Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS).

Abstract

INTRODUCTION:

Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice.

AIM:

To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS.

METHODS:

This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS).

RESULTS:

One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups).

CONCLUSIONS:

These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

• Authors: Johnny Mahlangu, Johannes Oldenburg, Michael U Callaghan, Midori Shima, Maria Elisa Mancuso, Peter Trask, Michael Recht, Claudia Garcia, Renchi Yang, Michaela Lehle, Harrison Macharia, Elina Asikanius, Gallia G Levy, Rebecca Kruse-Jarres, Sylvia von Mackensen
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 25 Issue 3 Pg. 382-391 (May 2019) ISSN: 1365-2516 [Electronic] England
• PMID: 31016855 (Publication Type: Journal Article, Multicenter Study)
• Copyright: © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd.

Topics

• Absenteeism
• Adolescent
• Adult
• Aged
• Child
• Female
• Health Status
• Hemophilia A (complications, epidemiology)
• Hemorrhage (complications)
• Hospitalization (statistics & numerical data)
• Humans
• Male
• Middle Aged
• Prospective Studies
• Quality of Life
• Schools (statistics & numerical data)
• Surveys and Questionnaires
• Work (statistics & numerical data)
• Young Adult