Meaningful score changes for SF-36v2, FACIT-fatigue, and RASIQ in rheumatoid arthritis.

BACKGROUND: Interpretation thresholds for patient-reported outcome (PRO) scores are of crucial importance, particularly when interpreting treatment benefit. This study was designed to determine the within-patient meaningful improvement (WPMI) thresholds for the Short-Form 36 Health Survey version 2 (SF-36v2), the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and the novel Rheumatoid Arthritis Symptoms and Impact Questionnaire (RASIQ) among patients with rheumatoid arthritis (RA). METHODS: In this post-hoc analysis, anchor-based and supportive distribution-based methods were used to derive WPMI based on blinded data from all treatment arms in two Phase 2 RA trials with otilimab. Patient's Global Assessment of Disease Activity (PtGA) was the general anchor for all SF-36v2 scales. SF-36 Patient's Global Impression of Status (PGIS), PtGA, and VT03 (an SF-36v2 item) were used as anchors for FACIT-Fatigue. SF-36 PGIS, PtGA, and Patient's Assessment of Arthritis Pain (PAIN) were anchors for RASIQ. Mean change was calculated for the anchor category associated with minimal meaningful improvement from baseline to Week 24 for SF-36v2 and FACIT-Fatigue, and to Week 12 for RASIQ. Sensitivity and specificity were used to evaluate the accuracy of estimated WPMI values. RESULTS: For the SF-36v2 physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health domains, anchor-based estimates of WPMI based on 0-100 scores were 24.5, 24.5, 25.4, 13.6, 21.5, 20.5, 16.9, and 14.3, respectively. Anchor-based WPMI estimates were 9.7 for the Physical Component Summary score and 7.6 for the Mental Component Summary score (using norm-based T-score metric). For FACIT-Fatigue (range 0-52), WPMI estimates ranged from 9.7 to 11.3 points. For RASIQ (range 0-100), anchor-based WPMI was determined as a change between -32.7 and -21.7 points for the Joint Pain scale, -26.7 to -23.7 for the Joint Stiffness scale, and -21.1 to -17.4 for the Impact scale. CONCLUSIONS: This study derived WPMI thresholds for SF-36v2, FACIT-Fatigue, and RASIQ among patients with RA, using multiple anchors. Derivation of WPMI thresholds for these PRO instruments will enable their broader use in evaluating and interpreting treatment benefit in future RA studies.

When assessing medical treatments in clinical trials, it is important to understand whether the treatment improves symptoms or impacts of a disease to an extent which is meaningful for patients. Patients are often asked to complete questionnaires about their symptoms throughout clinical trials to measure if and how symptoms change. Questionnaire responses are used to calculate a score that is compared before and after treatment. This study was designed to investigate how much scores in three questionnaires (SF-36v2, FACIT-Fatigue, and RASIQ) changed for patients with rheumatoid arthritis who reported experiencing meaningful symptom improvement based on data from two clinical trials. As the RASIQ is a new questionnaire that was designed specifically for rheumatoid arthritis, this research is particularly important for interpretation of RASIQ results.

Association Between Hematologic Response and Change in Health-Related Quality of Life Among Patients with Light-Chain (AL) Amyloidosis.

Purpose: The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis. Patients and Methods: Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients' most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation). Results: For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response. Conclusion: Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic response does not necessarily mean that a patient will experience increased or stable HRQoL. When defining treatment success, it is important to recognize that clinical markers such as hematologic response may not fully encapsulate the patient experience.

Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group.

Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public-private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2® Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use-including specific research objectives, trial population, and investigational product under study-should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.

Quality of Life in the Management of Home Parenteral Nutrition.

INTRODUCTION: Home parenteral nutrition (HPN) is a rare but challenging therapy for patients with mostly severe underlying diseases. We aimed to investigate patient-reported health-related quality of life (QOL) of patients receiving HPN and its development over time in particular. METHODS: We assessed QOL of HPN patients in a prospective multicenter observational study (SWISSHPN II study). We designed a questionnaire to record symptoms and negative impacts of HPN and completed the validated Optum® SF-36v2® Health Survey with the patients. RESULTS: Seventy patients (50% women) on HPN were included. HPN commonly affected feelings of dependency (n = 49, 70%), traveling/leaving home (n = 37, 53%), attending cultural and social events (n = 25, 36%), and sleep (n = 22, 31%). Most frequently reported symptoms were diarrhea (n = 30, 43%), polyuria (n = 28, 40%), nausea/emesis (n = 27, 39%), dysgeusia (n = 23, 33%), and cramps (n = 20, 29%). At baseline, mean (standard deviation) SF-36v2® physical and mental health component summary scores (PCS and MCS) were 45 (20) and 57 (19), respectively, and there was a trend toward improvement in PCS over the study period, while MCS remained stable. Satisfaction with health care professionals involved in HPN care was high. CONCLUSION: QOL is a crucial and decisive aspect of HPN patient care. Symptoms related to the underlying disease and PN are frequent. Impaired social life and an ambivalent attitude toward the life-saving therapy are major concerns for these patients and should be addressed in their care.

Effect of once-weekly subcutaneous semaglutide 2.4 mg on weight- and health-related quality of life in an East Asian population: Patient-reported outcomes from the STEP 6 trial.

We assessed the effect of semaglutide 2.4 and 1.7 mg versus placebo on weight-related quality of life (WRQOL) and health-related quality of life (HRQOL) in the STEP 6 trial. Adults from East Asia (body mass index [BMI] ≥27.0 kg/m2 with ≥2 weight-related comorbidities, or ≥35.0 kg/m2 with ≥1 weight-related comorbidity) were randomized 4:1:2:1 to once-weekly subcutaneous semaglutide 2.4 mg or placebo, or semaglutide 1.7 mg or placebo, plus lifestyle intervention for 68 weeks. WRQOL and HRQOL were assessed from baseline to Week 68 using the Impact of Weight on Quality of Life-Lite Clinical Trials Version (IWQOL-Lite-CT) and the 36-Item-Short-Form-Survey-version-2.0 acute (SF-36v2), with changes in scores by categories of baseline BMI (

Content validation of the SF-36v2® Health Survey Acute for use in hypoparathyroidism.

PURPOSE: The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults diagnosed with chronic hypoparathyroidism (HP) to assess the content validity of the SF-36v2® Health Survey Acute (SF-36v2) measure in this population. METHODS: CD interviews were conducted with adults with HP in the United States (US). Interviews were conducted by a trained moderator using a semi-structured interview guide, employing a think-aloud method in conjunction with verbal probing. Participants were asked whether each item was understandable, relevant, important, and sensitive to change in relation to HP. Additionally, comprehension of instructions, response options, and the appropriateness of a 1-week recall period was assessed. RESULTS: Sixteen adults with HP participated in individual CD telephone interviews. All items in the SF-36v2 were reported to be understood, relevant, important, and sensitive to change by at least half, and in most cases, by a strong majority of study participants. Most of the study sample confirmed comprehension of the instructions and the entire sample understood all response options. CONCLUSION: The study findings show that the items in the SF-36v2® are applicable to adults with HP. The overall high levels of endorsement of items provide strong evidence of the measure's content validity for this population. The SF-36v2 is therefore recommended for usage in clinical trials examining adults with HP, although it is recommended that this generic measure be supplemented with disease-specific instruments such as the recently developed Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom) and Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact) measures.

Long-Term Effect on Health-Related Quality of Life in Patients With COVID-19 Requiring Hospitalization Compared to Non-hospitalized COVID-19 Patients and Healthy Controls.

Background In this study, we aimed to evaluate the health-related quality of life (HRQL) in patients with severe coronavirus disease 2019 (COVID-19) six months after their hospitalization and compare it to that of non-hospitalized patients with mild COVID-19 and healthy controls. Methodology Participants were enrolled between September 2021 and April 2022 and included hospitalized COVID-19 patients at General Hospital of Athens "Hippocration" who had been discharged at least six months prior to enrollment, non-hospitalized patients with COVID-19, and healthy controls. Collected data included demographics, disease severity, medication history, and comorbidities. Participants completed a EuroQol 5 Dimensions 5Levels (EQ5D5L), a Short Form 36 version 2 (SF36v2), a Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and a Post-COVID-19 Functional Status Scale (PCFSS) regarding HRQL before and six months after infection with severe acute respiratory syndrome coronavirus 2. In the case of healthy controls, two sets of questionnaires were completed at least six months apart. Statistical analysis was performed using the SPSS version 25 software (IBM Corp., Armonk, NY, USA). Results A total of 151 participants were enrolled. Hospitalized patients with COVID-19 demonstrated a statistically significant deterioration in most parameters of SF36v2 as well as both parameters of the EQ5D5L and FACIT-F questionnaires. Hospitalized patients exhibited worse results in SF36v2 and EQ5D5L when compared to both healthy controls as well as those with mild COVID-19 (p < 0.05). Hospitalized women, in particular, were shown to fare worse than other women in parameters associated with both mental/psychological and physical health (p < 0.05). Hospitalized patients between 41 and 60 years old demonstrated a statistically significant drop in the scores of all three main questionnaires compared to their previous health status (p < 0.05). Hospitalized patients between 61 and 80 years old exhibited a similar trend, but statistical significance was achieved in fewer parameters. HRQL decline was greater in both age groups compared to that of healthy and milder disease counterparts. There was a significant correlation between the results from the three main questionnaires. Similarly, PCFS scale values were shown to correlate with disease severity (hospitalization or not) and age. Conclusions HRQL remained noticeably impacted six months after hospitalization due to COVID-19. The physical and mental/psychological stress of severe COVID-19 translated into lasting health deterioration, especially for women and those aged 41-60 years old. The use of questionnaires, such as those implemented in this study, might help in the early detection of patients who could benefit from rehabilitation programs. Psychological, as well as physical and social, support is crucial to alleviate the burden of post-COVID-19 symptomatology and expedite the recovery of this group of patients.

Validation of the Italian version of wisconsin stone quality of life (WISQOL): a prospective Italian multicenter study.

Urolithiasis is a worldwide spread condition that affects patients' Health-Related Quality of Life (HRQOL), which measurement is an important tool for routine clinical and research practice. Disease-specific HRQOL measures demonstrated to perform better in assessing the effects of specific conditions. A disease-specific questionnaire for kidney stones, the WISQOL, has been validated in different languages, but an Italian version is still missing. Our aim is to produce and validate the Italian version of WISQOL (IT-WISQOL). Patients undergoing any elective treatment for upper urinary tract stones were enrolled. A multi-step process with forward- and back-translation was used to translate WISQOL into Italian. Patients were evaluated within 15 days pre-operatively and then at 30-, 90 days post-operatively and administered both IT-WISQOL and SF-36v2. Post-operative data such as 30 days postoperative complications, late stone-related events, successful status, and stone complexity were collected. Cronbach's α was used to evaluate the internal consistency of IT-WISQOL, while Spearman's rho was used for item and inter-domain correlations and IT-WISQOL with SF-36v2 correlation. We found excellent internal consistency across all domains (α ≥ 0.88), particularly when the total score is considered (α = 0.960). Test-retest reliability showed excellent results for the total questionnaire (Pearson correlation value: 0.85). The Inter-domain association ranged from 0.497 to 0.786. Convergent validity was confirmed by a good correlation with subdomains of the SF-36v2 measures. IT-WISQOL is a reliable tool to measure HRQOL in stone patients. It shows analog characteristics if compared to English WISQOL.

Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study.

The use of available treatments for Fabry disease (FD) (including enzyme replacement therapy [ERT]) may be restricted by their limited symptom improvement and mode of administration. Lucerastat is currently being investigated in the MODIFY study as oral substrate reduction therapy for the treatment of FD. By reducing the net globotriaosylceramide (Gb3) load in tissues, lucerastat has disease-modifying potential to improve symptoms and delay disease progression. MODIFY is a multicenter, double-blind, randomized, placebo-controlled, parallel-group Phase 3 study (ClinicalTrial.gov: NCT03425539); here we present the rationale and design of this study. Eligible adults with a genetically confirmed diagnosis of FD and FD-specific neuropathic pain entered screening. Patients were randomized (2:1) to receive either oral lucerastat twice daily or placebo for 6 months; treatment allocation was stratified according to sex and ERT treatment status. The main objectives of MODIFY are to assess the effects of lucerastat on neuropathic pain, gastrointestinal (GI) symptoms, FD biomarkers, and determine its safety and tolerability. Neuropathic pain and GI symptoms are key features of FD that have a significant impact on quality of life. Despite various tools available to assess pain and GI symptoms, there are currently limited tools available to assess neuropathic and GI symptoms in FD, validated according to health authority guidelines. Based on FDA recommendations, we undertook a patient-reported outcome (PRO) validation study, using a novel eDiary-based PRO tool to assess the validity of evaluating neuropathic pain as a primary efficacy endpoint in MODIFY. Results from the PRO validation study are included. To date, MODIFY is the largest Phase 3 clinical study conducted in patients with FD. Enrollment to MODIFY is now complete, with 118 patients randomized. Results will be presented in a separate publication. Long-term effects of lucerastat are being assessed in the ongoing open-label extension study (NCT03737214).

Trastornos mentales y calidad de vida relacionada con la salud en profesionales de Unidades de Cuidados Intensivos / Mental disorders and health-related quality of life in Intensive Care Units professionals

Resumen Los profesionales de la salud de las Unidades de Cuidados Intensivos (UCI) enfrentan situaciones de sufrimiento humano, competitividad y demanda, que podrían perjudicar su calidad de vida y su salud mental. El objetivo del estudio fue describir los trastornos mentales comunes y la calidad de vida relacionada con la salud en profesionales de la salud de la UCI en Salvador, Brasil. Este estudio transversal con 195 profesionales utilizó un cuestionario sociodemográfico y laboral, el Self-Reporting Questionnaire (SRQ-20) de trastornos mentales comunes y el 36-Item Short Form Health Survey (SF-36v2) de calidad de vida relacionada con la salud. El 29.7 % de los profesionales presentaron trastornos mentales comunes, especialmente entre profesionales de enfermería (RP = 2.28; IC 1.19-4.39; p = .007). La calidad de vida relacionada con la salud para todos los profesionales estuvo disminuida, principalmente en función social (44.25 ± 10.15) y rol emocional (45.86 ± 10.58). El SRQ-20 correlacionó fuertemente con los dominios dolor corporal (r = -.502), salud general (r = -.526), vitalidad (r = -.656), función social (r = -.608), salud mental (r = -.631) y el componente de salud mental (r = -.638) del SF-36v2 (p < .01). Los profesionales con trastornos mentales comunes mostraron una calidad de vida relacionada con la salud más deteriorada, esencialmente en dominios del componente de salud mental, y refirieron también dolor corporal. Es necesario discutir e implementar estrategias de evaluación, prevención y promoción de la salud mental entre los profesionales de las UCI para que sean consideradas dentro de las políticas de salud laboral.

Development of the SF-6Dv2 health utility survey: comprehensibility and patient preference.

BACKGROUND: The SF-6Dv2 classification system assesses health states in six domains-physical functioning, role function, bodily pain, vitality, social functioning, and mental health. Scores have previously been derived from the SF-36v2® Health Survey. We aimed to develop a six-item stand-alone SF-6Dv2 Health Utility Survey (SF-6Dv2 HUS) and evaluate its comprehensibility. METHODS: Two forms of a stand-alone SF-6Dv2 HUS were developed for evaluation. Form A had 6 questions with 5-6 response choices, while Form B used 6 headings and 5-6 statements describing the health levels within each domain. The two forms were evaluated by 40 participants, recruited from the general population. Participants were randomized to debrief one form of the stand-alone SF-6Dv2 HUS during a 75-min interview, using think-aloud techniques followed by an interviewer-led detailed review. Participants then reviewed the other form of SF-6Dv2 and determined which they preferred. Any issues or confusion with items was recorded, as was as overall preference. Data were analyzed using Microsoft Excel and NVivo Software (v12). RESULTS: Participants were able to easily complete both forms. Participant feedback supported the comprehensibility of the SF-6Dv2 HUS. When comparing forms, 25/40 participants preferred Form A, finding it clearer and easier to answer when presented in question/response format. The numbered questions and underlining of key words in Form A fostered quick and easy comprehension and completion of the survey. However, despite an overall preference for Form A, almost half of participants (n = 19) preferred the physical functioning item in Form B, with more descriptive response choices. CONCLUSION: The results support using Form A, with modifications to the physical functioning item, as the stand-alone SF-6Dv2 HUS. The stand-alone SF-6Dv2 HUS is brief, easy to administer, and comprehensible to the general population.

Patient-reported health-related quality of life from a randomized, placebo-controlled phase 2 trial of zuranolone in adults with major depressive disorder.

BACKGROUND: Major depressive disorder (MDD), a disabling, potentially life-threatening condition, negatively affects health-related quality of life (HRQoL). This secondary analysis aimed to understand the impact of the neuroactive steroid zuranolone on HRQoL using the Short Form-36v2 Health Survey (SF-36v2). METHODS: Adult patients with MDD and 17-item Hamilton Rating Scale for Depression total score ≥22 were randomized 1:1 to receive zuranolone 30 mg or placebo for 2 weeks, with 4 weeks follow-up. SF-36v2 scores were assessed at Day 15 across 8 domains (Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health) and 2 summary scores (Physical and Mental Component), using a mixed-effects model for repeated measures. Correlations between SF-36v2 scores and clinician-reported efficacy endpoints were assessed using Pearson's correlation. RESULTS: Eighty-nine patients were treated with zuranolone 30 mg (n = 45) or placebo (n = 44). In zuranolone-treated patients, HRQoL improved across all SF-36v2 domains and summary scores at Day 15. Improvements exceeding established minimally important difference thresholds were observed in Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health scores. Improvements in General Health, Vitality, Mental Health, and Mental Component Summary were statistically significant versus placebo (p ≤ 0.025). Clinician-rated endpoints negatively correlated with SF-36v2 scores. LIMITATIONS: The small unipolar depression sample may not be representative of all US MDD patients. HRQoL measures could be impacted by factors unrelated to depression. CONCLUSIONS: Zuranolone-treated patients reported rapid and significant improvements in HRQoL versus placebo at Day 15. HRQoL improvements correlated with improvements in clinician-rated assessments. TRIAL REGISTRATION: clinicaltrials.gov:NCT03000530; https://clinicaltrials.gov/ct2/show/NCT03000530.

Positive Effects of an Online Workplace Exercise Intervention during the COVID-19 Pandemic on Quality of Life Perception in Computer Workers: A Quasi-Experimental Study Design.

Computer workers' sedentary work, together with less active lifestyles, aggravated by the COVID-19 pandemic, represents a high risk for many chronic diseases, leading to a decrease in health-related quality of life (QoL). Workplace exercises consist of a set of physical exercises, implemented during work breaks, that have multiple benefits for workers' health. AIM: To assess the impact of online workplace exercises on computer workers' perception of quality of life. METHODS: Quasi-experimental study with two groups: a control group (n = 26) and an intervention group (n = 13). The inclusion criteria were that participants must be aged between 18 and 65 years old and the exclusion criteria included diagnosis of non-work-related medical conditions. The interventions consisted of workplace exercises, which were applied for 17 consecutive weeks, each session lasting 15 min, three times a week. The exercise programme, performed online and guided by a physiotherapist, consisted of mobility exercises, flexibility and strength exercises, with the help of a TheraBand® for elastic resistance. The control group were not subjected to any intervention. A socio-demographic questionnaire and the Health Survey Questionnaire (SF-36v2) were used in two assessment stages (M0-baseline and M1-final of intervention). A mixed ANOVA with interaction time*group was used to evaluate the effect of the exercise programme. RESULTS: A good perception of the QoL was obtained in both stages. The exercise programme had a positive effect in the domains of Pain (ptime*group = 0.012, η2p = 0.158), Physical Function (ptime*group = 0.078, η2p = 0.082), Physical Performance (ptime*group = 0.052, η2p = 0.098), and Emotional Performance (ptime*group = 0.128, η2p = 0.061). CONCLUSION: After 17 weeks of workplace exercises, it became clear that the intervention group positively increased their QoL perception, with this improvement being significant in the Pain domain, which resulted in an improvement in their health condition. Therefore, further studies are needed to determine the optimal exercise for CWs, with detailed exercise types, different intensities and focused on various health conditions.

Psychometric study of the SF-36v2 in hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE).

BACKGROUND: The generic 36-item Short-Form Health Survey (SF-36v2) has been used to assess health related quality of life in adult patients with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) even though it has not yet been validated for use in this specific disease. OBJECTIVE: This study aims to validate the SF-36v2 for use in adult patients with C1-INH-HAE. RESULTS: There was a very low item non-response rate (1-3.4%), with a high ceiling effect in 25/35 items and a low floor effect in 3/35 items. A moderate ceiling effect was observed in 5/8 dimensions of the SF-36v2, whereas no floor effect was noticed in any of the dimensions. Internal consistency was good to excellent with Cronbach's alpha coefficient ranging between 0.82 and 0.93 for the different dimensions. Construct validity was good: seven out of the 8 hypotheses defined on clinical criteria were confirmed, discriminant validity assessment showed significant differences among patients with different C1-INH-HAE severity, convergent validity showed a good correlation among the physical and mental component summaries of the SF-36v2 and the HAE-QoL total score (0.45 and 0.64 respectively, P < 0.001). Test-retest reliability was high with intraclass correlation coefficient varying from 0.758 to 0.962. The minimal clinically important difference was calculated by distribution methods and small differences in the domain scores and in the component summaries scores were shown to be meaningful. CONCLUSIONS: The psychometric properties of the SF-36v2 show it can be a useful tool to assess HRQoL in adult patients with C1-INH-HAE, although with some content validity limitation. METHODS: The psychometric properties of the SF-36v2 were evaluated in an international setting based on responses from 290 adult C1-INH-HAE patients in 11 countries.

Health-Related Quality of Life and Risk Factors among Chinese Women in Japan Following the COVID-19 Outbreak.

The COVID-19 pandemic has significantly affected individuals' physical and mental health, including that of immigrant women. This study aimed to evaluate the health-related quality of life (HRQoL), identify the demographic factors and awareness of the COVID-19 pandemic contributing to physical and mental health, and examine the risk factors associated with poor physical and mental health of Chinese women in Japan following the COVID-19 pandemic outbreak. Using an electronic questionnaire survey, we collected data including items on HRQoL, awareness of the COVID-19 pandemic, and demographic factors. One hundred and ninety-three participants were analyzed. Approximately 98.9% of them thought that COVID-19 affected their daily lives, and 97.4% had COVID-19 concerns. Married status (OR = 2.88, 95%CI [1.07, 7.72], p = 0.036), high concerns (OR = 3.99, 95%CI [1.46, 10.94], p = 0.007), and no concerns (OR = 8.75, 95%CI [1.17, 65.52], p = 0.035) about the COVID-19 pandemic were significantly associated with poor physical health. Unmarried status (OR = 2.83, 95%CI [1.20, 6.70], p = 0.018) and high COVID-19 concerns (OR = 2.17, 95%CI [1.04, 4.56], p = 0.040) were significantly associated with poor mental health. It is necessary to provide effective social support for Chinese women in Japan to improve their well-being, especially in terms of mental health.

Long-term effect of aripiprazole lauroxil on health-related quality of life in patients with schizophrenia.

BACKGROUND: This post hoc analysis of clinical trial data evaluated long-term, self-reported mental and physical health-related quality of life (HRQoL) scores in schizophrenia patients receiving aripiprazole lauroxil (AL), an atypical long-acting injectable (LAI) antipsychotic approved for the treatment of schizophrenia in adults. METHODS: The study population included 291 stable schizophrenia outpatients enrolled in 2 consecutive long-term safety studies of AL given every 4 weeks for up to 124 weeks. HRQoL was measured using the SF-36v2® Health Survey (SF-36v2) over the course of the follow-up. The primary outcome was change in SF-36v2 mental component summary (MCS) and physical component summary (PCS) scores from baseline to 124 weeks. To contextualize these scores, descriptive analyses were conducted to compare the scores with available scores for the general population as well as for other populations with chronic medical (ie, hypertension and type 2 diabetes) or psychiatric (ie, depression) conditions. RESULTS: Results from this post hoc analysis indicated that the mean MCS score for patients continuing AL improved significantly from baseline over 124 weeks (P < .05, all timepoints), while mean PCS score showed little change over 124 weeks. At baseline, patients had lower (worse) MCS scores than the normed general population, but by week 124, patients had MCS scores comparable to those in the general population. This pattern of change was not observed with PCS scores. Comparison of study MCS scores with those associated with other diseases showed that this schizophrenia cohort had lower scores than those with chronic medical conditions but higher scores than those with depression. PCS scores were higher in the study population than published scores for all reference populations at baseline and week 124. CONCLUSIONS: In this post hoc analysis, outpatients with schizophrenia who continued the LAI antipsychotic AL showed gradual and sustained improvement in self-reported mental HRQoL over several years of follow-up, whereas self-reported physical HRQoL did not change. By the end of follow-up, mental health scores of study patients with schizophrenia were comparable to those of the general population and better than those of patients with depression. TRIAL REGISTRATION: ClinicalTrials.gov (NCT01626456 [trial registration date: June 15, 2012] and NCT01895452 [trial registration date: July 5, 2013]).

Achieving clinical response in postpartum depression leads to improvement in health-related quality of life.

OBJECTIVE: To evaluate the health-related quality of life (HRQoL) burden associated with postpartum depression (PPD), determine the extent to which clinical response impacts HRQoL, and estimate the impact of PPD and clinical response on healthcare resource utilization (HRU) and productivity. METHODS: Patient data (n = 127) from two multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trials evaluating the safety and efficacy of brexanolone injection in adults with PPD were employed for these posthoc analyses. HRQoL and health utility was assessed with the SF-36-v2 Health Survey (SF-36v2) acute version. The 17-item Hamilton Rating Scale for Depression (HAMD-17) total score was used to identify clinical response (≥50% reduction in HAMD-17 total score). Baseline HRQoL burden was assessed by comparison to age- and gender-adjusted population normative data from the 2009 QualityMetric PRO Norming study. The impact of clinical response was evaluated by comparing day 7 and day 30 SF-36v2 scores between clinical responders and non-responders. Interpretations of the meaningfulness of clinical response were indirectly estimated via 2017 National Health and Wellness Survey data linking SF-36v2 mental component summary (MCS) scores to (HRU) and productivity. RESULTS: Baseline HRQoL of patients with PPD was significantly below normative values. Day 7 and day 30 clinical response were associated with large and statistically significant improvements in HRQoL, greater likelihood of meeting SF-36v2 responder definitions, and reduced impairment. MCS levels corresponding to those observed in clinical responders were linked to lower HRU and productivity loss relative to non-responders. CONCLUSIONS: PPD places a substantial burden on HRQoL. Achievement of rapid clinical response (at day 7) and clinical response sustained several weeks following the end of treatment (day 30) led to significant improvement in HRQoL, suggesting the importance of identifying women with PPD and providing effective treatment options.

Constipation and the Quality of Life in Conservatively Treated Chronic Kidney Disease Patients: A Cross-sectional Study.

Background: Constipation is a common gastrointestinal disorder that in general population is associated with worse health-related quality of life (HRQoL). The epidemiology of constipation has not been reliably determined in conservatively-treated CKD patients. We aimed to determine the prevalence of constipation and constipation-related symptoms in conservatively-treated CKD patients, to find factors associated with their altered prevalence ratio (PR), and to verify the associations between constipation and HRQoL. Methods: In this cross-sectional study, 111 conservatively-treated CKD outpatients fulfilled questionnaires that included questions addressing HRQoL (SF-36v2®), constipation-related symptoms (The Patient Assessment of Constipation-Symptoms questionnaire), the Bristol stool form scale (BSFS), Rome III criteria of functional constipation (FC), and frequency of bowel movement (BM). Results: Depending on the used definition, the prevalence of constipation was 6.6-28.9%. Diuretics and paracetamol were independently associated with increased PR of BSFS-diagnosed constipation (PR 2.86, 95% CI 1.28-6.37, P = 0.01) and FC (PR 2.67, 95% CI 1.07-6.64, P = 0.035), respectively. The most commonly reported symptoms were bloating (50.9%) and straining to pass a BM (42.7%). Abdominal discomfort (37.3%) was independently associated with worse scores in all analyzed HRQoL domains. In multiple regressions, FC and having <7 BM/week, but not BSFS-diagnosed constipation, were associated with lower scores in several HRQoL domains. Conclusions: Constipation and related symptoms are prevalent in CKD patients. FC and decreased frequency of defecation, but not BSFS-diagnosed constipation, are associated with worse assessment of HRQoL in conservatively-treated CKD patients.

