Akshat Jain: Turning Real-World Data into Smarter Prophylaxis for Hemophilia A
Akshat Jain, Hematology Pediatric Workforce Task Force at American Society of Hematology, shared on LinkedIn about a recent article he and his colleagues co-authored, adding:
“Excited to share that our paper — A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study – is now published in Advances in Therapy!
Transitioning patients from standard half-life (SHL)
Factor VIII concentrates to extended half-life (EHL) products has relied on trial and error. The PREDICT study set out to change that.
What we did
We developed and prospectively tested a novel clinical risk score built from five real-world phenotypic and biological variables:
- Annualised bleeding rate (ABR)
- Treatment frequency on SHL therapy
- Number of active target joints
- von Willebrand factor (vWF) antigen levels
- Physical activity level
Patients were scored as low, medium, or high risk – and prophylaxis with damoctocog alfa pegol were individualised accordingly, across 14 US centres.
What we found
- 70.6% of evaluable participants achieved a favourable outcome — improved bleeding rates and/or reduced infusion frequency compared to their prior SHL regimen
- Mean annualised bleeding rate fell by 8.01
- Infusion frequency reduced by a mean of 6.9 infusions/month
- 25 active target joints identified at baseline → 96% resolved by study completion
- No participant experienced an increase in monthly infusion frequency
- No new safety signals identified
Why this matters globally
Millions of people with haemophilia A worldwide remain on suboptimal or over-burdensome prophylaxis regimens — not because better options don’t exist, but because clinicians lack a structured, evidence-based tool to guide individualised dosing decisions, particularly when full pharmacokinetic profiling is not feasible.
The PREDICT scoring system offers a practical, reproducible, clinic-ready framework that can be applied without complex PK testing. For patients in resource-limited settings, in community haemophilia treatment centres this has the potential to meaningfully shift the standard of care.
Huge thanks to the Bayer team and co-investigators Robert Sidonio, MD, MSc Fernando Corrales-Medina Jessica Charlet Doris Quon etc. , the patients who gave their time, and the teams at all 14 participating centres.”
Title: A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study
Authors: Doris V. Quon, Miguel Escobar, Lisa Boggio, Fernando F. Corrales-Medina, Akshat Jain, Mark T. Reding, Robert F. Sidonio Jr., Janice M. Staber, Jessica Charlet
Read the Full Article on Advanced in Therapy
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