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Purpose: The aim of this study was to generate evidence supporting the development and content validity of a new PRO instrument, the Small Intestinal Bacterial Overgrowth (SIBO) Symptom Measure (SSM) daily diary. The SSM assesses symptom severity in SIBO patients, with the ultimate goal of providing a fit for purpose PRO for endpoint measurement., Methods: Qualitative research included 35 SIBO patients in three study stages, using a hybrid concept elicitation (CE)/cognitive interview (CI) method with US patients, ≥ 18 years. Stage 1 included a literature review, clinician interviews, and initial CE interviews with SIBO patients to identify symptoms important to patients for inclusion in the SSM. Stage 2 included hybrid CE/CI to learn more about patients' SIBO experience and test the draft SSM. Finally, stage 3 used CIs to refine the instrument and test its content validity., Results: In stage 1 (n = 8), 15 relevant concepts were identified, with items drafted based on the literature review/clinician interviews and elicitation work. Within stage 2 (n = 15), the SSM was refined to include 11 items; with wording revised for three items. Stage 3 (n = 12) confirmed the comprehensiveness of the SSM, as well as appropriateness of the item wording, recall period, and response scale. The resulting 11-item SSM assesses the severity of bloating, abdominal distention, abdominal discomfort, abdominal pain, flatulence, physical tiredness, nausea, diarrhea, constipation, appetite loss, and belching., Conclusions: This study provides evidence supporting the content validity of the new PRO. Comprehensive patient input ensures that the SSM is a well-defined measure of SIBO, ready for psychometric validation studies., (© 2023. The Author(s).)

Objective: Voxelotor is a first-in-class sickle hemoglobin-polymerization inhibitor that was approved in 2019 by the US Food and Drug Administration for treatment of patients with sickle cell disease (SCD) aged ≥12 years; in 2021, the approval was extended to children with SCD aged 4 to 11 years. Additionally, both the Ministry of Health and Prevention for the United Arab Emirates and the European Commission granted marketing authorization for voxelotor in September 2021 and February 2022, respectively, for treatment of SCD in adults and pediatric patients aged ≥12 years. Thus, additional information on the patient experience with voxelotor would be useful for patients, caregivers, and healthcare professionals alike. The purpose of this study was to conduct semistructured interviews in an effort to understand the experiences and perspectives of voxelotor-treated patients with SCD., Methods: One-time semistructured interviews with adults, adolescents, and children with SCD and their primary caregivers were conducted in the United States. Twenty-three adults and adolescents were recruited across 4 clinical sites, and 10 children-caregiver dyads were recruited from a single site. The interview was designed to elicit patient perspectives on symptomatic changes with voxelotor and the impact of treatment on patients' perceived health-related quality of life. Individual interview transcripts were analyzed using a thematic analytic approach, and concept saturation was assessed in both cohorts., Results: Most patients reported improvements in their SCD symptoms with voxelotor treatment, specifically regarding pain crises, jaundice, and fatigue. Almost all patients experienced improvements in self-reported health-related quality of life with voxelotor treatment., Conclusions: This study provides patient and caregiver perspectives on the symptomatic benefits of voxelotor treatment. These findings not only highlight the benefits of voxelotor treatment in improving symptoms and increasing health-related quality of life across the entire SCD population but also can inform further research on SCD-specific patient-reported outcomes., Competing Interests: Clark Brown is an employee at Global Blood Therapeutics, Inc.; a consultant at Imara and Novo Nordisk; and a recipient of the research funding from Forma Therapeutics, Global Blood Therapeutics, Inc., Imara, and Novartis. Modupe Idowu is on the advisory board at Novartis; on the advisory board and a speaker at Global Blood Therapeutics, Inc.; and a recipient of the research funding from Pfizer Inc., Novartis, Global Blood Therapeutics, Inc., and Forma Therapeutics. Richard Drachtman is a consultant and speaker at Global Blood Therapeutics, Inc. and Agios; a speaker at Jazz Pharmaceuticals; and a consultant at Bluebird Bio. Anne Beaubrun is an employee at Global Blood Therapeutics, Inc. Irene Agodoa and Andy Nguyen are former employees at Global Blood Therapeutics, Inc. Kelly Lipman, Olga Moshkovich, Ryan Murphy, and M. Alex Bellenger are employees at ICON plc. Wally Smith is a consultant at Global Blood Therapeutics, Inc., Novartis, Pfizer Inc., GlycoMimetics, Ironwood, Novo Nordisk, Emmaus Pharmaceuticals, Fera Pharmaceuticals, and Agios Pharmaceuticals and a recipient of the research funding from Global Blood Therapeutics, Inc., NHLBI, PCORI, HRSA, Novartis, Forma Therapeutics, Imara, and Shire., (Copyright © 2023 Clark Brown et al.)

Objectives: Lupus imposes a substantial burden on patients; however, little is known about its impact on those caring for patients with the disease. In this study, we examined the impact 'caring for patients with lupus' has on caregivers from their own perspective., Methods: UNVEIL was a one-time online national cross-sectional survey developed in partnership with the Lupus Foundation of America and fielded targeting the US Lupus Foundation of America constituents in 2014. Eligible caregivers were adults who self-identified as unpaid caregivers of patients with lupus. Eligible caregivers had to complete a series of sociodemographic questions as well as a series of well established outcome measures, such as the Short Form 12v2 Health Survey, the Work Productivity and Activity Index, the Caregiver Burden Inventory, and the Perceived Benefits of Caregiving Scale., Results: A total of 253 caregivers completed the survey. The majority of caregivers (90.1%) were aged 60 years or younger, more than half (54.2%) were men, and more than half (59.7%) identified themselves as either a spouse or a partner to the patient with lupus they were caring for. Overall health-related quality of life was close to the norm mean of the general US population. Caregivers who were employed missed an average of 12.8% of paid work time due to caregiving responsibilities and reported a 33.5% reduction in on-the-job effectiveness. Nearly half of the caregivers surveyed (49.4%) indicated that their caregiving responsibilities impacted their ability to socialize with friends, and almost all caregivers (97.6%) reported experiencing increased anxiety and stress in relation to their caregiving role., Conclusions: Caregiving for patients with lupus has a substantial impact on the work productivity and the social and emotional functioning of caregivers. Healthcare professionals and policymakers should continually assess the impact of healthcare decisions on the well-being of those caring for patients with lupus., (© The Author(s) 2016.)