Psychometric validation of a Saudi Arabian version of the sf-36v2 health survey and norm data for Saudi Arabia.

BACKGROUND: Adaptation of a patient-reported outcomes survey into a new language requires careful translation procedures as well as qualitative and quantitative psychometric testing. This study aimed to evaluate the basic psychometric properties of the new Saudi Arabian SF-36v2 and establish norm data for Saudi Arabia. METHODS: Translation and adaptation of the SF-36v2 used standard methodology. Psychometric validation included two stages: 1) A qualitative study (n = 100) explored the components of health and health-related quality of life considered important in Saudi Arabia and evaluated the content validity of the SF-36v2 in Saudi Arabia, and 2) A quantitative study (n = 6166) evaluated the basic psychometric properties of the Saudi SF-36v2 and established norm data for Saudi Arabia. Comparison with US general population data (n = 4040) evaluated differential item function (DIF) and cross-national differences. RESULTS: The qualitative study supported the content validity of the Saudi SF-36v2. Cognitive debriefing identified only few and minor problems. Psychometric analyses supported item convergence within scales and differentiation across scales of the SF-36v2. Scale level exploratory factor analyses did not support the typical distinction between physical health and mental health components. Internal consistency reliability was satisfactory for all scales except the social function scale (alpha = 0.67). Cross-national DIF was identified for 9 items. In the Saudi general population, the average vitality score was lower for women (- 2.71 points) compared to men. For men, older age groups scored lower on the physical function scale (- 3.31) and the physical health component (- 3.06). For women, older age groups scored lower on the role physical (- 3.72), bodily pain (- 3.66), and vitality (- 2.32) scales as well as the physical health component (- 3.52). Compared to the 2009 United States general population, and after adjusting for age, gender, and differential item function, persons in Saudi Arabia had lower average scores for the physical function (- 3.10), role physical (- 4.75), social function (- 4.23), role emotional (- 5.67), and mental health (- 4.82) scales, as well as the mental health component (- 4.57). CONCLUSION: This Saudi normative study of patient reported outcomes supported the validity and reliability of the new Saudi SF-36v2 and found cross-national differences with the USA.

Development and validation of risk stratification models for adult spinal deformity surgery.

OBJECTIVE: Adult spinal deformity (ASD) surgery has a high rate of major complications (MCs). Public information about adverse outcomes is currently limited to registry average estimates. The object of this study was to assess the incidence of adverse events after ASD surgery, and to develop and validate a prognostic tool for the time-to-event risk of MC, hospital readmission (RA), and unplanned reoperation (RO). METHODS: Two models per outcome, created with a random survival forest algorithm, were trained in an 80% random split and tested in the remaining 20%. Two independent prospective multicenter ASD databases, originating from the European continent and the United States, were queried, merged, and analyzed. ASD patients surgically treated by 57 surgeons at 23 sites in 5 countries in the period from 2008 to 2016 were included in the analysis. RESULTS: The final sample consisted of 1612 ASD patients: mean (standard deviation) age 56.7 (17.4) years, 76.6% women, 10.4 (4.3) fused vertebral levels, 55.1% of patients with pelvic fixation, 2047.9 observation-years. Kaplan-Meier estimates showed that 12.1% of patients had at least one MC at 10 days after surgery; 21.5%, at 90 days; and 36%, at 2 years. Discrimination, measured as the concordance statistic, was up to 71.7% (95% CI 68%-75%) in the development sample for the postoperative complications model. Surgical invasiveness, age, magnitude of deformity, and frailty were the strongest predictors of MCs. Individual cumulative risk estimates at 2 years ranged from 3.9% to 74.1% for MCs, from 3.17% to 44.2% for RAs, and from 2.67% to 51.9% for ROs. CONCLUSIONS: The creation of accurate prognostic models for the occurrence and timing of MCs, RAs, and ROs following ASD surgery is possible. The presented variability in patient risk profiles alongside the discrimination and calibration of the models highlights the potential benefits of obtaining time-to-event risk estimates for patients and clinicians.