Unmet needs with antipsychotic treatment in schizophrenia and bipolar I disorder: patient perspectives from qualitative focus groups.

BACKGROUND: Schizophrenia (SZ) and bipolar I disorder (BD-I) are chronic mental health disorders often treated with antipsychotic medications. This qualitative study sought to better understand disease burden and treatment experiences with oral antipsychotic medications in participants living with SZ or BD-I. METHODS: Six 90-min focus groups were conducted with participants diagnosed with SZ or BD-I. Trained moderators facilitated discussions using a semistructured guide. Participants described symptoms, impacts of disease, and experiences with oral antipsychotic medications, whether favourable or unfavourable. RESULTS: Among participants with SZ (n = 15; 3 groups, 5 per group), 53% were male and 33% were white, with a mean of 18.6 years since diagnosis. Of participants with BD-I (n = 24; 3 groups, 8 per group), 33% were male and 42% were white, with a mean of 13.0 years since diagnosis. Participants described numerous symptoms of their illnesses that impacted relationships and daily life, including effects on emotional health, the ability to work, and encounters with law enforcement. Previous antipsychotic medications were deemed effective by 14/15 (93%) participants with SZ and 12/16 (75%) participants with BD-I. Most participants with SZ (13/15; 87%) or with BD-I (16/24; 67%) reported discontinuing their antipsychotic medication at some point. Side effects were a common reason for discontinuing or switching medications for participants with SZ (8/15; 53%) and for those with BD-I (11/24; 46%). The most common side effects reported in both cohorts were weight gain, drowsiness, sexual problems, and neurologic symptoms. Side effects negatively affected quality of life, leading to serious health problems and issues with self-esteem. CONCLUSIONS: People living with SZ or BD-I cited a range of favourable and unfavourable experiences with oral antipsychotic medications. Most participants reported that their antipsychotics were effective at controlling their symptoms, but multiple side effects impacted their quality of life, caused additional serious health problems, and often led to discontinuation of or switching antipsychotics. Findings from this study contribute to a better understanding of patients' experiences with antipsychotics and highlight a need for new medications with favourable benefit/risk profiles.

Deciding Between SF-6Dv2 Health States: A Think-Aloud Study of Decision-Making Strategies Used in Discrete Choice Experiments.

OBJECTIVE: This study aimed to gain insight into decision-making strategies individuals used when evaluating pairs of SF-6Dv2 health states in discrete choice experiments (DCEs). METHODS: This qualitative, cross-sectional, noninterventional study asked participants to use a think-aloud approach to compare SF-6Dv2 health states in DCEs. Thematic analysis focused on comprehension and cognitive strategies used to compare health states and make decisions. RESULTS: Participants (N = 40) used 3 main strategies when completing DCEs: (1) trading, (2) reinterpretation, and (3) relying on previous experience. Trading was the most common strategy, used by everyone at least once, and involved prioritizing key attributes, such as preferring a health state with significant depression but no bodily pain. Reinterpretation was used by 17 participants and involved reconstructing health states by changing underlying assumptions (eg, rationalizing selecting a health state with significant pain because they could take pain medications). Finally, some (n = 13) relied on previous experience when making decisions on some choice tasks. Participants with experience dealing with pain, for instance, prioritized health states with the least impact in this dimension. CONCLUSIONS: Qualitatively evaluating the decision-making strategies used in DCEs allows researchers to evaluate whether the tasks and attributes are interpreted accurately. The findings from this study add to the understanding of the generation of SF-6Dv2 health utility weights and the validity of these weights (e.g., reinterpreting health states could undermine the validity of DCEs and utility weights), and the overall usefulness of the SF-6Dv2. The methodology described in this study can and should be carried forth in valuing other health utility measures, not just the SF-6Dv2.

Content validation of observer-reported sickle cell pain diaries (SCPD-CS and SCPD-CN): results from interviews with caregivers.

BACKGROUND: Patients with sickle cell disease (SCD) experience daily pain and acute episodes known as sickle cell pain crises (SCPCs). The Sickle Cell Pain Diary-Caregiver Report (SCPD-C) is an observer-reported diary for use by caregivers of children ages < 12 years with SCD. This study reports on the content validity of the SCPD-C. METHODS: The SCPD-C was developed based on a literature review, measurement expert input, and a patient advisory board including clinicians. Three rounds of interviews (including both concept elicitation and cognitive debriefing methodologies) were conducted with caregivers of children with SCD aged < 12 to evaluate the content validity of the SCPD-C. RESULTS: Across three rounds of interviews, caregivers confirmed concepts in the SCPD-C and described observed impacts that were important and were added. Overall, caregivers evaluated the SCPD-C as easy to understand, with some minor adaptations for clarity. Additionally, the diary was split into two versions based on the child's age and school enrollment status (SCPD-CS for school-aged and SCPD-CN for non-school age children). CONCLUSIONS: Caregivers provided valuable input that led to important additions and changes to the measures. The SCPD-CS and SCPD-CN are appropriate and fit-for-purpose observer-reported outcome measures of SCPC-related pain frequency and severity, and impacts on health-related quality of life.

The relationship between frequency and severity of vaso-occlusive crises and health-related quality of life and work productivity in adults with sickle cell disease.

PURPOSE: Patients with sickle cell disease (SCD) may experience sickle cell-related pain crises, also referred to as vaso-occlusive crises (VOCs), which are a substantial cause of morbidity and mortality. The study explored how VOC frequency and severity impacts health-related quality of life (HRQoL) and work productivity. METHODS: Three hundred and three adults with SCD who completed an online survey were included in the analysis. Patients answered questions regarding their experience with SCD and VOCs, and completed the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) and the Workplace Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP). Differences in ASCQ-Me and WPAI:SHP domains were assessed according to VOC frequency and severity. RESULTS: Nearly half of the patient sample (47.2%) experienced ≥ 4 VOCs in the past 12 months. The most commonly reported barriers to receiving care for SCD included discrimination by or trouble trusting healthcare professionals (39.6%, 33.3%, respectively), limited access to treatment centers (38.9%), and difficulty affording services (29.4%). Patients with more frequent VOCs reported greater impacts on emotion, social functioning, stiffness, sleep and pain, and greater absenteeism, overall productivity loss, and activity impairment than patients with less frequent VOCs (P < 0.05). Significant impacts on HRQoL and work productivity were also observed when stratifying by VOC severity (P < 0.05 for all ASCQ-Me and WPAI domains, except for presenteeism). CONCLUSIONS: Results from the survey indicated that patients with SCD who had more frequent or severe VOCs experienced deficits in multiple domains of HRQoL and work productivity. Future research should examine the longitudinal relationship between these outcomes.

Burden of hereditary transthyretin amyloidosis on quality of life.

INTRODUCTION: Hereditary transthyretin (hATTR) amyloidosis is a progressive, degenerative disease, with peripheral neuropathy, cardiomyopathy, and other clinical manifestations. In this study we examine the impact of hATTR amyloidosis on quality of life (QOL). METHODS: Neuropathy-specific QOL, measured with the Norfolk QOL-Diabetic Neuropathy questionnaire, was compared between patients with hATTR amyloidosis and patients with type 2 diabetes, whereas generic QOL, measured with the 36-item Short Form Health Survey version 2 (SF-36v2), was compared between patients with hATTR amyloidosis, the general population, and patients with chronic diseases. RESULTS: Neuropathy-specific QOL for patients with hATTR amyloidosis was nearly equivalent to that of patients with type 2 diabetes with diabetic neuropathy accompanied by a history of ulceration, gangrene, or amputation. Generic QOL was worse than that seen in the general population, with physical functioning worse than that for patients with multiple sclerosis and congestive heart failure. DISCUSSION: Patients with hATTR amyloidosis show significant burden on QOL, particularly in physical functioning. Muscle Nerve 60: 169-175, 2019.

