Dermatomyositis (DM) is a rare systemic autoimmune disease characterized by a distinctive debilitating skin rash and skeletal muscle weakness. It is unclear if existing clinical outcome assessment (COA) measures include the concepts of priority to patients and those necessary to fully capture improvements in the active cutaneous manifestations of DM. This study aimed to develop the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA), a de novo IGA, for use in clinical trials of adult DM. Eight DM clinical experts participated in 60-min qualitative interviews consisting of concept elicitation and cognitive debriefing methodologies. Concept elicitation comprised open-ended questions with follow-up probes to explore clinicians’ experiences of treating patients with DM, the impact of symptoms on patients’ quality of life, and the severity levels of disease characteristics to explore DM progression. Cognitive debriefing required the clinical experts to perform a review of the CDM-IGA, designed to assess the severity of cutaneous disease activity of DM. After the interviews, a consensus meeting with three clinical experts was held to agree on any outstanding issues relating to the CDM-IGA. The CDM-IGA was iteratively developed using the opinions of nine clinical experts. Feedback provided by all clinicians agreed that erythema was the main active cutaneous manifestation of DM and should be the primary characteristic on the CDM-IGA, split by erythema color and extent. To determine cutaneous disease severity, experts suggested adding a metric called secondary changes, which combined erosion/ulceration and lichenification, which could modify the patient’s final score. Three clinical experts suggested that a photo-guide to support assessments of erythema across different skin tones could be beneficial. A novel CDM-IGA was developed for use with adult patients with DM in clinical trials, based on an iterative development process that combined qualitative feedback from clinical experts of DM and importantly adult patients living with DM.

Dermatomyositis (DM) is a rare and severely debilitating autoimmune disease that can affect children and adults; however, there is little understanding of the patient-reported experience and uncertainty around validated clinical outcomes assessments (COAs) that could measure changes in the condition during clinical trials of new treatments. The aim of this study was to understand the patient experience of DM, with a focus on its cutaneous manifestations, to describe the patient experience and determine the suitability of existing COA measures. Adult (≥ 18 years) patients (N = 28) with severe active cutaneous manifestations of DM were interviewed. In the 90-min interviews, open-ended questions and probes were used to elicit descriptions of key clinical manifestations and patients’ experiences of DM, including the symptoms and impacts on their daily lives and wellbeing. Patients reported 13 different skin manifestations of DM. The most common were rash (n = 28, 100

Immunoglobulin A nephropathy (IgAN) is a kidney disorder that can lead to progressive kidney disease. Currently, there lacks a comprehensive overview of the symptoms and impacts experienced by those living with IgAN that would help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in clinical trials. The aim of this study was to develop a conceptual model of the adult and pediatric patient experience of IgAN, including disease signs and symptoms, treatment side effects, and impact on functioning and well-being. This study comprised a systematic review and thematic analysis of qualitative studies with adults and children diagnosed with IgAN. Data sources were identified through an electronic database search of journal articles (MEDLINE, Embase, PsycINFO; June 2021), hand-searching of conference proceedings, patient advocacy group websites, and gray literature. Non-English articles were excluded. Identified data (patient/caregiver quotes, author summaries, and interpretations of patient experiences) were extracted from articles. Extracted data were qualitatively analyzed, aided by ATLAS.ti v7. Codes were applied to data; concepts (i.e., symptoms) were identified, named, and refined. A conceptual model was developed by grouping related concepts into domains. In total, five sources were identified for analysis: two journal articles, two online anthologies of patient stories, and one patient organization-sponsored “Voice of the Patient” meeting report. Conceptual model symptom domains included swelling/puffiness (edema), pain/aches/discomfort, fatigue, weight gain, sleep problems, urinary problems, and gastrointestinal problems. Impact domains included emotional/psychological well-being, physical functioning/activities of daily living, social functioning, work/school, and relationships. Secondary analysis of published qualitative literature permitted development of a novel conceptual model depicting the patient experience of IgAN; however, its depth is limited by a lack of available literature. Further qualitative research is recommended to refine and/or confirm the concepts and domains, determine any relationships between them, and explore the outcomes that are most meaningful to patients. The refined model will provide a useful tool to inform the selection, development, and/or amendment of COAs for use in future IgAN clinical trials.

Objective Brepocitinib is a TYK2/JAK1 inhibitor in development for the treatment of several immunologic diseases. The efficacy and safety of oral brepocitinib were assessed in participants with moderately‐to‐severely active psoriatic arthritis (PsA) for up to 52 weeks. Methods In this placebo‐controlled, dose‐ranging, phase IIb study, participants were randomized to receive 10 mg, 30 mg, or 60 mg of brepocitinib once daily or placebo, advancing to 30 mg or 60 mg of brepocitinib once daily at week 16. The primary endpoint was the response rate according to the American College of Rheumatology criteria for 20% improvement (ACR20) in disease activity at week 16. Secondary endpoints included response rates according to the ACR50/ACR70 response criteria, 75% and 90% improvement in the Psoriasis Area and Severity Index (PASI75/PASI90) score, and minimal disease activity (MDA) at weeks 16 and 52. Adverse events were monitored throughout the study. Results Overall, 218 participants were randomized and treated. At week 16, the brepocitinib 30 mg and 60 mg once daily groups had significantly greater ACR20 response rates (66.7% [ P = 0.0197] and 74.6% [ P = 0.0006], respectively), versus the placebo group (43.3%), and significantly higher ACR50/ACR70, PASI75/PASI90, and MDA response rates. Response rates were maintained or improved through week 52. Adverse events were mostly mild/moderate; serious adverse events (15) in 12 participants (5.5%) included infections in 6 participants (2.8%) in the brepocitinib 30 mg and 60 mg once daily groups. No major adverse cardiovascular events or deaths occurred. Conclusion Treatment with brepocitinib at dosages of 30 mg and 60 mg once daily was superior to placebo at reducing signs and symptoms of PsA. Brepocitinib was generally well tolerated throughout the 52‐week study, with a safety profile consistent with those found in other brepocitinib clinical trials.

Objective: A review of new oncology indications approved by the European Medicines Agency (EMA) for 2012-2016 showed that 33% of new drugs had labeling based on patient-reported outcomes (PROs). We reviewed labeling text based on PRO endpoints for new oncology indications approved during 2017-2021.Methods: New oncology drugs approved by EMA to treat indications of cancers during 2017-2021 were identified from the EMA website. PRO-related language reported in EMA summaries of product characteristics (SmPCs) were summarized and compared with similar findings reported for oncology indications approved during 2012-2016.Results: Review documents by the EMA during 2017-2021 were available for 49 new oncology drugs for 70 cancer indications. Submissions for 52 (74.3%) of the 70 indications included PRO data for EMA review. Of all submissions, 14 (20.0%) approvals contained PRO-related language in the SmPC. Broad concepts such as health-related quality of life were most common and found in 8 of 14 (57.1%) PRO-related labels.Conclusion: PRO-related language appeared in SmPCs for 20% of all indications of new oncology drugs approved by EMA during 2017-2021 compared with approximately 33% of EMA approvals during 2012-2016. PRO-related labeling during the same periods showed a greater decline (from 47% to 27%) for indications of new oncology drugs that also included PRO data. One possible reason for this decline may be the increase in open-label studies from 62% between 2012 and 2016 to approximately 79% between 2017 and 2021.

IntroductionFocal segmental glomerulosclerosis (FSGS) is a leading cause of kidney disease and can progress to end stage kidney disease (ESKD). An overview of symptoms and impacts of the disease experienced will help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in FSGS clinical trials. This study aimed to develop a conceptual model (CM) of the adult and pediatric patient experience of FSGS including disease signs/symptoms, treatment side-effects, and impact on functioning and wellbeing.MethodsThis study comprised a systematic review and thematic analysis of qualitative studies with adults and pediatric patients diagnosed with FSGS. Data sources were identified through an electronic database search of journal articles (Medline, Embase, PsycINFO; June 2021) and hand-searching of conference proceedings, patient advocacy group websites, and gray literature. Non-English articles were excluded. Identified data (patient/caregiver quotes, author summaries, and interpretations of patient experiences) were extracted from the articles. Extracted data were qualitatively analyzed aided by ATLAS.ti v7. Codes were applied to data and concepts (symptoms/impacts) were identified, named, and refined. A CM was developed by grouping related concepts into domains.ResultsIn total, 12 sources were identified for analysis: 6 journal articles and 6 series of patient testimonials. Salient sign/symptom/side-effect domains included swelling/puffiness (edema), pain/aches/discomfort, fatigue, weight changes, skin problems, respiratory problems, and sleep problems. Salient impact domains included emotional/psychological wellbeing, physical functioning/activities of daily living, social functioning, and work/school.ConclusionSecondary analysis of published qualitative literature permitted development of a CM describing the adult and pediatric experience of FSGS. Concept elicitation interviews are recommended to refine the CM, confirm the salient/most bothersome concepts, and confirm the extent of impact on daily life. The refined CM will provide a useful tool to inform the selection, development, and/or amendment of COAs for use in future FSGS clinical trials.

