Document Type

Article

Publication Date

5-2026

Identifier

DOI: 10.1111/hae.70271; PMCID: PMC13175439

Abstract

INTRODUCTION: Despite available prophylactic therapies for haemophilia A, breakthrough bleeding and consequent pain and joint damage still occur.

AIM: To provide insights into the unmet treatment needs of people with moderate-to-severe haemophilia A in the US.

METHODS: For this descriptive, observational cohort study, eligible participants diagnosed with moderate-to-severe haemophilia A were enrolled in the PicnicHealth database on or before 3 October 2022. Three patient groups were established to identify: (1) treatment patterns; (2) clinical outcomes by therapy class (standard half-life [SHL] factor VIII [FVIII]; extended half-life FVIII [EHL]; emicizumab); (3) health-related quality of life (HRQoL) using the Patient-Reported Outcomes Measuring Instruments System (PROMIS)-29 survey and a supplemental pain survey. Patients switching treatment classes were included in multiple treatment groups.

RESULTS: Of 211 participants in the 'treatment patterns population', 123 (58.3%) used SHL FVIII prophylaxis, 81 (38.4%) EHL FVIII prophylaxis, and 83 (39.3%) emicizumab prophylaxis. In the 'clinical outcomes population', 63 participants (70.8%) receiving SHL, 43 (61.4%) EHL and 36 (62.1%) emicizumab had ≥1 bleed at Year 1. Pain-related events within the past year occurred in 18 (12.8%), 16 (18.2%) and 10 (12.2%) participants, respectively. The most frequent patient-reported symptoms were 'sleep disturbance' (84.5%), 'inability to participate in social roles and activities' (69.7%) and 'fatigue' (66.9%). The most impacted PROMIS scores were anxiety and pain interference.

CONCLUSION: Regardless of the prophylaxis product class used, most participants experienced ≥1 bleeding event per year and reduced QoL. Further treatment optimisation may be needed to prevent bleeding episodes and improve patient QoL.

Journal Title

Haemophilia : the official journal of the World Federation of Hemophilia

Volume

32

Issue

3

First Page

760

Last Page

771

MeSH Keywords

Humans; Hemophilia A; Male; Adult; Quality of Life; Middle Aged; Factor VIII; United States; Treatment Outcome; Female; Young Adult; Adolescent; Antibodies, Monoclonal, Humanized; Cohort Studies; Aged; Antibodies, Bispecific; Child

PubMed ID

41906926

Keywords

Factor VIII; emicizumab; haemophilia A; patient‐reported outcome; real‐world evidence; treatment patterns

Comments

Grants and funding

This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

Publisher's Link: https://onlinelibrary.wiley.com/doi/10.1111/hae.70271

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