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

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.