The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare condition.

ABSTRACT:

Background

CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is a newly identified condition with an estimated worldwide prevalence of < 100 patients. Patient interviews can ensure that what is important to patients is assessed in a clinical trial program. Due to the rare and potentially fatal nature of CHAPLE disease, interviews were conducted as part of the pozelimab clinical trial, rather than in a separate study before the trial. The aim of the interviews was to identify the key disease-related signs, symptoms, and health-related quality-of-life (HRQoL) impacts that are important and relevant to patients with CHAPLE disease.

Methods

Interviews were conducted with patients and/or caregivers at two

SUBMITTER: Litcher-Kelly L 

PROVIDER: S-EPMC11817392 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature