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CC BY 4.0 · TH Open 2025; 09: a26219749
DOI: 10.1055/a-2621-9749
Original Research

Prospective, Observational Study of the Clinical Outcomes of FVIII Treatment in Adults and Adolescents with Severe Haemophilia A

Pratima Chowdary
1   Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, United Kingdom
2   Department of Haematology, Cancer Institute, University College London, London, United Kingdom
,
Liane Khoo
3   Haemophilia Treatment Centre, Royal Prince Alfred Hospital, Sydney, Australia
,
Michael Wang
4   Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Colorado, United States
,
Hervé Chambost
5   AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France
,
Anthony K.C. Chan
6   Department of Pediatrics, McMaster Children's Hospital, McMaster University, Ontario, Canada
,
Annemieke Willemze
7   Sanofi, Amsterdam, The Netherlands
,
Johannes Oldenburg
8   Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany
› Institutsangaben
Funding This study was funded by Sanofi and Sobi. Medical writing support was funded by Sanofi and Sobi. All authors had access to the primary clinical data and approved the decision to submit the manuscript for publication.
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Abstract

Objective

To assess real-world treatment patterns and outcomes in previously treated patients ≥12 years old with severe haemophilia A treated with marketed factor VIII (FVIII) replacement products.

Methods

Data were collected prospectively between 25 January 2019 and 30 November 2020 across 45 sites in 17 countries. Primary endpoint was annualized bleed rate (ABR). Secondary endpoints included factor consumption, bleed treatment, joint health, and safety. Exploratory endpoints included pain and quality of life outcomes.

Results

A total of 157 patients received ≥1 FVIII injection (prophylaxis n = 139, on-demand n = 19). Mean (standard deviation; SD) observation period was 43.1 (13.3) weeks. Median (quarter [Q]1, Q3) ABR was 2.0 (0.0, 5.7) for those on prophylaxis. Those receiving standard half-life FVIII products or extended half-life FVIII products had a median (IQR) ABR of 2.2 (0.0, 6.1) and 1.3 (0.0, 5.0), respectively. Still, only 35% of patients on prophylaxis experienced zero bleeds and 18% had five or more bleeds in a year. Approximately 23% of bleeding episodes required >1 FVIII dose for resolution. The mean (SD) number of routine prophylaxis injections/week was 2.2 (1.1). Median (Q1, Q3) annualized factor consumption for patients on prophylaxis was 4,106.4 (3,151.6, 5,291.2) IU/kg/year. No changes in Haemophilia Joint Health Score (mean [SD] of 16.1 [19.3] versus 15.7 [17.7]), PROMIS pain intensity 3a T-score (mean [SD] 41.6 [8.2] versus 40.9 [9.1]), or Haem-A-QoL (mean [SD] 30.6 [17.3] versus 29.5 [17.4]) were observed between baseline and the end of the observation period for those using prophylaxis.

Conclusions

Prophylaxis using standard or extended half-life FVIII replacement therapies achieves adequate haemostatic control in only about half of patients, with some experiencing very poor outcomes. Real-world data highlight the urgent need to optimize prophylaxis to enhance haemostatic control, ideally achieving a zero ABR and its associated benefits.

Keywords

observational study - clinical study - haemophilia a - factor VIII - prospective studies

Data Availability Statement

Qualified researchers may request access to patient-level data and related study documents. Patient-level data will be anonymized, and study documents will be redacted, including to protect the privacy of our trial participants. Further details on Sanofi's data-sharing criteria, eligible studies, and process for requesting access can be found at: https://www.vivli.org/.


Authors' Contributions

A.W. was responsible for the study design. P.C., L.K., M.W., H.C., A.K.C.C., A.W., and J.O. contributed to data acquisition. Data analysis and interpretation were carried out by P.C., L.K., M.W., H.C., A.K.C.C., A.W., and J.O. All authors—P.C., L.K., M.W., H.C., A.K.C.C., A.W., and J.O.—were involved in drafting, reviewing, and revising the manuscript.


Supplementary Material



Publikationsverlauf

Eingereicht: 29. Oktober 2024

Angenommen: 15. April 2025

Artikel online veröffentlicht:
17. Juni 2025

© 2025. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)

Georg Thieme Verlag KG
Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

Bibliographical Record
Pratima Chowdary, Liane Khoo, Michael Wang, Hervé Chambost, Anthony K.C. Chan, Annemieke Willemze, Johannes Oldenburg. Prospective, Observational Study of the Clinical Outcomes of FVIII Treatment in Adults and Adolescents with Severe Haemophilia A. TH Open 2025; 09: a26219749.
DOI: 10.1055/a-2621-9749
 

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