Individuals with alopecia areata (AA) may experience significant impacts on their health-related quality of life. The novel Alopecia Areata Patient Priority Outcomes (AAPPO) questionnaire has been developed to assess hair loss signs, emotional symptoms, and activity limitations associated with AA. The objective of this study was to evaluate psychometric properties and establish scoring of the AAPPO in adults and adolescents with AA. Scoring and measurement properties of the AAPPO were examined using baseline and 2-week follow-up data from a prospective, noninterventional, web-based study of 121 patients with AA (85 adults aged ≥ 18 years, 36 adolescents aged 12–17 years) with Severity of Alopecia Tool (SALT) ≥ 25% scalp hair loss. Exploratory and confirmatory factor analysis supported four single Hair Loss (HL) items, an Emotional Symptoms domain (ES; 4 items), and an Activity Limitations domain (AL; 3 items). Among all patients, the multi-item ES and AL domains had strong internal consistency (α ≥ 0.87); all HL items and domain scores had strong test-retest reliability (weighted kappa or intraclass correlation coefficients ≥ 0.78). All HL item scores demonstrated strong construct validity (r ≥ 0.52) compared with the patient-reported Alopecia Areata Symptom and Impact Scale (AASIS) hair loss subscale score; ES and AL domain scores exhibited strong construct validity (r ≥ 0.66) compared with the SF-36 Mental Component Summary (MCS) score. Using SALT scores, HL mean item scores were better (lower) in the 25–49% SALT subgroup versus those with highest SALT scores (76–100%); however, ES mean domain scores were better in the SALT 76–100% subgroup in the same comparison (p < 0.0001). Using AASIS and MCS score–created subgroups, ES and AL mean domain scores demonstrated hypothesized differences across subgroups (all p values < 0.0001). The AAPPO questionnaire is a reliable, valid disease-specific measure of hair loss severity and impact in individuals with AA.

The phase 2a ALLEGRO trial (NCT02974868) investigated the safety and efficacy of ritlecitinib (PF‐06651600) and brepocitinib (PF‐06700841) in adults with alopecia areata. No randomized controlled trial for alopecia areata has evaluated correlations between clinician‐assessed hair loss and patient‐reported outcomes.

INTRODUCTION:Although alopecia areata (AA) profoundly impacts patients' physical appearance, emotional state, and daily activities, no treatment approved for AA currently exists. Patient-reported outcome (PRO) instruments currently used to capture patients' AA experiences do not meet the requirements to support claims of treatment benefit as described in the US Food and Drug Administration's 2009 PRO guidance. Our objective was to explore the consequences and priority treatment outcomes among individuals with AA and develop a PRO measure consistent with regulatory requirements that assesses these priorities and represents clinical benefit from the AA patient perspective.METHODS:Targeted literature and instrument reviews informed an initial concept set. Concept elicitation interviews with 20 adults with AA confirmed the relevance and importance of the initial concepts, identified additional relevant concepts, and informed an AA consequence model. Thematic analysis yielded a draft item pool, which was evaluated through two iterative rounds of cognitive debriefing interviews with 16 patients with AA (9 adults; 7 adolescents).RESULTS:Hair loss was the primary consequence of importance to patients with AA. Patients emphasized the need to differentiate hair loss by location: scalp, eyebrows, eyelashes, and body. Consequences of AA include difficulty conducting daily activities, particularly outdoor activities and exercise, and emotional impacts such as sadness, frustration, and negative self-image. Following cognitive debriefing interviews, 11 items were included to form the Alopecia Areata Patient Priority Outcome (AAPPO), assessing AA-related symptoms and impacts over the past week.CONCLUSIONS:The AAPPO is a novel, content-valid PRO that captures the consequences of AA of the highest priority to patients.

Alopecia areata (AA) is a chronic, autoimmune disease of hair loss, which can significantly affect the emotional and psychological well-being of patients. A systematic literature review was conducted to better understand the burden of AA from the patient perspective. Embase, MEDLINE and Cochrane databases were searched for published studies (2008–2018) reporting on assessments of health-related quality of life (HRQoL) for patients with AA. Qualitative, and quantitative data were collected. The review included 37 studies encompassing a range of clinical outcome assessment (COA) tools. None of the COA tools were specific for AA, and only one study used the Hairdex scale, which was designed to evaluate HRQoL in patients with disorders of the hair and scalp. All studies reported substantial impact on HRQoL due to AA, both overall and in domains related to personality (i.e. temperament and character), emotions and social functioning. Acute stress was also noted, and several studies identified lack of emotional awareness (alexithymia) in 23–50% of the patients with AA. Although it is well-established that patients with AA experience anxiety and depression, they also experience a decrease in HRQoL in many other areas, including personality, emotions, behaviors and social functioning, and these changes may be accompanied by acute stress and alexithymia. There is a need to achieve consensus on a core set of measures for AA and to develop and validate AA-specific measurement tools for use in future studies, to attain a clearer understanding of the impact of AA on patients. PROSPERO registration number; CRD42019118646. Alopecia areata (AA) is a disease in which a person’s immune system attacks their hair follicles, from which hairs grow, causing hair loss. Studies have shown that people with AA may have a lower quality of life, and studies have reported higher rates of depression and anxiety in people with AA than in people without AA. Study design: We reviewed published studies to better understand how AA affected people socially, emotionally and in their day-to-day functioning. We also looked at how healthcare providers measured these social, emotional and day-to-day effects on people with AA. Our review included 37 published studies that used several evaluation tools to measure the impacts of AA. These included a variety of questionnaires that were answered by people with AA. Results: The studies reported that AA negatively affected the personality, emotions, behaviors and/or social functioning of many people with AA. However, none of the evaluation tools that were used in those studies were specific for AA, and most of the evaluation tools did not include questions about the hair or scalp. Conclusions: We recommend that a group of people familiar with AA (practitioners, researchers and patients) work together to develop an evaluation tool that is designed specifically for people with AA. This evaluation tool can then be used in future studies to better understand how AA affects people socially, emotionally and in their day-to-day functioning.

To establish the domain structure and scoring, and evaluate the psychometric properties of the Alopecia Areata Patient Priority Outcomes (AAPPO) questionnaire in adults and adolescents with Alopecia Areata (AA). Measurement properties of the AAPPO were examined using data from a prospective, non-interventional, web-based study of 121 AA patients (36 adolescents ages 12-17 years and 85 adults ages ≥18 years) with ≥25% hair loss, who completed two assessments (baseline and follow up 2 weeks later). Analyses provided here are inclusive of all patients (n=121). Exploratory and confirmatory factor analysis supported 6 domains: 4 individual Hair Loss (HL) items, Emotional Symptoms (ES, 4 items) and Activity Limitations (AL, 3 items). The multi-item ES and AL domains had strong internal consistency (Cronbach’s alpha≥0.87). All domain scores had strong test-retest reliability (ICC≥0.78). The four HL domain scores demonstrated strong construct validity (r≥0.52) when compared to the patient-reported Alopecia Areata Symptom and Impact Scale hair loss scale score. The ES and AL domain scores exhibited strong construct validity (ǀrǀ≥0.66) when compared to the patient-reported SF-36 Mental Component Summary score. Known groups analyses revealed that the HL mean domain scores were better (lower) in the clinician-reported Severity of Alopecia Tool (SALT) 25%-49% subgroup compared to those with highest SALT scores (76%-100%; p<0.0001). However, the ES mean domain scores were better for the SALT 76%-100% group in the same comparison (p<0.0001). These data demonstrate the reliability and validity of the AAPPO to measure symptom severity and impacts in adults and adolescents with AA.

Vitiligo is an autoimmune depigmentation disorder that affects patients’ appearance and health related quality of life. This research evaluated the content validity of the Self-Assessment Vitiligo Extent Score (SA-VES) measure. The SA-VES assesses extent of vitiligo-related body surface area (BSA) depigmentation, on a 7-point pictorial scale ranging from no involvement to most involvement, in 12 body locations. This study consisted of in-person qualitative interviews with adolescents and adults with vitiligo. Participants completed the measure on an electronic tablet device and were debriefed about their understanding of the SA-VES items (comprehensibility), conceptual relevance, suitability of response options, and the ease of tablet use. The study included twenty adults and ten adolescents, aged 13-77 years, predominantly non-segmental vitiligo (n=23, 76%) and Fitzpatrick skin type I-III (n=24, 80%). Clinician-reported vitiligo BSA involvement ranged from 4-70%. The SA-VES was generally well received, with participants reporting high levels of item understanding (n=29/30, 96%), conceptual relevance (n=26/30, 86%), suitability of response options (n=27/30, 90%), and ease of tablet use (n=25/30, 83%). However, some limitations related to the understanding of the written instructions were observed, with participants selecting responses based on lesion location rather than extent of the lesions. Revised instructions were tested with six participants and improved understanding in most (n = 4/6, 66%). The interviews also revealed that in a few items, some body parts were pictured in the response options but were not specifically named in the question. This may have caused confusion when selecting responses. For adolescents and adults with vitiligo, the SA-VES pictorial scale was well-understood, conceptually relevant, and easy to complete using a tablet. Following discussions with a vitiligo clinical expert, six item labels were amended for clarification. With some modifications and testing, the SA-VES may be suitable for assessing patient-reported vitiligo BSA involvement in clinical research.

Vitiligo is an autoimmune depigmentation disorder that affects patients’ appearance and health related quality of life. This research explored patients’ understanding of the Vitiligo-Specific Quality of Life (VitiQoL) measure including the instructions, item content, response scale, and a modified recall period. The VitiQoL consists of 15 items assessing impacts of vitiligo, using a 0–5 Likert response scale with 1-month recall period and a global impression of severity item. Recall was modified to 1-week (VitiQoL 2.0). The study included face-to-face qualitative interviews with adolescents and adults with vitiligo. Participants were asked about their understanding of the VitiQoL 2.0 items, conceptual relevance, suitability of response options, and appropriateness of the modified recall period. The study included 20 adults and 10 adolescents, aged 13-77 years, predominantly non-segmental vitiligo (n=23/30, 76.7%) and Fitzpatrick skin type I–III (n=24/30, 80%). Clinician-reported vitiligo body surface area (BSA) involvement ranged from 4-70% overall (90% adolescents and 30% adults with BSA= 4-20%) The VitiQoL 2.0 was generally well received, with participants reporting high levels of item understanding (n=29/30, 96%), as well as suitability of response options (n=29/30, 96%) and of the modified recall period (n=28/30, 93%). Most adults (n=17/20, 85%) and adolescents (n=6/10, 60%) reported that the items were relevant. For two items (3: effect on showing affection; 6: fear of others finding fault), 50% of participants found the concepts relevant and nearly all understood the items, but interpretations were inconsistent. While the VitiQoL 2.0 adequately captured the impacts of vitiligo in adults, it had limited conceptual relevance among adolescents. This was possibly due to lower average BSA involvement among adolescent participants who were therefore less likely to experience similar disease impact. With minor modifications, the VitiQoL 2.0 may be suitable to capture patients’ perspective of disease impact in vitiligo clinical trials.

BACKGROUND:Soft tissue sarcomas (STS) are a heterogenous group of rare tumors that involve the connective tissue in the body (e.g. muscle, tendons). As with many rare tumors, little is known about the impact of STS on patient well-being.OBJECTIVE:The aim of this review was to better understand current knowledge related to patient experience and quality of life (QOL) following diagnosis of STS.METHODS:A systematic review of English-language articles published from 2005 to 2015 was conducted in the PubMed/MEDLINE, Embase, PsychINFO, and Evidence-Based Medicine databases. The review included recent conference proceedings and advocacy websites. Articles were eligible if they included adult STS patient-reported outcomes (PROs) or details on patient experience.RESULTS:Overall, 3430 articles were identified and 20 were eligible for inclusion. Of these, 14 were clinical studies that included PRO measures, 1 summarized PRO measures used in STS studies, and 5 described the STS patient experience. Patients with STS report a range of impacts on QOL, including emotional well-being, body image, functional deficit following surgery, and practical considerations such as child care and work.CONCLUSIONS:Few studies have published either qualitative or quantitative data on the patient experience with STS. While STS has a measurable impact on QOL, there is a lack of detailed information in the published literature. Although PROs are used in clinical studies of STS, they are not STS-specific and may not capture the unique needs of this population. There is a need for qualitative research to better understand both patient and caregiver experiences in STS.

