Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study.

ABSTRACT:

Background

Acute hepatic porphyria (AHP) is characterized by debilitating and potentially life-threatening neurovisceral attacks, possible chronic symptoms, and long-term complications. In a phase 1/2 open-label extension (OLE) study and the phase 3 ENVISION study, givosiran led to sustained improvement in annualized attack rate and quality of life (QOL) measures. To capture the patient experience of symptoms and impacts of AHP, and any changes experienced during treatment with givosiran, qualitative interviews were conducted with study participants.

Methods

Participants who continued givosiran treatment after completing the phase 1/2 OLE study and the phase 3 ENVISION study participated in semi-structured interviews (i.e., loosely structured interviews on a predetermined to

SUBMITTER: Naik H 

PROVIDER: S-EPMC11731774 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature