Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab : Results From the Phase IV HemiNorth 2 Study
(2025) In Haemophilia- Abstract
Introduction: Despite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL). Aim: HemiNorth 2 (EudraCT# 2020-003256-32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries. Methods: Following completion of the HemiNorth non-interventional study (NIS), eligible participants (aged ≥ 12–61 years) were enrolled in HemiNorth 2. The primary endpoint was health-related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary... (More)
Introduction: Despite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL). Aim: HemiNorth 2 (EudraCT# 2020-003256-32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries. Methods: Following completion of the HemiNorth non-interventional study (NIS), eligible participants (aged ≥ 12–61 years) were enrolled in HemiNorth 2. The primary endpoint was health-related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary endpoints included PA (International Physical Activity Questionnaire-Short Form [IPAQ-SF]), treatment preference (Emicizumab Preference [EmiPref] survey), joint health, model-based annualised bleeding rates (ABRs) and adverse events. Results: Overall, 28 physically active male PwSHA were enrolled. Most baseline CATCH domains were ≤ 25 and remained consistent; mean treatment burden considerably improved from baseline for adults (−17.8) and adolescents (+16.7). IPAQ-SF scores were consistent throughout the study. Overall, 23 of 25 (92.0%) EmiPref respondents preferred emicizumab over FVIII prophylaxis. Model-based ABRs for treated bleeds decreased from 5.9 (95% confidence interval [CI]: 3.8–9.1) to 1.6 (95% CI: 0.9–3.0) from the NIS to HemiNorth 2, and participants with zero treated bleeds increased from 8 (28.6%) to 16 (57.1%). No new safety signals were reported. Conclusions: Emicizumab improved treatment burden and was preferred by most participants over FVIII prophylaxis. PA levels were consistently high, and bleeding rates improved with emicizumab versus prior FVIII prophylaxis.
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- author
- Astermark, Jan LU ; Ranta, Susanna ; Myrin-Westesson, Linda ; Hoffmann, Marianne ; Quere, Sandrine ; Sanchez, Amparo Yovanna Castro ; Robson, Susan ; Czirok, Tünde ; Lassila, Riitta and Holme, Pål André
- organization
- publishing date
- 2025
- type
- Contribution to journal
- publication status
- epub
- subject
- keywords
- emicizumab, exercise, factor VIII, haemophilia A, physical activity
- in
- Haemophilia
- publisher
- John Wiley & Sons Inc.
- external identifiers
-
- scopus:105015207976
- pmid:40898745
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.70121
- language
- English
- LU publication?
- yes
- id
- 2db715bf-f16d-40b8-b405-4f65797aaf3f
- date added to LUP
- 2025-11-14 12:00:19
- date last changed
- 2025-11-14 12:01:37
@article{2db715bf-f16d-40b8-b405-4f65797aaf3f,
abstract = {{<p>Introduction: Despite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL). Aim: HemiNorth 2 (EudraCT# 2020-003256-32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries. Methods: Following completion of the HemiNorth non-interventional study (NIS), eligible participants (aged ≥ 12–61 years) were enrolled in HemiNorth 2. The primary endpoint was health-related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary endpoints included PA (International Physical Activity Questionnaire-Short Form [IPAQ-SF]), treatment preference (Emicizumab Preference [EmiPref] survey), joint health, model-based annualised bleeding rates (ABRs) and adverse events. Results: Overall, 28 physically active male PwSHA were enrolled. Most baseline CATCH domains were ≤ 25 and remained consistent; mean treatment burden considerably improved from baseline for adults (−17.8) and adolescents (+16.7). IPAQ-SF scores were consistent throughout the study. Overall, 23 of 25 (92.0%) EmiPref respondents preferred emicizumab over FVIII prophylaxis. Model-based ABRs for treated bleeds decreased from 5.9 (95% confidence interval [CI]: 3.8–9.1) to 1.6 (95% CI: 0.9–3.0) from the NIS to HemiNorth 2, and participants with zero treated bleeds increased from 8 (28.6%) to 16 (57.1%). No new safety signals were reported. Conclusions: Emicizumab improved treatment burden and was preferred by most participants over FVIII prophylaxis. PA levels were consistently high, and bleeding rates improved with emicizumab versus prior FVIII prophylaxis.</p>}},
author = {{Astermark, Jan and Ranta, Susanna and Myrin-Westesson, Linda and Hoffmann, Marianne and Quere, Sandrine and Sanchez, Amparo Yovanna Castro and Robson, Susan and Czirok, Tünde and Lassila, Riitta and Holme, Pål André}},
issn = {{1351-8216}},
keywords = {{emicizumab; exercise; factor VIII; haemophilia A; physical activity}},
language = {{eng}},
publisher = {{John Wiley & Sons Inc.}},
series = {{Haemophilia}},
title = {{Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab : Results From the Phase IV HemiNorth 2 Study}},
url = {{http://dx.doi.org/10.1111/hae.70121}},
doi = {{10.1111/hae.70121}},
year = {{2025}},
}