom management and may prolong survival among advanced cancer patients. Ovarian cancer is a serious disease in which the majority of patients experience recurrence during the follow-up period and suffer from a number of severe symptoms from underlying disease. This systematic review was conducted to assess the evidence on the proactive use of PROMs as a dialogue tool during follow-up of ovarian cancer patients. Results The following databases were searched for relevant literature; PubMed, EMBASE, CINAHL, and the Cochrane Library. The search was conducted in April 2019 without any filters or limits. A total of 643 publications were identified, and 48 studies were found to be potentially eligible. Of the 48 papers, none met the final inclusion criterion of using PROMs proactively as a dialogue tool for ovarian cancer patients during follow-up. Conclusion Studies have shown that PROMs can identify otherwise undetected symptoms. Using PROMs proactively during the consultation has been shown to improve symptom management for patients with some other types of cancer. However, we found no studies that had examined the proactive use of PROMs during follow-up of ovarian cancer patients. Future studies should evaluate if the proactive use of PROMs could facilitate a more individualized and more effective follow-up program tailored to the ovarian cancer patient’s needs and preferences. Electronic supplementary material The online version of this article (10.1186/s13048-019-0538-9) contains supplementary material, which is available to authorized users.

Objective To assess psychometric properties and cross-language measurement equivalence of six versions of the Bristol Rheumatoid Arthritis Fatigue Scale (BRAF-MDQ) and the Rheumatoid Arthritis Impact of Disease Score (RAID in rheumatoid arthritis (RA). Methods Both questionnaires were completed by French (n = 206), German (n = 206), Dutch (n = 317), Spanish (n = 157), Swedish (n = 170) and UK (n = 210) RA patients. The presence of cross-language differential item functioning (DIF) was examined using the generalized partial credit model. The impact of DIF on the item and total scores was examined by comparing DIF unadjusted and DIF adjusted expected item and scale scores. IRT-based methods were used to assess psychometric properties of the instruments. Results 11 of the 20 BRAF-MDQ (55%) and 4 of the 7 RAID items (57%) exhibited significant DIF in at least one of the six countries. The mean number of items with DIF per country was 2.6 for BRAF-MDQ and 1.1 for RAID. However, the impact of DIF on the total RAID and BRAF-MDQ scores, as well as the BRAF subscales, was found to be negligible at the group level. Only for the BRAF physical subscale was there evidence of minor DIF. Marginal reliabilities of BRAF-MDQ (0.93) and RAID (0.89) were excellent, and precise scores could be obtained across the spectrum of disease impact and fatigue scores measured by these PROMs. Conclusion This study supports the cross-language measurement equivalence of BRAF-MDQ and RAID and provides further support for the psychometric properties of these measures in RA. Electronic supplementary material The online version of this article (10.1007/s11136-019-02188-8) contains supplementary material, which is available to authorized users.

Introduction The objective was to identify the most commonly used patient-reported outcome (PRO) instruments for overactive bladder (OAB), determine which are the most useful for measuring burden in OAB and characterize the findings of recent studies that have employed PRO instruments to assess OAB symptoms and the effects of treatment. Methods A systematic search of OAB literature published between January 2006 and November 2017 using Medline/PubMed and EMBASE databases. Results Of 3425 abstracts and 500 full-text articles reviewed, 58 studies (both clinical trials and observational studies) were included in the review. The most commonly used PRO instruments were the OAB Questionnaire (OAB-q; 64%), followed by the King’s Health Questionnaire (KHQ; 31%) and the Patient Perception of Bladder Condition (PCBC; 21%). Synthesis of data from studies using the OAB-q showed that OAB treatment with antimuscarinics, mirabegron and onabotulinumtoxinA all improve health-related quality of life (HRQoL) and symptoms beyond the benefits observed with placebo. The OAB-q could detect dose–response relationships in some studies and demonstrated there were no significant differences across therapies from different drug classes. Conclusion The HRQoL burden of OAB and response to treatment can be reliably measured by PRO instruments, and the OAB-q is the most commonly used instrument in OAB, particularly in clinical trials of OAB interventions. These data will be useful to provide benchmarks of burden levels for PRO scores obtained among those on contemporary therapies for comparison with outcomes from patients managed with emerging treatments. Funding Astellas Pharma Global Development, Inc. Electronic supplementary material The online version of this article (10.1007/s12325-019-0880-8) contains supplementary material, which is available to authorized users.

