Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.

ABSTRACT:

Background

Patient-reported outcomes (PROs) reflect patient perceptions of disease and treatment and are important for evaluating new therapies.

Objectives

Evaluate the effects of once-daily concizumab prophylaxis on health-related quality of life (HRQoL), treatment burden, and treatment preference in males aged ≥12 years with hemophilia A/B with inhibitors.

Methods

Patients enrolled in the multicenter, open-label explorer7 phase 3 study (ClinicalTrials.gov identifier: NCT04083781) were randomized to receive no prophylaxis (arm 1) or concizumab prophylaxis (arm 2) or were nonrandomly allocated to concizumab prophylaxis (arms 3 and 4). The study included questionnaires to assess patients' perception of HRQoL (Haemophilia Quality of Life Questionnaire for Adults), treat

SUBMITTER: Tran H 

PROVIDER: S-EPMC11295565 | biostudies-literature | 2024 May

REPOSITORIES: biostudies-literature