Patient-reported treatment-related symptom burden for patients with advanced melanoma in Canada.

BACKGROUND: Little is known on the impact of emerging treatments for advanced melanoma (stages III and IV) on patients' functioning and well-being. The objective of this study was to describe the patient-reported treatment-related symptom (TRS) burden in advanced melanoma. METHOD: Twenty-nine in-depth, qualitative interviews were conducted among adult patients with advanced melanoma in Canada using a semi-structured interview method. Interviews were transcribed verbatim, and key concepts were identified using a grounded theory analytic approach. RESULTS: The 29 patients reported 13 unique treatment journeys involving the following drug therapy categories: cytotoxic chemotherapies, CTLA-4 inhibitors, BRAF or MEK inhibitors, and PD-1 inhibitors. Patients typically underwent multiple treatment episodes over time. Common TRSs included nausea, fatigue, diarrhea or constipation, and skin rashes. Patients described these as impacting their physical functioning, ability to perform activities of daily living, social functioning, and overall quality of life. CONCLUSION: Our findings provide a description of the patient's experience with treatment for advanced melanoma. Our sample included patients typically diagnosed in mid-life, facing an urgent sequence of medical procedures and a pharmacological treatment journey that was burdensome. There is a need for less toxic and more efficacious treatments earlier in the patient journey to alleviate the impact of advanced melanoma treatment on patients' health-related quality of life.

Adapting traditional content validation methods to fit purpose: an example with a novel video assessment and training materials in Duchenne muscular dystrophy (DMD).

PURPOSE: The Duchenne muscular dystrophy (DMD) Video Assessment requires a caregiver to record six videos of their ambulatory child performing physical activities using a smartphone application (app). Innovative assessments that combine a performance measure, technology, and an observer require new approaches to content validation studies. This study presents a novel approach to establish the feasibility and content validity of the Video Assessment and usability of the app. METHODS: Interviews used concept elicitation and an adapted cognitive debriefing approach. Interviews were conducted with 8 clinicians who reviewed training materials prior to the interview and provided feedback on the appropriateness and usefulness of the DMD Video Assessment and the relevance of the physical activities. Four caregivers reviewed training materials and used the app to record their child performing the physical activities prior to the interview. Researchers reviewed the recordings against a checklist to identify discrepancies in caregivers' understanding of the training materials prior to an interview. During the interview, caregivers commented on comprehension of the materials, appropriateness of the activities, and feasibility of recording the videos. RESULTS: Clinicians found the DMD Video Assessment and materials appropriate and useful for assessing disease progression and treatment response. Caregivers found the activities appropriate and the training materials and app easy to understand and use. Feedback resulted in changes to the training materials, but not the activities or videotaping procedure. CONCLUSIONS: Researchers used an innovative methodological approach that adapted traditional methods of content validation for the purpose of evaluating a technology-based performance measure in its totality. While future studies should be conducted with a larger, more diverse sample, these study findings add to our understanding of the content validity of the DMD Video Assessment.

Humanistic burden of disease for patients with advanced melanoma in Canada.

BACKGROUND: Metastatic melanoma is a highly aggressive cancer, often striking in the prime of life. This study provides new information directly from advanced melanoma (stage III and IV) patients on how their disease impacts their health-related quality of life (HRQL). METHODS: Twenty-nine in-depth, qualitative interviews were conducted with adult patients with advanced melanoma in Canada. A semi-structured interview guide was used. Interviews were transcribed verbatim and key concepts were identified using a grounded theory analytic approach. RESULTS: Many patients' journeys began with the startling diagnosis of an invasive disease and a vastly shortened life expectancy. By the time they reached an advanced stage of melanoma, these patients' overall functioning and quality of life had been greatly diminished by this quickly progressing cancer. The impact was described in terms of physical pain and disability, emotional distress, diminished interactions with friends and family, and burden on caregivers. CONCLUSION: Our findings provide evidence of signs, symptoms, and functional impacts of advanced melanoma. Signs and symptoms reported (physical, mental, and social) confirm and expand on those reported in the existing clinical literature. Primary care physicians should be better trained to identify melanomas early. Oncology care teams can improve on their current approaches for helping patients navigate treatment options, with information about ancillary services to mitigate disease impacts on HRQL, such as mental health and social supports, as well as employment or financial support services.

A meta-analytic review of measurement equivalence study findings of the SF-36® and SF-12® Health Surveys across electronic modes compared to paper administration.

PURPOSE: Patient-reported outcome (PRO) measures originally developed for paper administration are increasingly being administered electronically in clinical trials and other health research studies. Three published meta-analyses of measurement equivalence among paper and electronic modes aggregated findings across hundreds of PROs, but there has not been a similar meta-analysis that addresses a single PRO, partly because there are not enough published measurement equivalence studies using the same PRO. Because the SF-36(R) Health Survey (SF-36) is a widely used PRO, the aim of this study was to conduct a meta-analysis of measurement equivalence studies of this survey. METHODS: A literature search of several medical databases used search terms for variations of "SF-36" or "SF-12" and "equivalence" in the title or abstract of English language publications. The eight scale scores and two summary measures of the SF-36 and SF-12 were transformed to norm-based scores (NBS) using developer guidelines. A threshold of within ± 2 NBS points was set as the margin of equivalence. Comprehensive meta-analysis software was used. RESULTS: Twenty-five studies were included in the meta-analysis. Results indicated that mean differences across domains and summary scores ranged from 0.01 to 0.39 while estimates of agreement ranged from 0.76 to 0.91, all well within the equivalence threshold. Moderator analyses showed that time between administration, survey language, and type of electronic device did not influence equivalence. CONCLUSIONS: The results of the meta-analysis support equivalence of paper-based and electronic versions of the SF-36 and SF-12 across a variety of disease populations, countries, and electronic modes.

A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time.

Light chain (AL) amyloidosis is a rare disease associated with significant, irreversible organ dysfunction and high case fatality. An observational study was conducted to assess health-related quality of life (HRQoL) in patients treated for AL amyloidosis between 1994 and 2014 with both high dose melphalan and stem cell transplantation (HDM/SCT) or non-SCT chemotherapy regimens. The SF-36v1® Health Survey (SF-36) was administered to assess HRQoL during clinic visits. Analysis of variance was used to compare pre- and post-treatment HRQoL within each treatment group to an age- and gender-adjusted general population (GP) normative sample. Cox proportional hazard models were fit to examine associations between pre-treatment levels of HRQoL and mortality within 1 and 5 years after initiating specific treatment regimens (HDM/SCT: n = 402; non-SCT chemotherapy regimens: n = 172). Among patients who received HDM/SCT, there were significant improvements following treatment in vitality, social functioning, role-emotional and mental health. Worse pre-treatment SF-36 physical component scores were associated with a greater risk of mortality in both treatment groups and follow-up periods (P ≤ 0·005 for both). [Correction added on 20 October 2017, after first online publication: This P value has been corrected]. Using HRQoL assessments in every physician visit or treatment may provide valuable insights for treating rare conditions like AL amyloidosis.

Reliability and Validity of the Persian HIT-6 Questionnaire in Migraine and Tension-type Headache.

OBJECTIVE: Headache Impact Test (HIT-6) measures the impact headaches in a 1-month period. We validated the Persian translation of HIT-6, compared the HIT-6 psychometric analysis between migraine and tension-type headache (TTH) patients, and evaluated the capability of HIT-6 to differentiate between TTH, chronic migraine, and episodic migraine. METHODS: Qualified participants, including 274 patients diagnosed with migraine or TTH, were required to complete HIT-6, SF-36v2, and a symptoms questionnaire on their first visit. At 3 and 8 weeks from first visit, participants completed HIT-6. Internal consistency (Cronbach's α) and test-retest reproducibility (Pearson's correlation coefficient) were used to assess reliability. Convergent validity was also assessed. RESULTS: Tension-type headache, episodic, and chronic migraines included 24.5%, 61.9%, and 13.6% of the participants, respectively. Internal consistency among all patients, TTH, and migraine in the first visit were 0.74, 0.77, and 0.73, respectively. Test-retest reliability for HIT-6 between visit 1 and 2 showed a moderate level of correlation (r = 0.50). Convergent validity and also item total correlation were acceptable. There was no significant difference in HIT-6 total score between TTH and migraine. CONCLUSION: Persian HIT-6 is a valid and reliable questionnaire for the evaluation of headache. However, it cannot differentiate between chronic migraine, episodic migraine, and TTH in Iranian population.

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.

Health-related quality of life (HRQL) for individuals with self-reported chronic physical and/or mental health conditions: panel survey of an adult sample in the United States.

BACKGROUND: In the US, approximately 53% of adults have at least one chronic condition. Comorbid physical and mental health conditions often have an incremental negative impact on health-related quality of life (HRQL). Primary study objectives were to quantify the impact on HRQL of a) ≥ 1 physical condition , b) ≥ 1 comorbid mental health conditions added to a physical one, c) ≥ 1 mental health condition, and d) ≥ 1 comorbid physical conditions added to at least one related to mental health. Decrements were based on a "Healthy" reference group reporting no chronic conditions. METHODS: Participants were sampled (n = 3877) from the US adult population as part of a 2009 normative survey. Demographics, number/ type of chronic conditions, and HRQL data were self-reported. HRQL was defined through SF-36v2® Physical Component Summary (PCS) scores and Mental Component Summary (MCS) scores. Participant "morbidity" groupings included Healthy; Physical Health Condition only, Mental Health Condition only, and Physical and Mental Health (Comorbid). PCS and MCS scores were also analyzed by physical disease clusters (e.g., cardiovascular, gastrointestinal). Multivariate regression models were used for all analyses. RESULTS: 81% of participants were Caucasian; 9% African American. Males and females were about equally represented; 63% were ≥ 45 years old. The average number of reported chronic conditions was 2.4 (SD = 2.4). Relative to the Healthy group, the Physical Condition group scored 6.4 (males) and 7.5 (females) points lower on PCS. The addition of a comorbid mental health condition resulted in a total reduction of 11 points in PCS and 15 points in MCS. Compared to the Healthy group, ≥ 1 mental health conditions was associated with MCS decrements of 11-12 points. A physical comorbidity led to additional decrements of 3-4 points for MCS, with a total of 15 points. Incremental HRQL burden defined by both MCS and PCS scores was relatively similar across the 5 defined physical disease clusters. CONCLUSION: Results provide quantitative information for US adults on specific PCS and MCS score decrements associated with a comorbid condition related to mental health, as well as a comorbid condition related to physical health.

Long-term Follow-up of Cytologically Indeterminate Thyroid Nodules Found Benign on Molecular Testing: A Validation Study.

Objective: Molecular testing has revolutionized management of indeterminate thyroid nodules (Bethesda categories III and IV). Few studies have attempted to validate the negative predictive value of molecular tests. Using long-term observation as a surrogate for surgical resection, we sought to examine the false-negative rate of "benign" indeterminate thyroid nodules on molecular testing. Study Design: Case series with retrospective data collection and chart review. Setting: Large community-based practice with multiple satellite offices. Methods: All patients with thyroid nodules that underwent ultrasound-guided fine-needle aspiration biopsy between 2013 and 2019 were evaluated through retrospective analysis. Cytologically indeterminate nodules reflexively underwent molecular testing to guide clinical management. Observation was recommended for lesions with benign molecular testing, and these nodules were followed clinically and by ultrasound. Results: A total of 2011 nodules underwent fine-needle aspiration, of which 280 (14%) were indeterminate thyroid nodules. Of those 280 nodules, 100 (36%) were benign on molecular testing. Three samples were excluded from analysis due to patient deaths from unrelated causes. Surgical resection was recommended in 16 of the 97 nodules (17%), with the majority due to size and compressive symptoms. Histopathology was available in 14 nodules that underwent surgery, with 1 demonstrating minimally invasive follicular carcinoma. Conclusion: While molecular testing is safe to use in guiding management of indeterminate thyroid nodules, consideration of individualized clinical factors and close long-term follow-up remains paramount.

