08 Dec Nemours Study Evaluates fitusiran for Bleeding Prophylaxis During Minor Surgery in hemophilia A

Dr. Zorrilla

MedicalResearch.com Interview with:
Julian Zorrilla, DO
Pediatric Hematologist/Oncologist
Nemours Children’s Health

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Non-factor therapies are a novel treatment approach for people with hemophilia A and B with and without inhibitors.

Fitusiran is a first-of-its-kind “rebalancing” agent that provides effective hemostatic control from bleeds in people with hemophilia. This study describes the experience of people with hemophilia who are on fitusiran in regard to minor surgeries. This data shows minor surgeries can be effectively and safely performed for those patients who are on fitusiran for their prophylaxis.  A subset of individuals on fitusiran did not require any other medications to help prevent surgical bleeding.

MedicalResearch.com: What should readers take away from your report?

Response: Fitusiran as a rebalancing agent, and in accordance with breakthrough bleed management guidelines, offers satisfactory bleed protection in cases of minor surgeries. This should provide reassurance of this new therapy’s ability in protecting those with hemophilia from bleeding.      

MedicalResearch.com: What recommendations do you have for future research as a results of this study?

Response: Assessment of fitusiran’s effectiveness in major surgeries and trauma setting. Also performing subset analysis if there were any thrombosis that occurred following surgery.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: The treatment landscape for hemophilia is changing from the traditional factor replacement products that have been around for decades. It is exciting times.

Citation: ASH 2025 Abstract:

Experience with minor surgeries in people with hemophilia A with or without inhibitors receiving fitusiran

Steven Pipe, Tienan Zhu, Kaan Kavakli, Stephanie P’ng, Chur-Woo You, Inga Hegemann, Gili Kenet, Teh-Liane Khoo, Margarita Timofeeva, Guy Young, Laurel Menapace, Abhimanyu Yarramaneni, Yuqian Shen, Umer Khan, Sandra Casiano, Arta Subagiarta, Julian Zorrilla,
Experience with minor surgeries in people with hemophilia A or B with and without inhibitors receiving fitusiran,
Blood, Volume 146, Supplement 1, 2025, Page 4850, ISSN 0006-4971,

https://doi.org/10.1182/blood-2025-4850

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Last Updated on December 8, 2025 by Marie Benz MD FAAD