Giancarlo Castaman, Cléa Percier, Naveen Shridhar, Neil Reynolds, Olivera Rajkovic-Hooley, Thomas Dewar, Victor Jiménez-Yuste
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引用次数: 0
Abstract
Introduction: Although prophylactic treatments can reduce bleeding risk for people with hemophilia (PwH), burdens associated with treatment and physical and psychological symptoms may persist.
Aim: To determine treatment and disease burden associated with hemophilia from a global perspective.
Methods: This cross-sectional patient survey captured patient-reported outcomes, experiences and clinical data from PwH in eight countries between December 2023 and March 2024. Treatment burden was measured using the child/adult Hemophilia Treatment Experience Measure (Hemo-TEM), validated questionnaires covering five domains. Disease burden was measured using the Patient-Reported Outcomes Measurement Information System 29+2 version 2.1 (PROMIS-29+2)/PROMIS Parent Proxy, validated questionnaires comprising eight domains. Here we report data on treatment, physical and psychological burdens for people with moderate and severe hemophilia without inhibitors by treatment class: standard half-life and extended half-life factor replacement for hemophilia A (HA) and hemophilia B (HB), and non-factor therapy for HA.
Results: Data from 495 PwH and parents/guardians of PwH were analyzed. Across the five Hemo-TEM domains, adults with HA experienced similar degrees of treatment burden regardless of treatment class, with comparable results observed for adults with HB. Around one-third of PwH experienced soreness (33% HA; 31% HB) and pain (31% HA; 26% HB) due to their current treatment at least sometimes, and one in five experienced some level of treatment bother. PROMIS-29+2 scores indicated that people with HA and HB experienced similar degrees of physical and psychological burden regardless of treatment class. Anxiety and worry were prevalent among PwH.
Conclusion: PwH continue to experience treatment- and disease-related burdens. Managing hemophilia still requires a balance between bleed protection, quality of life and ease of treatment administration.
期刊介绍：
Patient Preference and Adherence is an international, peer reviewed, open access journal that focuses on the growing importance of patient preference and adherence throughout the therapeutic continuum. The journal is characterized by the rapid reporting of reviews, original research, modeling and clinical studies across all therapeutic areas. Patient satisfaction, acceptability, quality of life, compliance, persistence and their role in developing new therapeutic modalities and compounds to optimize clinical outcomes for existing disease states are major areas of interest for the journal.
As of 1st April 2019, Patient Preference and Adherence will no longer consider meta-analyses for publication.
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