61 Background: Soft tissue sarcomas (STS) are a heterogenous group of rare tumors that involve the connective tissue in the body. As with many rare tumors, little is known about the impact of STS on patient well-being. The objective of this review was to understand patient experience and quality of life (QOL) following diagnosis of STS. Methods: This was a systematic review of English language articles published between 2005-2015 in PubMed/Medline, Embase, PsychINFO, and Evidence-Based Medicine. The review included recent conference proceedings and resources from advocacy websites. Articles were considered relevant if they included adult STS patient reported outcomes (PROs) or details on patient experience. Results: Of the 3,430 articles identified, 20 were eligible for inclusion. Of these, 14 were clinical studies that included PRO measures, 1 summarized PRO measures used in STS studies, and 5 described the STS patient experience. Patients with STS report a variety of concerns including emotional well-being (e.g. anxiety, depression), body image, functional deficit following surgery, and practical considerations such as obtaining child care and ability to work. Reports of patient experience vary widely given the heterogeneity of STS and individualized treatment. In clinical studies that used PROs, the European Organization for Research and Treatment of Cancer QOL Questionnaire Core 30 (EORTC QLQ-C30) was the most commonly used cancer-specific measure (N = 5) followed by the Functional Assessment of Cancer Therapy–General (N = 2). The generic EQ-5D was used in 5 publications but was limited to the visual analog scale portion of the measure in two of these studies. Similar to other cancers, STS patients report lower QOL when compared to the general population. Conclusions: Few studies have published either qualitative or quantitative data on the patient experience with STS. While STS has a measurable impact on QOL, there is a lack of detailed information in the published literature. Although PROs are often used in clinical studies of STS, they are not STS-specific tools and may not capture the unique needs of this population. There is a need for qualitative research to better understand the patient perspective of STS.

BackgroundPatient-reported outcomes (PROs) are increasingly used to demonstrate the value of interventions and support health technology assessment (HTA).ObjectiveThe objective of this work was to analyze trends regarding PROs in Latin America (LatAm), highlight challenges in the application of PROs in this region, and suggest solutions.MethodsA team of researchers with expertise in PROs conducted a nonsystematic PubMed literature search pertaining to the use of PROs in LatAm. The experts also drew on their experience working with PROs to assess the application of PROs in LatAm.ResultsThe literature search yielded more than 4000 publications, with an increasing publication rate in recent years. PROs are being used in LatAm in various study types: instrument validation, phase III international clinical trials, health service research. A large Inter-American Development Bank study demonstrates the growing importance of PROs in the region. The growth in local value sets for the EuroQol five-dimensional questionnaire in LatAm reflects the regional emergence of HTA systems. Operational challenges relate to ensuring the use of good-quality questionnaires that, at a minimum, have undergone appropriate cultural adaptation and ideally have established psychometric properties.ConclusionsPROs are increasingly important in LatAm. Future efforts should aim to strengthen the operational and research infrastructure around PROs in the region. Innovation should be encouraged, including studying alternative methods of eliciting health utilities for economic evaluation. A wider scope around PRO uses for decision making by HTA bodies is an international trend with potential positive prospects in LatAm.

BACKGROUND AND PURPOSE:To investigate resource use and burden associated with spina bifida (SB) in Germany. METHODS:A questionnaire was used to obtain information on SB-related healthcare resource use and assistive technologies used for the last 1 and 10 years. Individuals with SB were recruited at a tertiary specialist clinic. To participate, persons with SB required the cognitive ability to respond or a caregiver to answer questions on their behalf. They could use personal medical charts or other records to answer. The analyses included assessment of frequency and extent of resource use for both time frames. RESULTS:Data on 88 persons with a diagnosis of SB were collected (44% female). During the last year, 88.6% (N = 78) reported at least one visit to a general practitioner's (GP's) office, 77.3% (N = 68) to a urologist and 69.3% (N = 61) to a physiotherapist. The annual average number of visits was 7.6 GP, 3.6 urologist and 65.3 physiotherapist visits. Amongst those hospitalized, a single hospitalization lasted 7.3 days on average, whereas the average annual number of hospital days was 14.8 days. During the previous 10 years, 67.0% (N = 59) of responders used a wheelchair, 64.7% (N = 57) used glasses and 59.1% (N = 52) used orthopaedic shoes, with an average of 2.5, 2.8 and 6.1 new items used, respectively. CONCLUSIONS:The results indicate that persons with SB require a substantial amount of interaction with healthcare providers, as well as other healthcare-related resource use, both in the shorter and longer terms.