Background: Fractures of the calcaneus are severe injuries of the hindfoot, mostly resulting from high-energy axial loads, which still present enormous challenges to modern trauma surgery. Possible variables influencing the outcome are the type of fracture, age, and quality of fracture reduction. These might also be factors affecting the self-reported patient outcome, but large studies are still lacking. Therefore, the aim of this study was to analyze the patient-reported outcome of calcaneal fractures following operative and conservative treatment.Methods: All patients suffering from calcaneal fractures between 2002 and 2015 were enrolled in this retrospective analysis. The calcaneal fractures were classified according to Sanders and the AO classification system. For further analysis, two groups were formed: group I involved complex intra-articular fractures defined by the involvement of the posterior calcaneal facet, while group II consisted of extra-articular and process calcaneal fractures. Data were collected via the patient registry, radiographs, and a standardized questionnaire (Foot and Ankle Outcome Score, FAOS). For outcome analysis, non-parametric Mann–Whitney U-test was performed, and Spearman's rank correlation coefficient was calculated.Results: In total, the functional outcome of 79 patients with calcaneal fractures was analyzed. In group 1 (n = 43), the mean FAOS score was 65.5 ± 18.9. The surgically treated patients with a Sanders type II calcaneal fracture had a mean FAOS score of 72.9 ± 17.2, type III fractures had 65.6 ± 20.8, and type IV had 61.1 ± 19 (p = 0.15). The reoperation rate was 22%, most frequently caused by wound complications (10%). The mean follow-up time was 64.5 ± 44 months. The mean FAOS score of group 2 (n = 36) was 75.2 ± 18.4, and 83% of the patients (=30) were managed conservatively. Only one out of six operatively managed patients had a reoperation due to regular implant removal. The mean follow-up time was 31 ± 25.9 months.Conclusion: Intra-articular calcaneal fractures are severe injuries of the hindfoot leading to a fair to poor functional outcome in the majority of the patients. Complications regarding wound healing are the most common causes for revisional surgery. Extra-articular calcaneal fractures are a heterogenous entity commonly managed non-operatively. Overall, they show a better functional outcome in comparison to intra-articular calcaneal fractures.

Background/purpose No studies have comprehensively assessed short-term patient-reported outcomes after the provision of overdentures supported by two immediate implants. The purpose of this study was to evaluate short-term patient-reported outcomes for mandibular overdentures retained by ball attachments on two immediately loaded implants. Materials and methods Nineteen participants with mandibular edentulism were provided with overdentures retained by ball attachments on two immediately loaded, unsplinted implants. The participants' self-assessment of their dentures and oral health-related quality of life were evaluated with the 22-item Patient's Denture Assessment (PDA), and the 19-item Japanese version of the Oral Health Impact Profile (OHIP-EDENT), respectively. Patient satisfaction was measured on a 100 mm visual analogue scale. Assessments were conducted at baseline, and at 1 and 6 months after implant surgery. Results There were significant increases in the PDA “Lower denture” (P = 0.009) at 1 month, as well as “Function” (P = 0.002) and “Lower denture” (P = 0.009) scores at 6 months. Patient satisfaction was also significantly increased at 1 month (P = 0.007) and 6 months (P ≤ 0.000). Significant decreases were observed in the OHIP-EDENT “Physical pain” (P = 0.046) score at 1 month, as well as the summary score (P = 0.033), “Functional limitation” (P = 0.020) and “Psychological discomfort” (P = 0.019) scores at 6 months. Conclusion The use of two immediately loaded implants for lower mandibular complete overdentures is associated with improvements in patient's self-assessment of dentures, satisfaction, and oral health-related quality of life, up to 6 months after implant surgery.

