Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments.

BACKGROUND: CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is ultra-rare (< 100 children or young adults worldwide) and potentially fatal. The study used mixed-methods approaches to assess how pozelimab impacts the signs and symptoms of CHAPLE disease from the patient perspective by combining within-trial interviews and clinical outcome assessments (COAs) (ClinicalTrials.gov, NCT04209634). METHODS: Interviews conducted with patients/caregivers at screening and week 24 assessed the signs and symptoms of CHAPLE disease, including those which were most bothersome, and evaluated the change. Patients/caregivers and clinicians completed the COAs; interview data contextualized the meaningfulness of change. RESULTS: Ten patients (aged 3-19 years) were enrolled; caregivers contributed to nine interviews. Abdominal pain, diarrhea, facial and peripheral edema, nausea, and vomiting are the core signs and symptoms of CHAPLE disease (≥ 90% patients experienced pre-treatment); the most bothersome signs and symptoms were abdominal pain (n = 9) and facial edema (n = 1). All core signs and symptoms were reported as resolved at week 24 interviews. Severity on global assessments changed from "mild" to "very severe" at baseline to "no signs or symptoms" at week 24. Interview results were generally consistent with sign- or symptom-specific COA scores. CONCLUSIONS: Patients with CHAPLE disease treated with pozelimab for 24 weeks experienced complete resolution of core signs and symptoms. Mixed-methods approaches can contextualize the patient experience (how patients feel and function) in rare disease trials. TRIAL REGISTRATION: Clinicaltrials.gov, NCT04209634, registered December 24, 2019, https://classic. CLINICALTRIALS: gov/ct2/show/NCT04209634 .

Virtual voices: examining social support exchanged through participant-generated and unmoderated content in a mobile intervention to improve HIV antiretroviral therapy adherence among GBMSM.

Antiretroviral therapy (ART) adherence is suboptimal among gay, bisexual, and other men who have sex with men (GBMSM). Online interventions that incorporate social support represent new opportunities to improve adherence. This study focused on how social support was provided and sought within a technology-based ART adherence intervention. We coded and analyzed 1,751 messages. Within the social support messages, half of the time participants sought social support and half of the time they provided social support. Emotional and informational support were the most frequently exchanged forms. The most frequent topic that participants sought support around was interpersonal relationships (29%), followed by HIV care and treatment (28%). Similarly, 31% and 27% of messages in which participants provided support was related to HIV treatment and care and interpersonal relationships, respectively. HIV treatment and care issues most salient were ART adherence, lab results and upcoming tests, ART side effects, changes in ART regimens, and relationships with healthcare providers. Participants used the messaging feature in this intervention to spontaneously discuss and exchange support around HIV treatment and care. This analysis provided an opportunity to understand how participants informally interact with one another, how they seek and provide social support online, and their salient personal issues.