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, 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
Stay updated on all scientific advances with Hemostasis Today.
-
May 24, 2026, 17:48Flora Peyvandi: Crucial Role of Placental ADAMTS13 in Perinatal Survival
-
May 24, 2026, 17:13Mahtab Mashayekhi: The Evolving Role of Red Blood Cell Exchange in the Management of SCD
-
May 24, 2026, 17:12Silvina Graña: Helping Families Better Understand Hemophilia and Daily Life With Bleeding Disorders
-
May 24, 2026, 17:11Miikka Vikkula: Key Insights from the ISSVA 2026 World Congress
-
May 24, 2026, 17:10Mathieu Jackson: Proud to Be Present at This Historic Moment for the Global Bleeding Disorders Community
-
May 24, 2026, 17:09Ramina Fazeli: Highlighting the Emerging Role of Neutrophil Extracellular Traps in Ovarian Cancer
-
May 24, 2026, 16:12William Wallace: Normal B12 Levels May Still Cause Brain Damage and Cognitive Decline
-
May 24, 2026, 15:54Marc Carrier: Ottawa Race Weekend Raised Over 7,000 USD for Thrombosis Canada
-
May 24, 2026, 15:44Janak Sadhu: Building a Stroke-Free Mehsana Through Community Awareness and Healthcare Collaboration