Hemostasis Today
NewsFeed
Headlines Hematology Health Medicine VTE ASH Plasma Donation
October, 2026
October 2026
M T W T F S S
 1234
567891011
12131415161718
19202122232425
262728293031  
Akshat Jain: Turning Real-World Data into Smarter Prophylaxis for Hemophilia A
Akshat Jain/Loma Linda University Health
Mar 30, 2026, 15:26

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

Akshat Jain

Stay updated on all scientific advances with Hemostasis Today.

Akshat Jain Blood Doris V. Quon EHL Fernando F. Corrales-Medina Health Hematology Hemophilia Hemophilia A Hemostasis Hemostasis News Hemostasis Today ISTH Janice M. Staber Jessica Charlet Lisa Boggio Mark T. Reding Medicine Miguel Escobar Robert F. Sidonio Jr. SHL Von Willebrand Factor
NewsFeed