Aims High-grade dysplastic spondylolisthesis is a disabling disorder for which many different operative techniques have been described. The aim of this study is to evaluate Scoliosis Research Society 22-item (SRS-22r) scores, global balance, and regional spino-pelvic alignment from two to 25 years after surgery for high-grade dysplastic spondylolisthesis using an all-posterior partial reduction, transfixation technique. Methods SRS-22r and full-spine lateral radiographs were collected for the 28 young patients (age 13.4 years (SD 2.6) who underwent surgery for high-grade dysplastic spondylolisthesis in our centre (Scottish National Spinal Deformity Service) between 1995 and 2018. The mean follow-up was nine years (2 to 25), and one patient was lost to follow-up. The standard surgical technique was an all-posterior, partial reduction, and S1 to L5 transfixation screw technique without direct decompression. Parameters for segmental (slip percentage, Dubousset’s lumbosacral angle) and regional alignment (pelvic tilt, sacral slope, L5 incidence, lumbar lordosis, and thoracic kyphosis) and global balance (T1 spino-pelvic inclination) were measured. SRS-22r scores were compared between patients with a balanced and unbalanced pelvis at final follow-up. Results SRS-22r domain and total scores improved significantly from preoperative to final follow-up, except for the mental health domain that remained the same. Slip percentage improved from 75% (SD 15) to 48% (SD 19) and lumbosacral angle from 70° (SD 11) to 101° (SD 11). Preoperatively, 35% had global imbalance, and at follow-up all were balanced. Preoperatively, 63% had an unbalanced pelvis, and at final follow-up this was 32%. SRS-22r scores were not different in patients with a balanced or unbalanced pelvis. However, postoperative pelvic imbalance as measured by L5 incidence was associated with lower SRS-22r self-image and total scores (p = 0.029). Conclusion In young patients with HGDS, partial reduction and transfixation improves local lumbosacral alignment, restores pelvic, and global balance and provides satisfactory long-term clinical outcomes. Higher SRS-22r self-image and total scores were observed in the patients that had a balanced pelvis (L5I < 60°) at two to 25 years follow-up. Cite this article: Bone Jt Open 2021;2(3):163–173.

Background The main genetic causes of homocystinuria are cystathionine beta-synthase (CBS) deficiency and the remethylation defects. Many patients present in childhood but milder forms may present later in life. Some countries have newborn screening programs for the homocystinurias but these do not detect all patients. Results HCU Network Australia is one of the very few support groups for patients with homocystinurias. Here we report the results of its survey of 143 patients and caregivers from 22 countries, evaluating current diagnostic pathways and management for the homocystinurias. Most (110) of the responses related to patients with CBS deficiency. The diagnosis was made by newborn screening in 20% of patients and in 50% of the others within 1 year of the initial symptom but in 12.5% it took over 15 years. The delay was attributed mainly to ignorance of the disease. Physicians need to learn to measure homocysteine concentrations in children with neurodevelopmental problems, and in patients with heterogeneous symptoms such as thromboembolism, dislocation of the optic lens, haemolytic uraemic syndrome, and psychiatric disease. Even when the diagnosis is made, the way it is communicated is sometimes poor. Early-onset CBS deficiency usually requires a low-protein diet with amino acid supplements. More than a third of the participants reported problems with the availability or cost of treatment. Only half of the patients always took their amino acid mixture. In contrast, good adherence to the protein restriction was reported in 98% but 80% said it was hard, time-consuming and caused unhappiness. Conclusions There is often a long delay in diagnosing the homocystinurias unless this is achieved by newborn screening; this survey also highlights problems with the availability and cost of treatment and the palatability of protein substitutes.

Purpose of Review The goal of this paper is to review current and future uses of patient-reported outcomes in large vessel vasculitis. The large vessel vasculitides comprise Giant Cell Arteritis and Takayasu arteritis; both are types of systemic vasculitis which affect the larger blood vessels. Patient-reported outcomes (PROs) capture the impact of these diseases on health-related quality of life. Recent Findings Generic PROs such as the SF-36 are currently used to compare HRQOL of people with GCA and TAK within clinical trials and observational studies and to make comparisons with the general population and HRQoL in other diseases. The development of a disease-specific PRO for GCA is currently underway. Beyond clinical trials, there is much interest in the use of PROs within routine clinical care, particularly E-PROs for remote use. Summary Further work will be needed to complete the development of disease-specific PROs for people with large vessel vasculitis and to establish feasibility, acceptability, and utility of E-PROs.

BACKGROUND Two-stage revision arthroplasty with an antibiotic-loaded spacer is the treatment of choice in chronically infected total hip arthroplasties. Interval spacers can be functional articulating or prefabricated. Functional results of these spacers have scarcely been reported. AIM To compare retrospectively the patient reported outcome and infection eradication rate after two-stage revision arthroplasty of the hip with the use of a functional articulating or prefabricated spacer. METHODS All patients with two-stage revision of a hip prosthesis at our hospital between 2003 and 2016 were included in this retrospective cohort study. Patients were divided into two groups; patients treated with a functional articulating spacer or with a prefabricated spacer. Patients completed the Hip Osteoarthritis Outcome Score and the EQ-5D-3L (EQ-5D) and the EQ-5D quality of life thermometer (EQ-VAS) scores. Primary outcomes were patient reported outcome and infection eradication after two-stage revision. The results of both groups were compared to the patient acceptable symptom state for primary arthroplasty of the hip. Secondary outcomes were complications during spacer treatment and at final follow-up. Descriptive statistics, mean and range are used to represent the demographics of the patients. For numerical variables, students’ t-tests were used to assess the level of significance for differences between the groups, with 95% confidence intervals; for binary outcome, we used Fisher’s exact test. RESULTS We consecutively treated 55 patients with a prefabricated spacer and 15 patients with a functional articulating spacer of the hip. The infection eradication rates for functional articulating and prefabricated spacers were 93% and 78%, respectively (P > 0.05). With respect to the functional outcome, the Hip Osteoarthritis Outcome Score (HOOS) and its subscores (all P < 0.01), the EQ-5D (P < 0.01) and the EQ-VAS scores (P < 0.05) were all significantly better for patients successfully treated with a functional articulating spacer. More patients in the functional articulating spacer group reached the patient acceptable symptom state for the HOOS pain, HOOS quality of life and EQ-VAS. The number of patients with a spacer dislocation was not significantly different for the functional articulating or prefabricated spacer group (P > 0.05). However, the number of dislocations per patient experiencing a dislocation was significantly higher for patients with a prefabricated spacer (P < 0.01). CONCLUSION Functional articulating spacers lead to improved patient reported functional outcome and less perioperative complications after two-stage revision arthroplasty of an infected total hip prosthesis, while maintaining a similar infection eradication rate compared to prefabricated spacers.

Sexual dysfunction (SD) in people with multiple sclerosis (pwMS) has a detrimental impact on individual health-related quality of life (HRQoL). It is not clear whether SD in multiple sclerosis (MS) is an independent symptom or merely a byproduct of other symptoms such as depression or anxiety. This cross-sectional study of 93 pwMS determines risk factors for SD in MS based on prevalence, HRQoL, and associated disease outcomes. Diagnosis of SD was determined based on the Multiple Sclerosis Intimacy and Sexuality Questionnaire-19 (MSISQ-19) and correlated with physical disability (measured by Expanded Disability Status scale, EDSS), depression and anxiety [Hospital Anxiety and Depression Scale (HADS)], and HRQoL [Multiple Sclerosis Quality of Life-54 (MSQoL-54)]. Multivariate regression models were performed to determine independent risk factors for SD in pwMS. Almost half of the participants in this study (46%) reported SD. HRQoL was significantly poorer in patients with MS suffering from SD (median [IQR] MSQoL-54 scores: physical subscale 52 [41–68] vs. 81 [69–89], p < 0.001; mental subscale 50 [38–82] vs. 86 [70–89], p < 0.001). In the multivariate model, EDSS was the only independent risk factor for SD (OR 18.1 for EDSS ≥4 [95% CI 3.3–31.4, p < 0.001]), while depression and anxiety were not. We conclude that the risk for SD is growing with increasing EDSS and is independent of depression or anxiety. Screening for SD becomes particularly relevant in patients with growing disability.

Background The management of diabetes is complex. There is growing recognition of the use of patient-reported outcome measures (PROMs) as a standardized method of obtaining an outlook on patients’ functional status and well-being. However, no systematic reviews have summarized the studies that investigate the measurement properties of diabetes PROMs. Objective Our aims were to conduct a systematic review of studies investigating the measurement properties of diabetes PROMs by evaluating the methodological quality and overall level of evidence of these PROMs and to categorize them based on the outcome measures assessed. Methods This study was guided by the PRISMA (Preferred Reporting Items for Systematic Review and Meta-Analysis) guidelines. Relevant articles were retrieved from the Embase, PubMed, and PsychINFO databases. The PROMs were evaluated with the COSMIN (COnsensus-based Standards for the selection of health Measurement Instruments) guidelines. Results A total of 363 articles evaluating the measurement properties of PROMs for diabetes in the adult population were identified, of which 238 unique PROMs from 248 studies reported in 209 articles were validated in the type 2 diabetes population. PROMs with at least a moderate level of evidence for ≥5 of 9 measurement properties include the Chinese version of the Personal Diabetes Questionnaire (C-PDQ), Diabetes Self-Management Instrument Short Form (DSMI-20), and Insulin Treatment Appraisal Scale in Hong Kong primary care patients (C-ITAS-HK), of which the C-PDQ has a “sufficient (+)” rating for >4 measurement properties. A total of 43 PROMs meet the COSMIN guidelines for recommendation for use. Conclusions This study identified and synthesized evidence for the measurement properties of 238 unique PROMs for patients with type 2 diabetes and categorized the PROMs according to their outcome measures. These findings may assist clinicians and researchers in selecting appropriate high-quality PROMs for clinical practice and research. Trial Registration PROSPERO International Prospective Register of Systematic Reviews CRD42020180978; https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42020180978.

