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Vol. 42. Issue S2.
Pages 74-75 (November 2020)
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Vol. 42. Issue S2.
Pages 74-75 (November 2020)
123
Open Access
PERIOPERATIVE MANAGEMENT OF PATIENTS WITH HEMOPHILIA RECEIVING FITUSIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC TARGETING ANTITHROMBIN FOR THE TREATMENT OF HEMOPHILIA
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A.N.L. Prezottia, K.J. Pasib, C. Négrierc, M.V. Ragnid, P. Georgieve, T. Lissitchkovf, K. Salimg, B. Meig, S. Anderssong
a Centro de Hemoterapia e Hematologia do Espírito Santo (HEMOES), Vitória, ES, Brazil
b Royal London Hospital Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom
c Unité d’Hémostase Clinique, Centre Régional de Traitement de l’Hémophilie, Hôpital Louis Pradel, Lyon, France
d Department of Medicine and Hemophilia Center of Western Pennsylvania, University of Pittsburgh, Pittsburgh, United States
e University Multiprofile Hospital for Active Treatment Sveti Georgi and Medical University Plovdiv, Plovdiv, Bulgaria
f Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria
g Sanofi, Cambridge, United States
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Aims: To describe the hemostatic management of patients with hemophilia A or B who underwent surgical procedures while receiving fitusiran prophylaxis. Methods: Fitusiran was evaluated in a phase 1 dose-escalation study (NCT02035605) followed by a phase 2 open-label extension (OLE) study (NCT02554773) that included patients with hemophilia A or B, with or without inhibitors. Patients who were eligible to continue in the phase 2 Open-Label Extension study received monthly fixed subcutaneous doses of fitusiran 50mg or 80mg. Data on perioperative hemostatic therapies and hemostatic response were collected for patients undergoing surgical procedures who had lowered anti-thrombin (AT) during the study. Results: In addition to 5 previously presented surgical procedures, 1 total knee replacement and 1 total hip replacement were performed. Further details on perioperative treatment regimens and hemostatic responses in all 7 procedures will be presented. Conclusions: Successful perioperative hemostatic management of patients in the context of AT lowering with fitusiran has now been observed in several orthopedic, thoracic, gastrointestinal, maxillofacial, and dental surgical procedures. Data first presented at ISTH 2020, 12th-14th July 2020. Study funded by Alnylam and Sanofi.

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Hematology, Transfusion and Cell Therapy
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