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Updated: Nov 23, 2025
08:13
Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
Published on: September 30, 2021
6.9K
Outcome measures in Haemophilia: Beyond ABR (Annualized Bleeding Rate)
Marilyn J Manco-Johnson1, Beth Boulden Warren1, Tyler W Buckner2
1Department of Pediatrics, Hemophilia & Thrombosis Center, University of Colorado and Childrens Hospital Colorado, Aurora, CO, USA.
Summary
New haemophilia treatments require novel outcome measures beyond traditional factor assays. This review highlights the need for diverse assessments, including patient-reported outcomes, to compare evolving therapies effectively.
Area of Science:
- Hematology
- Therapeutic Innovation
- Clinical Trial Design
Background:
- Management of haemophilia is rapidly evolving with novel therapies.
- Traditional factor assays are insufficient for comparing dissimilar treatments.
- Improved prophylaxis has reduced bleeding rates, complicating impact assessment of new therapies.
Purpose of the Study:
- To review and present outcome measures for comparing novel haemophilia therapies.
- To emphasize the strengths and limitations of existing and emerging assessment tools.
- To address the urgent need for a comprehensive panel of outcome measures.
Main Methods:
- Review of existing and emerging outcome measures for haemophilia.
- Discussion of conventional objective measures (joint examination, imaging).
- Evaluation of factor assays, global assays (thrombin generation, factor X activation), and patient-reported outcomes (PROs).
Main Results:
- Conventional objective measures remain important.
- Factor assays are limited for novel non-factor or extended half-life products.
- Patient-reported outcomes (pain, quality of life, participation) are crucial for distinguishing therapy benefits.
Conclusions:
- A diverse panel of outcome measures is essential for comparing novel haemophilia treatments.
- Integrating objective and subjective patient-reported outcomes is necessary.
- Future research should focus on validating and standardizing these measures for comparative trials.
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