Factors Affecting Patient Satisfaction with Double-Eyelid Blepharoplasty.

BACKGROUND: Blepharoplasty is a common surgical technique performed in individuals seeking aesthetic enhancement. Thus, it is essential to investigate the factors influencing postoperative satisfaction from the patient's perspective. In this study, patient-rated outcome measure questionnaires were used to identify the factors affecting patient satisfaction after full-incision upper blepharoplasty. METHODS: This retrospective study analyzed patients who underwent full-incision upper blepharoplasty at an outpatient clinic in China. The questionnaire responses were collected by telephone, text messaging, or email at 6 and 12 months postoperatively. RESULTS: In total, 149 questionnaires were collected. After a mean follow-up of 23.23 months, the patients' overall satisfaction rate was 89.43%. The factors that significantly affected postoperative satisfaction were the patient's education level, the source of referral to the surgeon, the patient's understanding of the surgical risks, application of a cold compress after surgery as recommended, unsatisfactory postoperative double-eyelid width, postoperative bilateral asymmetry, apparent postoperative cicatrices, and postoperative caterpillar-like appearance of the double eyelids. Education level, apparent postoperative cicatrices, and postoperative bilateral asymmetry influenced the patient's satisfaction with the surgical outcome. The patient's understanding of the surgical risks, unsatisfactory postoperative double-eyelid width, postoperative bilateral asymmetry, apparent postoperative cicatrices, and postoperative caterpillar-like appearance influenced the satisfaction of the patient's family and friends. CONCLUSIONS: Postoperative bilateral asymmetry, apparent postoperative cicatrices, and a low education level of the patient are independent factors that negatively affect patient satisfaction with the outcome of double-eyelid blepharoplasty. LEVEL OF EVIDENCE IV: This journal requires that authors assign a level of evidence to each article. For a full description of these Evidence-Based Medicine ratings, please refer to the Table of Contents or the online Instructions to Authors www.springer.com/00266 .

Falls in people with mobility limitations: a cross-sectional analysis of a US registry of assistive device users.

Purpose of the Article: To (1) summarise the personal and clinical characteristics of persons with disabilities (PwDs) in the US who were evaluated for mobility assistive equipment (MAE) in the functional mobility assessment and uniform dataset (FMA/UDS) and (2) stratify subpopulations of PwD who reported falling versus those who do not report a fall.Materials and Methods: This study was a retrospective, descriptive cohort analysis of adults with disabilities using the FMA/UDS. Data are collected during a user's initial evaluation for a new mobility device. The sample is intentionally general to be inclusive of all mobility device users. The primary variable of interest was a patient-reported fall within the 3 months leading up to their evaluation for a new mobility device. Subpopulation characteristics were stratified by this binary fall variable.Results and Conclusions: This study provides descriptions of PwDs being evaluated for a new mobility device. There were 11,084 PwDs with 31 different primary diagnoses. During their new mobility device evaluation, 52.2% of PwDs reported at least one fall in the last 3 months. For those who reported a fall, 46.6% of PwDs were using a walking aid or no device at all before the new mobility device evaluation. Additionally, persons with progressively acquired disabilities (i.e., Parkinson's disease, osteoarthritis and cardiopulmonary disease) reported higher rates of falls than those with congenital disabilities (i.e., cerebral palsy and spina bifida). These findings will influence future studies comparing different types of devices and their influence on falls and user satisfaction.Implications for rehabilitation52.2% of persons with disabilities (PwDs) seeking a new wheelchair evaluation reported at least one fall in the last 3 months.Persons with progressively acquired disabilities (i.e., Parkinson's disease, osteoarthritis and cardiopulmonary disease) reported higher rates of falls than those with congenital disabilities (i.e., cerebral palsy and spina bifida).Earlier interventions for fall prevention including professional wheelchair evaluations may be warranted, but further research is necessary to explore long-term effectiveness.

Gender Differences in Perceptions of Psoriatic Arthritis Disease Impact, Management, and Physician Interactions: Results from a Global Patient Survey.

INTRODUCTION: We evaluated the impact of gender on disease severity, health-related quality of life (HRQoL), treatment management, and patient-healthcare professional (HCP) interactions from the perspectives of patients with psoriatic arthritis (PsA). METHODS: Data were collected from a global online patient survey conducted by The Harris Poll (November 2, 2017 to March 12, 2018). Eligible patients were aged ≥ 18 years, with a self-reported diagnosis of PsA for > 1 year, had visited a rheumatologist/dermatologist in the past 12 months, and had reported previously using ≥ 1 conventional synthetic or biologic disease-modifying antirheumatic drug. Data were stratified by gender and analyzed descriptively, inferentially by binomial (chi-square) tests, and by multivariate logistic regression models. RESULTS: Data from 1286 patients who participated were included: 52% were female, 48% were male. Varying perceptions of disease severity between males and females were indicated by differences in symptoms leading to a diagnosis of PsA, and in symptoms reported despite treatment; more females than males reported joint tenderness, skin patches/plaques, and enthesitis. More females than males reported a major/moderate impact of PsA on their physical activity and emotional/mental well-being. Reasons for switching medication differed between genders, with more females switching because they perceived their medication to not be effective enough related to their joint symptoms. More females than males were very satisfied with their communication with their rheumatologist and were more likely to discuss the impact of PsA on their daily lives, their treatment satisfaction, and treatment goals with their rheumatologist. CONCLUSIONS: Patients' perceptions of the impact of PsA on HRQoL, treatment management, and interactions with HCPs varied between males and females. More females than males reported major/moderate physical and emotional impacts of PsA. When treating patients, it is important for HCPs to consider the potential impact of gender on patients' experience of PsA and its symptoms. Graphical plain language summary available for this article.

Growth Factor Concentrate (GFC) for the Management of Osteoarthritis of the Knee: A Systematic Review.

Introduction: The knee is the most commonly affected joint in osteoarthritis (OA), affecting millions of people worldwide. Knee OA significantly impacts the activities of daily living (ADL) along with affecting overall quality of life of patients (QoL), thereby leading to substantial socio-economic burden. Conservative therapies are prioritized, resorting to surgery only when needed. However, these traditional approaches have limitations. Regenerative medicine, involving the use of orthobiologics, including autologous peripheral blood-derived orthobiologics such as growth factor concentrate (GFC), has evolved and shown potential for managing knee OA. The primary goal of this review is to summarize the results of in vitro, preclinical and clinical studies involving GFC for the management of knee OA. Methods: Multiple databases (PubMed, Scopus, Google Scholar, Web of Science and Embase) were searched applying terms for the intervention 'GFC' and treatment 'knee OA' for the studies published in the English language to March 10, 2024. Results: Only three clinical studies met our pre-defined criteria and were included in this review. Conclusion: Intra-articular administration of GFC is safe and potentially efficacious to manage OA of the knee. More, adequately powered, multi-center, prospective, RCTs are warranted to demonstrate the long-term effectiveness of GFC in patients suffering from mild-to-moderate knee OA and to justify its routine clinical use. Further studies evaluating the efficacy of GFC compared to other orthobiologics are also required to allow physicians/surgeons to choose the optimal orthobiologic for the treatment of OA of the knee.

Does Platelet-Rich Plasma Deserve a Role in Accelerating the Recovery of Reflex Sympathetic Dystrophy Following Distal Radius Fracture?

Introduction: This study was to evaluate the efficacy of multiple platelet-rich plasma injections in reflex sympathetic dystrophy following distal radius fracture after previous various treatments have failed. Materials and methods: This comparative prospective study was designed for 64 patients of reflex sympathetic dystrophy developed following distal radius fracture, from January 2009 to December 2020 were enrolled in this study. This cohort of patient was given either four multiple subcutaneous platelet-rich plasma injections at weekly interval (n = 32) or two injections in a month with 15 days interval (n = 32). The primary outcome measure assessed with patient rated wrist evaluation questionnaire score. The secondary outcome was a visual analogue scale pain score. The final follow up was at 2 years. p ≤ 0.05 is considered statistically. Results: The patient rated wrist evaluation score for usual and specific activities and EQ-VAS for pain level showed statistically significant greater improvement in group A (42 ± 21%) compared to group B (19 ± 24%), (p = 0.37). Patients also had improvement in wrist movements with no statistically significant differences in both groups. The standard difference in means of all three functional scores was almost similar between both groups A and B (standard difference in means = 0.032; 95% CI 0.236-0.830; p = 0.495), considered clinically meaningful. Conclusion: This study results suggest autologous platelet-rich plasma injections seem to be safe, cost effective, efficacious algorithm treatment for reflex sympathetic dystrophy following distal radius fracture patients where previous treatments have failed.

Real-World Data on the Effectiveness and Safety of Filgotinib for Ulcerative Colitis in Japanese Patients: A Single-Center Experience.

Introduction Filgotinib is a JAK-1 selective inhibitor approved for ulcerative colitis (UC) treatment in Japan. Its effectiveness has been confirmed but remains unknown in actual clinical practice. Therefore, we aimed to evaluate the effectiveness and safety of filgotinib and identify suitable patients in the Japanese population. Methods We retrospectively reviewed the background, clinical course, and laboratory data of patients treated with filgotinib 200 mg for UC between May 2022 and December 2023. Results The median observation period for the 25 patients was 232 days (interquartile ranges (IQR) 102-405). The median age of the patients was 43 years (IQR 29-55), disease duration was nine years (IQR 2-12), and 36% (9/25) of patients were biologic or small molecule naïve. The median patient-reported outcome (PRO2) and partial Mayo (pMayo) scores at agent initiation were 3 (IQR 1-4) and 4.5 (IQR 3-6), respectively. The PRO2 and pMayo scores improved significantly two weeks after treatment initiation (p < 0.05). Clinical remission rates at 24 weeks after treatment initiation were 60% (15/25) for PRO2 ≤ 1 and 52% (13/25) for pMayo ≤ 1. The Mayo endoscopic subscore significantly improved after filgotinib initiation (p=0.04), and the endoscopic remission rate was 47% (8/17). At 24 weeks, patients in clinical remission, compared to those not in remission, had significantly lower baseline PRO2 and pMayo scores and longer disease duration (p=0.03, p=0.03, and p=0.04, respectively). The filgotinib persistence rate was 68% (17/25), with no discontinuation because of adverse events. Patients who continued treatment had significantly lower PRO2, pMayo scores, and blood neutrophil counts at initiation than those who discontinued (p=0.02, p=0.03, and p=0.02, respectively). Conclusion Filgotinib appears to be effective and safe in Japanese patients with UC. Effectiveness and persistence were high in patients whose PRO2 and pMayo scores were low at the time of treatment initiation.

Patient-reported outcomes in HSCT for autoimmune diseases: Considerations on behalf of the EBMT ADWP, PAC, and Nurses Group.

Background: Over the last 3 decades, hematopoietic stem cell transplantation (HSCT) has been successfully used to treat severe and refractory autoimmune diseases (AIDs). A multidisciplinary appraisal of potential benefits and risks by disease and transplant specialists is essential to determine individual suitability for HSCT. Objective: Our aim was to observe that patient-reported outcomes (PROs) and health-related quality of life instruments can capture the unique patient perspective on disease burden and impact of treatment. Methods: Herein, we describe the basis and complexity of end points measuring patient-reported perceptions of efficacy and tolerability used in clinical practice and trials for patients with AIDs undergoing autologous HSCT. Results: PRO measures and patient-reported experience measures are key tools to evaluate the impact and extent of disease burden for patients affected by AIDs. For formal scientific assessment, it is essential that validated general instruments are used, whereas adaptations have resulted in disease-specific instruments that may help guide tailored interventions. An additional approach relates to qualitative evaluations, from carefully structured qualitative research to informal narratives, as patient stories. The patients' subjectively reported responses to HSCT may be influenced by their preprocedure expectations and investment in the HSCT journey. Conclusions: The complexity of AIDs advocates for individualized and multidisciplinary approach to positively affect the patient journey. PROs and health-related quality of life need to be collected using validated instruments in clinical practice and trials to enable robustness of data and to ensure the impact of the intervention is comprehensively assessed, addressing the main questions and needs of the involved stakeholders.

Examining the "revisability" benefit of hip resurfacing arthroplasty.

BACKGROUND: Hip resurfacing arthroplasty (HRA) is an alternative to total hip arthroplasty (THA) that is typically reserved for young active patients because it preserves bone. However, the benefits of HRA only hold true if conversion THA after failed HRA provides acceptable outcomes. AIM: To compare patient reported outcomes for conversion THA after HRA failure to primary THA. METHODS: A retrospective review of 36 patients (37 hips) that underwent conversion THA for failed HRA between October 2006 and May 2019 by a single surgeon was performed. Patient reported outcomes [modified Harris Hip Score (mHHS), University of California Los Angeles (UCLA) activity score] were obtained via an email-based responder-anonymous survey. Outcomes were compared to normative data of a primary THA cohort with similar demographics. Subgroup analysis was performed comparing outcomes of conversion THA for adverse local tissue reaction (ALTR) vs all other causes for failure. RESULTS: The study group had a lower mHHS than the control group (81.7 ± 13.8 vs 90.2 ± 11.6, P < 0.01); however, both groups had similar UCLA activity levels (7.5 ± 2.3 vs 7.2 ± 1.6, P = 0.51). Patients that underwent conversion for non-ATLR causes had similar mHHS (85.2 ± 11.5 vs 90.2 ± 11.6, P = 0.11) and higher UCLA activity levels (8.5 ± 1.8 vs 7.2 ± 1.6, P < 0.01) compared to the control group. Patients that underwent conversion for ATLR had worse mHHS (77.1 ± 14.5 vs 90.2 ± 11.6, P < 0.01) and UCLA activity levels (6.1 ± 2.3 vs 7.2 ± 1.6, P = 0.05) when compared to the control group. CONCLUSION: Patient outcomes equivalent to primary THA can be achieved following HRA conversion to THA. However, inferior outcomes were demonstrated for ALTR-related HRA failure. Patient selection and perhaps further studies examining alternative HRA bearing surfaces should be considered.

Asking the "Right" Questions about Financial Hardship: Using Cognitive Interviews with Adolescents and Young Adults with Cancer and Their Caregivers to Inform Measure Development.

Purpose: Financial hardship as a result of cancer treatment can have a significant and lasting negative impact on adolescents and young adults (AYAs) and their families. To address a lack of developmentally informed and psychometrically sound measures of financial hardship for AYAs and their caregivers, we used rigorous measurement development methods recommended by the National Institutes of Health's Patient-Reported Outcomes Measurement Information System® (PROMIS®) to determine comprehensibility and relevance of measure content. Methods: Our multi-step approach involved item identification, refinement, and generation; translatability and reading level review; and cognitive interviews. A purposive sample of 25 AYAs and 10 caregivers participated, ensuring representation across age, education, gender, race/ethnicity, and cancer type. Results: Fifty patient-reported and caregiver-reported items were developed across material, psychosocial, and behavioral subdomains of financial hardship. Translatability and reading level reviews resulted in 22 patient-reported and 25 caregiver-reported items being rewritten. Eighty-eight percent of patients and all caregivers described the items as easy to answer. Younger AYAs (15 to 25 years of age) were more likely to say the items were less relevant for them. Forty-six patient-reported and 48 caregiver-reported items were recommended for further testing. Conclusion: This study is the first to use in-depth qualitative methods to center AYA patient and caregiver experiences in the creation of new measures of financial hardship. Data support the comprehensibility and content validity of these preliminary item banks. Future large-scale, quantitative testing will lead to additional refinements and support the use of short forms and computer-adaptive testing for a diverse sample of AYAs and their caregivers.

Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial.

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

Comparison of early postoperative patient-reported outcomes after multiportal robotic-assisted thoracoscopic surgery and uniportal video-assisted thoracoscopic surgery for non-small cell lung cancer.

INTRODUCTION: We aimed to compare early postoperative patient-reported outcomes between multiportal robotic-assisted thoracoscopic surgery (M-RATS) and uniportal video-assisted thoracoscopic surgery (U-VATS) for non-small-cell lung cancer (NSCLC). MATERIALS AND METHODS: Symptom severity and functional status were measured using the Perioperative Symptom Assessment for Lung Surgery at pre-surgery, during postoperative hospitalisation, and within 4 weeks of discharge. A propensity score-matched (PSM) analysis of patients with NSCLC who were treated with M-RATS and U-VATS was performed. The symptom severity and daily functional status presented as proportion of moderate-to-severe scores on a 0-10-point scale, were compared using a generalised estimation equation model. RESULTS: We enrolled 762 patients with NSCLC from a prospective cohort (CN-PRO-Lung 3), including 151 and 611 who underwent M-RATS and U-VATS, respectively, before PSM analysis. After 1:1 PSM, two groups of 148 patients each were created. Pain severity (P = 0.019) and activity limitation (P = 0.001) during hospitalisation were higher in the M-RATS group. However, no significant differences existed post-discharge in pain (P = 0.383), cough (P = 0.677), shortness of breath (P = 0.526), disturbed sleep (P = 0.525), drowsiness (P = 0.304), fatigue (P = 0.153), distress (P = 0.893), walking difficulty (P = 0.242), or activity limitation (P = 0.513). M-RATS caused less intraoperative blood loss (P = 0.013), more stations of dissected lymph nodes (P = 0.001), more numbers of dissected lymph nodes (P = 0.001), and less tube drainage on the first postoperative day (P = 0.003) than U-VATS. CONCLUSION: M-RATS and U-VATS achieved comparable symptom burden and functional impairment after discharge. However, compared to U-VATS, M-RATS was associated with more severe pain and activity limitation in the short postoperative period. TRIAL REGISTRATION NUMBER: ChiCTR2000033016.

Measurement properties of utility-based health-related quality of life measures in cardiac rehabilitation and secondary prevention programs: a systematic review.

PURPOSE: To identify utility-based patient-reported outcome measures (PROMs) for assessing health-related quality of life (HRQoL) in cardiac rehabilitation and secondary prevention programs (CR) and appraise existing evidence on their measurement properties. Secondly, to link their items to the International Classification of Functioning Disability and Health (ICF) and the International Consortium of Health Outcome Measures (ICHOM) domains for cardiovascular disease (CVD). METHODS: Eight databases were searched. The review followed the COSMIN and JBI guidelines for measurement properties systematic reviews and PRISMA 2020 reporting guidelines. Non-experimental and observational empirical studies of patients ≥ 18 years of age with CVD undergoing CR and assessed quality of life (QoL) or HRQoL using utility-based PROMs or one accompanied by health state utilities were included. RESULTS: Nine PROMs were identified with evidence on measurement properties for three measures: the German translations of SF-12, EQ-5D-5L, and MacNew heart disease HRQoL questionnaire. There was moderate quality evidence for responsiveness and hypothesis testing of the SF-12 and EQ-5D-5L, and high-quality evidence for responsiveness and hypothesis testing for the MacNew. All items of SF-12 and EQ-5D were linked to ICF categories, but four items of the MacNew were not classified or defined. All the PROM domains were mapped onto similar constructs from the ICHOM global sets. CONCLUSION: Three utility-based PROMs validated in CR were identified: the German versions of the EQ-5D and SF-12 and the MacNew questionnaire. These PROMs are linked to a breadth of ICF categories and all ICHOM global sets. Additional validation studies of PROMs in CR are required.

Incorporation of patient-reported outcomes in early-phase clinical trials for childhood and adolescent cancer.

INTRODUCTION: The assessment of quality of life (QoL) should be one of the main objectives in paediatric clinical trials. Even though researchers, regulators and advocates support the use of patient-reported outcomes (PROs), this has not been fully implemented. The aim of this study is to assess the measurement of QoL and the usage of PROs, palatability assessments and medication diaries in early-phase clinical trials for childhood and adolescent cancer. METHODS: Early-phase clinical trials for children and adolescents with cancer opened between 2005 and 2022 at the Royal Marsden Hospital (London, UK) and Vall d'Hebron University Hospital (Barcelona, Spain) were interrogated for trial characteristics and the use of QoL questionnaires, PROs, palatability assessments and medication diaries. RESULTS: Overall, 72 clinical trials were analysed: 12 (16.7%) evaluated QoL and eight (11.1%) evaluated PROs. Palatability was tested in 21/40 (52.5%) trials of oral drugs and 23/72 (31.9%) incorporated medication diaries. No studies mentioned patient involvement in the trial protocol. Use of PROs increased from one of 36 (2.8%) to seven of 36 (19.4%) between the first period (2005-2016) and the second period (2017-2022) (p = .02). Implementation of medication diaries increased from seven of 36 (19.4%) to 16/36 (44.4%) in each period, respectively (p = .02). CONCLUSION: Only a minor proportion of the international/multicentric early-phase trials evaluated included QoL/PROs and medication diaries or palatability questionnaires to help assess these, although this trend seems to be increasing over recent years. Greater implementation of QoL/PROs has the potential to improve the patient's wellbeing and facilitate symptom control, to enhance patient/parent involvement in future trial designs and to provide information for drug prioritisation.

Exploring perceptions of inpatient hospital attire in children with cancer.

BACKGROUND: Attire bolsters identity, self-expression, and comfort. Hospital gowns are known to be distressing in adults. Attitudes of children with cancer toward hospital attire remain uninvestigated and may be a modifiable factor in overall well-being. METHODS: A 39-item mixed methods survey evaluated perceptions of patient attire in children with cancer. Children aged 7-18 years were recruited at an academic medical center. Data analysis included simple statistics and thematic analysis. RESULTS: Forty children with cancer receiving oncologic care participated. Participants' mean age was 12.4 (SD = 3.0, range = 7-17) years, and 25 (62%) were male. Quantitative data revealed 81% of participants preferred their own attire when admitted to the hospital, feeling more comfortable in such when well (91%) or sick (75%). They did not feel like they "must" wear a gown when admitted (60%) and did not want to be asked about preferred inpatient attire (63%). Thematic analysis revealed that children had strong negative views of gowns and preferred to wear their own attire in the hospital, which provided physical and emotional comfort. Children worried wearing their own clothing could impede their care. CONCLUSION: Children with cancer prefer wearing their own clothes in the hospital for physical and emotional comfort. They are willing to wear gowns for ease of care; however, they do not want to arbitrate when they need to make that choice. Providers may ease distress by considering a child's own clothes as default hospital attire with instructions for when a gown is necessary for good clinical care.

Exploring self-reported visual function and vision-related anxiety in patients with RPGR-associated retinal degeneration.

Variants in the retinitis pigmentosa GTPase regulator (RPGR) gene are responsible for the majority of X-linked retinitis pigmentosa cases, which not only affects male patients but also some heterozygous females. Vision-related disability and anxiety of patients with RPGR-associated retinal degeneration have never been explored before. This study aimed to evaluate self-reported visual function and vision-related anxiety in a Portuguese cohort of male and female patients with RPGR-associated retinal degeneration using two validated patient-reported outcome measures. Cross-sectional data of thirty-two genetically-tested patients was examined, including scores of the Michigan retinal degeneration questionnaire (MRDQ) and Michigan vision-related anxiety questionnaire. Patients were classified according to retinal phenotypes in males (M), females with male phenotype (FM), and females with radial or focal pattern. Both M and FM revealed higher rod-function and cone-function anxiety scores (p < 0.017). Most MRDQ disability scores were higher in M and FM (p < 0.004). Overall, positive correlations (p < 0.004) were found between every MRDQ domain and both anxiety scores. In RPGR-associated retinal degeneration, males and females with male phenotype show similar levels of increased vision-related anxiety and disability. Every MRDQ visual function domain showed a strong correlation with anxiety scores.

Effect of Residual Pain After Posterior Fusion Surgery for Lumbar Degenerative Disorders on Health-Related Quality of Life: A Two-Year Follow-Up Using Patient-Reported Outcome Measures.