Qualitative research with patients and caregivers of patients with PIK3CA related overgrowth spectrum: content validity of clinical outcome assessments.

BACKGROUND: PIK3CA-Related Overgrowth Spectrum (PROS) are rare syndromes caused by a mutation in the PIK3CA gene, including fibroadipose hyperplasia or overgrowth; congenital lipomatous overgrowth, vascular malformations, epidermal nevi, scoliosis/skeletal and spinal (CLOVES); megalencephaly-capillary malformation (MCAP or M-CM); fibro-adipose vascular anomaly (FAVA); Klippel-Trenaunay syndrome (KT; also known as, Klippel-Trenaunay-Weber syndrome); capillary, lymphatic, and venous malformations (CLVM); and lymphatic malformation (LM). Characterized by malformations and tissue overgrowth, PROS manifests at birth or in early childhood. Pain and functional limitations associated with these conditions may greatly impact the health-related quality of life (HRQoL) of persons with PROS including physical functioning, work/school, social functioning, and emotional well-being. RESULTS: Selected clinical outcome assessments (COAs), identified during a literature review, were tested with adults with PROS, and children with PROS and their caregivers to determine comprehensibility, relevance, and appropriateness for measuring symptom severity and HRQoL. Tested were the Patient Global Impression of Symptom Severity (PGI-S), Brief Pain Inventory (BPI), Wong-Baker FACES, Patient-Reported Outcomes Measurement Information System (PROMIS) Profile, PROMIS Pediatric Short Form Sleep Disturbance, and PROMIS Dyspnea Severity. Qualitative interviews tested the self-report adult, self-report pediatric, and observer-report COAs with adults with PROS, and children with PROS and their caregivers. Ten adults (≥ 18 years old) with PROS, and 20 children (6-17 years old) with PROS and their caregivers, participated. All reported positive feedback on item relevance. Adults and children over the age of 12 comprehended and responded to self-reported items. Secondary objectives examined the age children could self-report their conditions using pediatric versions and assessed available observer-report versions of the COAs with caregivers. Some participants under the age of 12 had trouble understanding some terminology. Further, adults and children with cognitive impairment associated with MCAP/M-CM sometimes had difficulty with self-report. Caregivers were able to report their child's symptoms and impacts using observer-report COAs. Participant feedback prompted further consideration of the measurement of pain in this population, including variability of pain over time, location of pain, and type. CONCLUSIONS: This study provided valuable information from patients about PROS, supporting the content validity of the COAs, with recommended revisions. COAs are easily understood by persons with PROS and caregivers and are appropriate for measuring symptoms and disease-related impacts across diverse PROS syndromes in clinical trials.

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.

Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy.