Background Listening to “patient voices” in terms of symptoms, emotional status and experiences with care, is crucial for patient empowerment in clinical practice. Despite convincing evidence that routine patient reported outcomes and experience measurements (PRMs) with rapid feed-back to oncologists can improve symptom control, patient well-being and cost effectiveness, PRMs are not commonly used in cancer care, due to barriers at various level. Part of these barriers may be overcome through electronic PRMs collection (ePRMs) integrated with the electronic medical record (EMR). The PATIENT VOICES initiative is aimed at achieving a stepwise integration of ePRMs assessment into routine cancer care. The feasibility project presented here is aimed at assessing the knowledge, use and attitudes toward PRMs in a comprehensive cancer centre; developing and assessing feasibility of a flexible system for ePRM assessment; identifying barriers to and developing strategies for implementation and integration of ePRMs clinical practice. Methods The project has been organized into four phases: a) pre-development; b) software development and piloting; c) feasibility assessment; d) post-development. A convergent mixed method design, based on concurrent quantitative and qualitative data collection will be applied. A web-survey on health care providers (HCPs), qualitative studies on patients and HCPs (semi-structured interviews and focus groups) as well as longitudinal and cross-sectional quantitative studies will be carried out. The quantitative studies will enroll 600 patients: 200 attending out-patient clinics (physical symptom assessement), 200 attending inpatient wards (psychological distress assessment) and 200 patients followed by multidisciplinary teams (patient experience with care assessment). The Edmonton symptom assessment scale, the Distress Thermometer, and a tool adapted from existing patient reported experience with cancer care questionnaires, will be used in quantitative studies. A multi-disciplinary stakeholder team including researchers, clinicians, health informatics professionals, health system administrators and patients will be involved in the development of potentially effective implementation strategies in the post development phase. Discussion The documentation of potential advantages and implementation barriers achieved within this feasibility project, will serve as a starting point for future and more focused interventions aimed at achieving effective ePRMs routine assessment in cancer care. Trial registration ClinicalTrials.gov (NCT03968718) May 30th, 2019.

Background Multiple Sclerosis (MS) clinical trials increasingly focus on progressive and advanced MS, with upper limb function (ULF) as a key outcome. Within clinical trials, Patient Reported Outcomes (PROs) quantify clinical variables and establish meaningfulness of changes. Scientific standards and regulatory criteria (from Food and Drug Administration [FDA]) require PROs be “fit-for-purpose”: well-defined and reliable measures of specific concepts in defined contexts. Objective To identify, from literature, existing PROs measuring ULF and determine which satisfy scientific and regulatory clinical trials requirements. Method We screened PubMed/Web of Science using multiple relevant terms. Abstracts and full texts were screened using suitability criteria. PRO development papers were evaluated using recently expanded Consensus Standards for Measurement Instruments (COSMIN) criteria for content development. Results We identified 3619 articles; 485 used 24 different ULF PROs. No PRO satisfied scientific and regulatory requirements as a well-defined measure of a clearly defined construct in a specific clinical context. Conclusions Existing ULF PROs don't meet fit-for-purpose criteria. MS clinical trials require new measures with greater emphasis on patient engagement to derive theoretical frameworks, concepts of interest, and contexts of use followed by systematic literature searches, expert input, and qualitative research to support item generation. Until then, trials will miss aspects of meaningful within-patient change and thereby misrepresent (likely underestimating) treatment effects., Highlights • With MS clinical trials increasingly focusing on progressive and advanced MS, we searched for and assessed existing Upper Limb Function (ULF) Patient Reported Outcome (PRO) for MS clinical trials • We established that existing PROs for ULF in MS do not meet regulatory and scientific criteria for content validity. • In fact, we established that the content development of existing PROs is worrisome – often falling short of guidelines around conceptual development, qualitative involvement of patients, use of representative samples and well-documented literature searches. • These results reiterate the work of other authors, where poor historical PRO content development has been established in various clinical contexts. • New measures are required.