STUDY DESIGN: This is a prospective cohort study. PURPOSE: The present study aimed to investigate the effects of residual pain after fusion surgery for lumbar degenerative diseases on quality of life (QOL). OVERVIEW OF LITERATURE: Residual symptoms after spinal surgery often restrict patients' activities of daily living and reduce their QOL. However, few studies have comprehensively addressed physical, psychological, and social factors. METHODS: The study population included a cohort of 208 patients (mean age: 67.9 years) who had undergone posterior interbody fusion for lumbar degenerative disease between 2012 and 2019. We asked the patients to complete the Japanese Orthopaedic Association Back Pain Evaluation Questionnaire (JOABPEQ) and Short Form Health Survey (SF-36) preoperatively, as well as at six, 12, and 24 months postoperatively. The presence of residual postoperative pain (RPP) was determined using the low back pain score of the JOABPEQ at six months postoperatively, and patients with an improvement of < 20 points compared to preoperative assessment were classified as RPP+ based on a previous study. RESULTS: In all patients, there was a notable postoperative improvement in all JOABPEQ and SF-36 domains compared to preoperative scores. The RPP+ group comprised 60 patients (69.6 years), while the RPP- group comprised 148 patients (67.2 years). In the RPP+ group, the lumbar function in the JOABPEQ and general health in the SF-36 showed limited postoperative enhancement. The pace of improvement in the role-emotional, role-physical, social functioning, vitality, and mental health scores was slower in the RPP+ group compared to the RPP- group. CONCLUSIONS: In the current study, we found that the presence of residual pain at six months postoperatively affected QOL improvement up to 24 months after surgery. Lingering postoperative pain substantially impacted functional incapacity, social engagement, and psychological well-being. Notably, the lumbar function in the JOABPEQ and general health in the SF-36 showed distinct progression patterns in the RPP+ group.

Development and initial validation of parent and child versions of the Juvenile Arthritis Disease Activity Score.

OBJECTIVE: To develop parent- and child-centered versions of the Juvenile Arthritis Disease Activity Score (JADAS) and to provide preliminary evidence of their validity. METHODS: Validation analyses were conducted on two large multinational datasets of patients with juvenile idiopathic arthritis (JIA) and included assessment of construct validity, internal consistency and structure, discriminative validity, responsiveness to change, and predictive validity. RESULTS: The parJADAS and patJADAS include four parent/patient-reported outcomes, each measured on a 0-10 scale: assessment of overall disease activity; rating of pain intensity; assessment of activity of joint disease; duration of morning stiffness. Both scores are calculated as the simple linear sum of the scores of their 4 components, which yields for both of them a global score of 0-40. The parJADAS and patJADAS demonstrated good construct validity, yielding high correlations with other JIA composite disease activity measures and moderate correlations with physician global rating and joint counts. Internal consistency was satisfactory, with Cronbach' s alpha > 0.80, and exploratory factor analysis showed that both indices are monodimensional. Both instruments discriminated well between different disease states, with discriminative ability being not affected by the presence of damage, proved able to predict important disease outcomes, and showed fair responsiveness to clinically important change, with standardized response mean of 0.71. CONCLUSION: Both parJADAS and patJADAS were found to possess good measurement properties and to serve as surrogate of physicians' evaluations. Regular home completion of the two instruments through digital technologies offers a suitable and pragmatic approach to deliver remote symptom monitoring and telehealth.

The efficacy of simple oral nutritional supplements versus usual care in postoperative patients with gastric cancer: study protocol for a multicenter, open-label, parallel, randomized controlled trial.

BACKGROUND: Body weight loss (BWL) after gastrectomy impact on the short- and long-term outcomes. Oral nutritional supplement (ONS) has potential to prevent BWL in patients after gastrectomy. However, there is no consistent evidence supporting the beneficial effects of ONS on BWL, muscle strength and health-related quality of life (HRQoL). This study aimed to evaluate the effects of ONS formulated primarily with carbohydrate and protein on BWL, muscle strength, and HRQoL. METHODS: This will be a multicenter, open-label, parallel, randomized controlled trial in patients with gastric cancer who will undergo gastrectomy. A total of 120 patients who will undergo gastrectomy will be randomly assigned to the ONS group or usual care (control) group in a 1:1 ratio. The stratification factors will be the clinical stage (I or ≥ II) and surgical procedures (total gastrectomy or other procedure). In the ONS group, the patients will receive 400 kcal (400 ml)/day of ONS from postoperative day 5 to 7, and the intervention will continue postoperatively for 8 weeks. The control group patients will be given a regular diet. The primary outcome will be the percentage of BWL (%BWL) from baseline to 8 weeks postoperatively. The secondary outcomes will be muscle strength (handgrip strength), HRQoL (EORTC QLQ-C30, QLQ-OG25, EQ-5D-5L), nutritional status (hemoglobin, lymphocyte count, albumin), and dietary intake. All analyses will be performed on an intention-to-treat basis. DISCUSSION: This study will provide evidence showing whether or not ONS with simple nutritional ingredients can improve patient adherence and HRQoL by reducing BWL after gastrectomy. If supported by the study results, nutritional support with simple nutrients will be recommended to patients after gastrectomy for gastric cancer. TRIAL REGISTRATION: jRCTs051230012; Japan Registry of Clinical Trails. Registered on Apr. 13, 2023.

