Steven W. Pipe, MD, on Evaluating siRNA Therapy Fitusiran in Combination With Antithrombin Modulation for Hemophilia

Commentary
Video

The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

This interview originally appeared on our sister site, HCPLive®.

"Fitusiran is a highly effective prophylactic agent. The modulation of thrombin generation allows us to achieve effective bleed control with substantially lower doses of factor and bypassing agents, as well as fewer infusions. And this is just an overall better patient experience."

FItusiran, an siRNA therapeutic targeting antithrombin being evaluated as prophylaxis in combination with appropriate bleed management guidelines in the ATLAS-OLE clinical trial (NCT03754790), enabled a lower number of infusions and lower doses of clotting factor concentrates (CFC) or bypassing agents (BPAs) to manage breakthrough bleeding in people with hemophilia A and hemophilia B. Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, recently presented these results at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California.

Pipe and colleagues found that doses per bleed for participants without inhibitors and with inhibitors were reduced during fitusiran prophylaxis as compared to the CFC/BPA prophylaxis period, and overall, participants necessitated fewer infusions for the management of breakthrough bleeds with fitusiran antithrombin-based dosing regimen (with inhibitors, 57; without inhibitors, 148) versus those who received CFC (n = 189) or BPA (n = 465) prophylaxis. CGTLive®'s sister site, HCPLive®, interviewed Pipe at the conference to learn more.

REFERENCE
Pipe SW, Kavakli K, Matsushita T, et al. Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 128.
Recent Videos
Robert Califf, MD, MACC, a cardiologist and former FDA commissioner
Natalie Goedeker, CPNP, on Handling Neuromuscular Gene Therapy at Real-World Sites
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
Michael Flanagan, PhD, chief scientific officer at Avidity
David Barrett, JD, the chief executive officer of ASGCT
David-Alexandre C. Gros, MD, Eledon’s chief executive officer
David Barrett, JD, the chief executive officer of ASGCT
Alfred L. Garfall, MD, MS, associate professor of medicine (hematology-oncology) and director, Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program, Hospital of the University of Pennsylvania; and section chief, Multiple Myeloma, Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania,
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Nirav Shah, MD, MSHP, associate professor of medicine, at the Medical College of Wisconsin
© 2025 MJH Life Sciences

All rights reserved.