BACKGROUND: Patients with Duchenne muscular dystrophy (DMD) adopt compensatory movement patterns as muscles weaken. The Duchenne Video Assessment (DVA) measures patient ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients performing specific movement tasks at home using a secure mobile application, and DVA-certified physical therapists (PTs) score the videos using scorecards with prespecified compensatory movement criteria. The goal of this study was to develop and refine the DVA scorecards. METHODS: To develop the initial scorecards, 4 PTs collaboratively created compensatory movement lists for each task, and researchers structured the lists into scorecards. A 2-round modified Delphi process was used to gather expert opinion on the understandability, comprehensiveness, and clinical meaningfulness of the compensatory movements on the scorecards. Eight PTs who had evaluated ≥50 patients with DMD and participated in ≥10 DMD clinical trials were recruited for the panel. In Round 1, panelists evaluated compensatory movement criteria understandability via questionnaire and tested the scorecards. In Round 2, panelists participated in an in-person meeting to discuss areas of disagreement from Round 1 and reach consensus (≥75% agreement) on all revisions to the scorecards. RESULTS: During the Round 1 revisions to the scorecards, there were 67 changes (44%) to the wording of 153 original compensatory movement criteria and 3 criteria were removed. During the Round 2 revisions to the scorecards, there were 47 changes (31%) to the wording of 150 compensatory movement criteria, 20 criteria were added, and 30 criteria were removed. The panel reached 100% agreement on all changes made to scorecards during Round 2. CONCLUSION: PTs with extensive experience evaluating patients with DMD confirmed that the compensatory movement criteria included in the DVA scorecards were understandable, comprehensive, and clinically meaningful.

Psychometric validation of the revised SCOPA-Diary Card: expanding the measurement of non-motor symptoms in Parkinson's disease.

BACKGROUND: To identify key non-motor symptoms of Parkinson's disease (PD) to include in a daily diary assessment for off-time, revise the Scales for Outcomes of Parkinson's disease Diary Card (SCOPA-DC) to include these non-motor symptoms, and investigate the validity, reliability and predictive utility of the Revised SCOPA-DC in a U.S. population. METHODS: A convenience sample was used to recruit four focus groups of PD patients. Based on findings from focus groups, the SCOPA-DC was revised and administered to a sample of 101 PD patients. Confirmatory factor analysis was conducted to test the domain structure of the Revised SCOPA-DC. The reliability, convergent and discriminant validity, and ability to predict off-time of the Revised SCOPA-DC were then assessed. RESULTS: Based on input from PD patients, the Revised SCOPA-DC included several format changes and the addition of non-motor symptoms. The Revised SCOPA-DC was best represented by a three-factor structure: Mobility, Physical Functioning and Psychological Functioning. Correlations between the Revised SCOPA-DC and other Health-Related Quality of Life scores were supportive of convergent validity. Known-groups validity analyses indicated that scores on the Revised SCOPA-DC were lower among patients who reported experiencing off-time when compared to those without off-time. The three subscales had satisfactory predictive utility, correctly predicting off-time slightly over two-thirds of the time. CONCLUSIONS: These findings provide evidence of content validity of the Revised SCOPA-DC and suggest that a three-factor structure is an appropriate model that provides reliable and valid scores to assess symptom severity among PD patients with symptom fluctuations in the U.S.

Measuring the health status burden in hemodialysis patients using the SF-36® health survey.

PURPOSE: The SF-36, a generic measure of 8 domains of health-related quality of life (HRQOL), has been widely used to examine HRQOL of end-stage renal disease (ESRD) patients undergoing hemodialysis (HD). The current study synthesizes existing literature to examine which SF-36 domains capture the largest burden in this patient population. METHODS: A literature search of published studies that presented descriptive statistics for baseline SF-36 scale scores from HD patients was conducted. Disease burden was estimated by comparing HD patients' SF-36 scores to those from either a control group or a general population normative sample taken from the same country. For each study, Cohen d effect sizes for between-sample differences were calculated for each scale. RESULTS: Twenty-six articles that matched set criteria were identified. Estimation of differences between HD patients and comparison groups showed that the SF-36 physical functioning scale yielded the largest weighted mean effect size across studies (d = 1.46), followed by the general health (d = 1.29) and role physical (d = 1.21) scales. CONCLUSIONS: Among the eight domains of the SF-36, physical functioning, general health, and role physical scales best captured disease burden for HD patients. The disease burden negatively impacts physical HRQOL more strongly than mental HRQOL.