Disease-specific health-related quality of life trajectories up to 15 years after curative treatment for esophageal cancer-a prospective cohort study.

BACKGROUND: The presence of distinct long-term disease-specific HRQL trajectories after curative treatment for esophageal cancer and factors associated with such trajectories are unclear. MATERIALS AND METHODS: This population-based and longitudinal cohort study included 425 esophageal cancer patients who underwent curative treatment, including esophagectomy, in Sweden in 2001-2005 and were followed up until 2020, that is, 15-year follow-up. The outcomes were 10 disease-specific HRQL symptoms, measured by the well-validated EORTC QLQ-OES18 questionnaire at 6 months (n = 402 patients), and 3 (n = 178), 5 (n = 141), 10 (n = 92), and 15 years (n = 52) after treatment. HRQL symptoms were examined for distinct trajectories by growth mixture models. Weighted logistic regression models provided odds ratios (OR) with 95% confidence intervals (95% CI) for nine factors in relation to HRQL trajectories: age, sex, education, proxy baseline HRQL, comorbidity, tumor histology, chemo(radio)therapy, pathological tumor stage, and postoperative complications. RESULTS: Distinct HRQL trajectories were identified for each of the 10 disease-specific symptoms. HRQL trajectories with more symptoms tended to persist or alleviate over time, while trajectories with fewer symptoms were more stable. Eating difficulty had three trajectories: persistently less, persistently moderate, and persistently more symptoms. The OR of having a persistently more eating difficulty trajectory was decreased for adenocarcinoma histology (OR = 0.44, 95% CI 0.21-0.95), and increased for pathological tumor stage III-IV (OR = 2.19, 95% CI 0.99-4.82) and 30-day postoperative complications (OR = 2.54, 95% CI 1.26-5.12). CONCLUSION: Distinct trajectories with long-term persistent or deteriorating disease-specific HRQL symptoms were identified after esophageal cancer treatment. Tumor histology, tumor stage, and postoperative complications may facilitate detection of high-risk patients for unwanted trajectories.

U-PRO-CRM: designing patient-centred dose-finding trials with patient-reported outcomes.

BACKGROUND: Determining the maximum tolerated dose (MTD) remains the primary objective for the majority of dose-finding oncology trials. Whilst MTD determination often relies upon clinicians to identify dose-limiting toxicities (DLTs) experienced by patients during the trial, research suggests that clinicians may underreport patient's adverse events. Therefore, contemporary practice may be exposed to recommending intolerable doses to patients for further investigation in subsequent trials. There is increasing interest in patients self-assessing their own symptoms using patient-reported outcomes (PROs) in dose-finding trials. DESIGN: We present Utility-PRO-Continual Reassessment Method (U-PRO-CRM), a novel trial design which simultaneously uses clinician-rated and patient-rated DLTs (Clinician-DLTs and Patient-DLTs, respectively) to make dose (de-)escalation decisions and to recommend an MTD. U-PRO-CRM contains the published PRO-CRM as a special case and provides greater flexibility to trade-off the rate of Patient-DLTs and Clinician-DLTs to find an optimal dose. We present simulation results for U-PRO-CRM. RESULTS: For specified trade-offs between Clinician-DLT and Patient-DLT rate, U-PRO-CRM outperforms the PRO-CRM design by identifying the true MTD more often. In the special case where U-PRO-CRM generalises to PRO-CRM, U-PRO-CRM performs as well as its published counterpart. U-PRO-CRM minimises the number of patients overdosed whilst maintaining a similar proportion of patients allocated to the true MTD. CONCLUSIONS: By using a utility-based dose selection approach, U-PRO-CRM offers the flexibility to define a trade-off between the risk of patient-rated and clinician-rated DLTs for an optimal dose. Patient-centric dose-finding strategies, which integrate PROs, are poised to assume an ever more pivotal role in significantly advancing our understanding of treatment tolerability. This bears significant implications in shaping the future landscape of early-phase trials.