Emicizumab prophylaxis in infants: Single-centre experience.

Autor: Levy-Mendelovich S; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel.; Talpiot Medical Leadership Program, Sheba Medical center, Ramat Gan, Israel., Greenberg-Kushnir N; Department of Paediatric Hematology-Oncology, The Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel., Budnik I; Department of Internal Medicine, Division of Hematology/Oncology, University of Iowa, Iowa City, Iowa, USA., Barg AA; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel., Cohen O; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel.; Department of Medicine and Surgery, University of Insubria, Varese, Italy., Avishai E; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel., Barazani-Brutman T; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel., Livnat T; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel., Kenet G; National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.; Faculty of Medicine, Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Tel Aviv, Israel.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 Apr; Vol. 204 (4), pp. 1375-1382. Date of Electronic Publication: 2024 Jan 24.
DOI: 10.1111/bjh.19312
Abstrakt: The hallmark of haemophilia A (HA) therapy is prophylaxis, aimed at spontaneous bleeding prevention. Emicizumab provides a viable alternative to intravenous factor replacement therapy. However, data on its use in infants are limited. This single-centre open arm prospective study reports on emicizumab prophylaxis in infants. We included severe HA patients under 1 year who started emicizumab prophylaxis since 2018, with longitudinal follow-up. The study collected data on demographics, clinical and laboratory variables, the occurrence of bleeding events, surgeries and treatment outcomes. Of the 27 enrolled infants, whose median age at prophylaxis initiation was 7 months, 24 primarily choose to start emicizumab therapy (3/27 switched from FVIII prophylaxis due to development of FVIII inhibitors). The median age for prophylaxis initiation decreased to 3 months in 2023. Following emicizumab initiation, the median calculated ABR decreased, and no intracranial haemorrhages were observed. Thrombin generation showed a significant improvement in peak height and endogenous thrombin potential at steady state after a loading period. Our study highlights a shift towards early prophylaxis in the era of non-replacement therapies. It underscores the need for continuous evaluation and refinement of treatment approaches, emphasizing personalized care and diligent monitoring in the evolving field of paediatric haemophilia care.
(© 2024 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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