Abstract
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Since the early 2000s, FVIII prophylaxis has been the standard for severe hemophilia A, but patients with inhibitors who failed immune tolerance induction (ITI) had no effective prophylaxis until emicizumab, which significantly reduced bleeding rates and improved quality of life. A 67-year-old man with severe hemophilia A and high-titer inhibitors, managed previously with bypassing agents, began emicizumab prophylaxis in 2021 and experienced over three years without bleeding, including safe dental procedures. In 2024, he was diagnosed with stage III pancreatic cancer, and despite emicizumab, he had multiple severe bleeding episodes during diagnostic and therapeutic procedures, requiring large doses of rFVIIa; bleeding ceased after stereotactic radiotherapy. This case highlights emicizumab’s transformative impact but also its limitations during major medical interventions, emphasizing the continued need for on-demand treatment and consideration of ITI even in the era of non-factor therapies.
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Authors
Mario Biglietto
Hematology, Department of Translational and Precision Medicine Sapienza University of Rome, Italy
Silvia Linari
Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy
Anna Laura Faccini Hematology, Department of Translational and Precision Medicine Sapienza University of Rome, Italy
Silvia Sorella
Hematology, Department of Translational and Precision Medicine Sapienza University of Rome, Italy
Erica Crisanti
Hematology, Department of Translational and Precision Medicine Sapienza University of Rome, Italy
Marco Antonacci
Hematology, Department of Translational and Precision Medicine Sapienza University of Rome, Italy
Silvio Ligia
Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy
Rosaria Mormile Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy
Roberta Ciciani
Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy
Erminia Baldacci
Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy
Cristina Santoro
Hematology, Department of Translational and Precision Medicine, Sapienza University of Rome, Italy
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