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Quantifying the optimal factor VIII levels to achieve patient-centric and clinician-relevant outcomes among people with hemophilia A: a SHELF elicitation study
Burke, Tom Blenkiron, Tom Mancuso, Maria Elisa Khair, Kate O’Mahony, Brian McLaughlin, Paul Mighiu, Claudia
Burke, Tom
Blenkiron, Tom
Mancuso, Maria Elisa
Khair, Kate
O’Mahony, Brian
McLaughlin, Paul
Mighiu, Claudia
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Publication Date
2025-11-03
Submitted Date
2025-06-06
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Abstract
Background: Hemophilia A is an inherited bleeding disorder caused by a deficiency of clotting factor VIII (FVIII), leading to joint bleeding and arthropathy. While prophylactic FVIII therapy reduces bleeding, evidence suggests maintaining higher FVIII levels (FL) may better protect joint health, particularly in physically active individuals and those with joint damage. However, data on optimal FLs required to prevent joint deterioration and complications remains limited.
Research design and methods: This study utilized the Sheffield Elicitation Framework (SHELF) methodology to elicit expert opinions on optimal FLs for patient-centric and clinical outcomes. Five European hemophilia experts participated in virtual workshops, providing probability-based estimates of FLs required to prevent bleed-related hospitalizations, orthopedic procedures, target joint incidence, and support physical activity without additional infusions or joint damage.
Results: Experts consistently recommended higher FLs for individuals with joint damage than for those without. Optimal average FLs ranged from 24% to 51%, exceeding traditionally recommended prophylactic trough levels (3–5%). Considerable uncertainty was noted around FLs for physical activity, reflecting the complexity of individualized care.
Conclusions: Standard prophylaxis regimens may not provide sufficient protection for all patients, particularly those with joint damage. A personalized treatment approach, targeting higher FLs when necessary, may be critical for optimizing outcomes.
Citation
Burke, T., Blenkiron, T., Mancuso, M. E., Khair, K., O’Mahony, B., McLaughlin, P., & Mighiu, C. (2026). Quantifying the optimal factor VIII levels to achieve patient-centric and clinician-relevant outcomes among people with hemophilia A: a SHELF elicitation study. Expert Review of Hematology, 19(1), 55-61. https://doi.org/10.1080/17474086.2025.2574715
Publisher
Taylor & Francis
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Expert Review of Hematology
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Article
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© 2025 Sobi. Published by Informa UK Limited, trading as Taylor & Francis Group.
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ISSN
1747-4086
EISSN
1747-4094
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The HCD Economics research team received funding for this study from Sobi.