Objectives For valid and reliable assessment of patients’ Health-Related Quality of Life (HRQoL), it is crucial to use psychometrically robust instruments. In the context of rare diseases such as Merkel cell carcinoma (MCC), validated disease-specific instruments are often not available. The Functional Assessment of Cancer Therapy – Melanoma (FACT-M) was originally developed for use in melanoma. Its psychometric performance for use in MCC and minimal important difference (MID) thresholds have been previously reported based on a cohort of metastatic MCC patients who had disease progression following one or more prior line of chemotherapy (NCT02155647 Part A; n = 70). Since then, new data from the phase II JAVELIN Merkel 200 trial among treatment-naïve patients are available (NCT02155647 Part B; n = 102). This study aims to increase accuracy and precision of previously established psychometric properties and MID thresholds of FACT-M in metastatic MCC patients. Methods Published qualitative research suggests that patients with metastatic MCC had similar experiences and described similar concepts associated with their disease independent of whether they were treatment naïve or had prior treatment. Therefore, it was deemed appropriate to pool FACT-M data from Part A (previously treated) and Part B (treatment-naïve) cohorts for this study. Construct validity was assessed by evaluating item-factor correlations (convergent validity) and known-groups validity using ECOG performance status 0 versus 1. Concurrent validity was assessed using EQ-5D items. Internal consistency reliability was assessed using Cronbach’s α. Anchor- and distribution-based approaches were used to derive MID thresholds. Results Overall, psychometric tests based on various validity (convergent, known-groups, concurrent) and reliability (Cronbach α) analyses confirmed previous findings in that FACT-M performs well in MCC patients. MID thresholds derived from this study are largely in line with previously established thresholds with some minor adjustments. Conclusions In the context of rare diseases, which often have limited data available for psychometric testing, a reasonably large MCC patient sample was available for this study, enhancing accuracy and precision of previously established FACT-M psychometric properties and MID thresholds with only small deviations for use in metastatic MCC patients. Results suggest that the FACT-M is suitable for Merkel cell carcinoma regardless of patients’ treatment status. Trial registration This study is a pre-planned post-hoc analysis conducted on data collected in Part A and Part B of the JAVELIN Merkel 200 trial. This trial was registered on 2 June 2014 with ClinicalTrials.gov as NCT02155647.

Objective Wrist deformity in older people is common following treatment for a wrist fracture, particularly after non-surgical treatment. A cohort of older wrist fracture patients were surveyed by telephone regarding perceived deformity, bother with deformity and patient-reported wrist function. The objectives were to: (1) determine whether older patients with wrist fractures perceived a deformity of their wrist and if they were bothered by it; (2) test if there were associations between deformity and treatment-type and between deformity and function; (3) test for associations between bother and treatment-type and between bother and function; (4) measure the test–retest reliability of the ‘bother’ question. Results Of 98 eligible patients who were invited to participate, 41 responded. Out of 41, 14 (34%) believed they had a deformity and 4 (10%) reported that they were bothered by the appearance of their wrist. Deformity was associated with non-surgical treatment (RR = 3.85, p = 0.006) but was not significantly associated with functional outcomes (p = 0.15). All those who were bothered belonged to the non-surgical treatment group. Bother was significantly associated with poorer functional outcomes (p = 0.006) and this association was clinically significant (MD = 35 points). The deformity and bother questions were found to have excellent test–retest reliability; κ = 1.00 and κ = 0.92, respectively.

Background: Urethral stricture disease (USD) represents a complex urological problem. Urethroplasty is considered the gold standard for the treatment of USD. Most available studies report outcome data obtained from retrograde urethrography and uroflowmetry. Only a limited number of papers describe the effect of urethroplasty on erectile function and their results are inconsistent. The goal of this prospective study was to evaluate the effect of urethroplasty on both lower urinary tract and erectile function using objective parameters and standardized patient-reported outcome measurement tools. Materials and Methods: A total of 55 consecutive patients with USD were enrolled into the study. Patients underwent ventral onlay urethroplasty, urethroplasty according to the Asopa technique, dorsal onlay urethroplasty, cutaneous flap urethroplasty using the Orandi technique or anastomotic repair. All patients were evaluated using uroflowmetry, urethrography, the PROM-USS questionnaire and the International Index of Erectile Function-5 questionnaire (IIEF-5) pre-operatively and consequently post-op, in 3-month intervals. This study presents the comparison of baseline pre-op parameters and parameters 12 months after the surgery using the Wilcoxon signed rank test, Wilcoxon rank sum test and the Kruskal-Wallis one-way analysis of variance. Results: A significant improvement in uroflowmetry parameters, all domains of the PROM-USS questionnaire, as well as the overall score of the IIEF-5 was observed. No statistically significant differences between sub-groups were found when comparing treatment results in patients with short versus long strictures and patients with penile urethra stricture versus bulbar or membranous urethra stricture. Conclusions: Urethroplasty yielded very good functional results with respect to both lower urinary tract and erectile functions.

Purpose The Short Musculoskeletal Function Assessment (SMFA) questionnaire can be used to evaluate physical functioning in patients with traumatic injuries. It is not known what change in score reflects a meaningful change to patients. The aim was to determine minimal important change (MIC) values of the subscales (0–100) of the Dutch SMFA-NL in a sample of patients with a broad range of injuries. Methods Patients between 18 and 65 years of age completed the SMFA-NL and the Global Rating of Effect (GRE) questions at 6-week and 12-month post-injury. Anchor-based MIC values were calculated using univariable logistic regression analyses. Results A total of 225 patients were included (response rate 67%). The MIC value of the Upper Extremity Dysfunction (UED) subscale was 8 points, with a misclassification rate of 43%. The Lower Extremity Dysfunction subscale MIC value was 14 points, with a misclassification rate of 29%. The MIC value of the Problems with Daily Activities subscale was 25 points, with a misclassification rate of 33%. The MIC value of the Mental and Emotional Problems (MEP) subscale was 7 points, with a misclassification rate 37%. Conclusion MIC values of the SMFA-NL were determined. The MIC values aid interpreting whether a change in physical functioning can be considered clinically important. Due to the considerable rates of misclassification, the MIC values of the UED and MEP subscales should be used with caution.

Background Composite measures combine data to provide a comprehensive view of patient outcomes. Despite composite measures being a valuable tool to assess post-intervention outcomes, the patient perspective is often missing. The purpose of this study was to develop a composite measure for an established cardiac outcome registry, by combining clinical outcomes following percutaneous coronary interventions (PCI) with a patient-reported outcome measure (PROM) developed specifically for this population (MC-PROM). Methods Two studies were undertaken. Study 1: Patients who had undergone a PCI at one of the three participating registry hospital sites completed the 5-item MC-PROM. Clinical outcome data for the patients (e.g. death, myocardial infarction, repeat vascularisation, new bleeding event) were collected 30 days post-intervention as part of routine data collection for the cardiac registry. Exploratory factor analysis of clinical outcomes and MC-PROM data was conducted to determine the minimum number of constructs to be included in a composite measure. Study 2: Clinical experts participated in a Delphi technique, consisting of three rounds of online surveys, to determine the clinical outcomes to be included and the weighting of the clinical outcomes and MC-PROM score for the composite measure. Results Study 1: Routine clinical outcomes and the MC-PROM data were collected from 266 patients 30 days post PCI. The MC-PROM score was not significantly correlated with any clinical outcomes. Study 2: There was a relatively consistent approach to the weighting of the clinical outcomes and MC-PROM items by the expert panel (n = 18) across the three surveys with the exception of the clinical outcome of ‘deceased at 30 days’. The final composite measure included five clinical outcomes within 30 days weighted at 90% (new heart failure, new myocardial infarction, new stent thrombosis, major bleeding event, new stroke, unplanned cardiac rehospitalisation) and the MC-PROM score (comprising 10% of the total weighting). Conclusions A single patient level composite score, which incorporates weighted clinical outcomes and a PROM was developed. This composite score provides a more comprehensive reported measure of individual patient wellbeing at 30 days post their PCI-procedure, and may assist clinicians to further assess and address patient level factors that potentially impact on clinical recovery.

Background Patient‐reported outcomes ( PRO s) objectively measure health‐related quality of life and provide prognostic information. Advances in technology now allow for rapid, patient‐friendly PRO assessment and scoring, yet the adoption of PRO s in clinic has been slow. We conducted a multicenter qualitative study of diverse providers to describe the barriers and facilitators of routine PRO use in heart failure clinics. Methods and Results Sixty heart failure providers from 5 institutions participated in 8 focus groups to explore provider perspectives on the use of heart failure‐specific and generic PRO s in clinical practice. A qualitative editing approach was used to analyze the data, whereby a coding dictionary was iteratively developed and applied using the qualitative software program Altas.ti. Three main themes, supporting and impeding PRO use, emerged: (1) data collection; (2) presentation and interpretation; and (3) utility and value. For each construct, we identified perspectives that highlighted both barriers and facilitators. Providers identified burden, survey fatigue, and language/health literacy barriers as potentially impeding data collection. Optimal workflow, PRO frequency and length, use of PRO translations, and assistance of a patient's proxy were suggested as facilitators. Focus group discussions provided insight on how to display PRO s to support its interpretability and sharing. Furthermore, the need to educate providers on the utility and value PRO s over and above current clinical approaches emerged. Conclusions Overcoming the barriers and supporting facilitators of PRO adoption could potentially lead to more successful adoption of PRO s in heart failure clinics.

International audience; Background - Heart failure with preserved ejection fraction (HFpEF) is associated with poor quality of life (QoL), or patient reported outcome (PRO). Despite female predominance in HFpEF, sex-specific differences in PROs remain poorly studied. We assessed PRO measures and their association with HF-severity and outcome in HFpEF by sex. Methods and results - In 378 patients with HFpEF from the KaRen study, EQ-5D-3L® and Minnesota Living with Heart Failure Questionnaire® (MLHFQ) were assessed. Characteristics and comorbidities were largely similar in women (n = 212, 57%) and men. Women expressed worse QoL in EQ-5D-3L®(EQ-VAS), independent of age and HF-severity, mean (SD), 57 (20) vs. 61 (19), p = 0.010. There was no difference in MLHFQ, 31 (21) vs. 29 (21), p = 0.269. Spearman's correlations with HF-severity (NYHA-class) were for MLHFQ in women r 0.37 vs. men 0.41, p for both

In the last few decades, strategies for the management of rheumatoid arthritis (RA) have been increasingly oriented toward more comprehensive control of the disease, taking into account even RA extra-articular manifestations, comorbidities, and the patient's perception about the disease. The need for improving the shared decision-making process suggested by European League Against Rheumatism recommendations is leading to an increasing interest in the role of patient-reported outcomes (PROs) beside the usual more objective criteria for defining clinical response based on disease-activity composite indices. Measurement of such PROs as pain or fatigue may be significantly influenced by mood disorders often complicating RA, the pathogenesis of which is deeply interconnected with phlogistic processes mediated by proinflammatory cytokines. IL6 is a pleiotropic mediator involved in neuroendocrine and neuropsychological processes, besides its well known effects on immune, cardiovascular, and metabolic systems. Therefore, there is a growing body of evidence about the efficacy of IL6 blockade in PRO improvement in RA patients. Sarilumab is a monoclonal antibody binding both soluble and membrane-bound IL6Rα, inhibiting the IL6-mediated signaling pathway with favorable efficacy and safety profile. This review analyzes the importance of PROs in strategies for the management of RA and the pathogenic mechanisms linking IL6 with the patient's perception of the disease. Moreover, the main findings from sarilumab randomized controlled trials are summarized in detail, emphasizing the potential role of this IL6 blocker in the holistic treatment of RA.

Objective To identify and assess patient motivation to initiate or maintain behavior changes. Methods Attitudinal statements were developed from structured patient interviews and translated into 18 survey items. Items were analyzed with exploratory factor analysis (EFA). Results An EFA with 340 type 2 diabetes patients identified three areas of patient attitudes toward changing health behaviors: (1) willingness to make changes (3 items; α = 0.69), (2) perceived ability to make or maintain changes (3 items; α = 0.74), and (3) and feeling changes are worthwhile (3 items; α = 0.61). Greater perceived ability and feelings of worthwhileness were associated with positive psychosocial and behavioral management indicators. All three areas were associated with confidence and attitudes toward making a specific behavioral change (e.g., improve diet). Conclusions MATCH is an internally consistent and valid 9-item scale that provides a profile of factors influencing motivation that can be used in clinical and research settings.

Patients with fibromyalgia (FM) have often tried many outpatient treatments, and breakthrough pain frequently stops them from performing everyday tasks and participating in society. FM-pain that has become chronic, therefore, affects quality of life. This case study describes the administration of interdisciplinary inpatient pain therapy due to a primary diagnosis of fibromyalgia with integrated complementary medicine. The female patient, who had several concomitant disorders and had been suffering from pain for many years, benefited from the holistic treatment approach in terms of a reduction in pain and an improvement in physical functions and mental health. The ability to have a positive effect on pain symptoms in the longer term is essential. Compliance with the treatments used should also be improved, so that a healthier lifestyle and better pain management can continue after discharge from hospital. These results are supported by other study results and should provide the impetus for major studies to evaluate holistic pain therapies in FM. Keywords: Fibromyalgia, Chronic pain, Integrative medicine, OPS 8-983, Patient reported outcome, Complementary medicine