Pediatric blood & cancer · 2025
Emicizumab in Previously Untreated Patients and Minimally Treated Patients With Hemophilia A: A Comparative Study Between Two International Cohorts.
СтатьяСравнительное исследованиеМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Levy-Mendelovich Sarina, Barhod Tlalit, Budnik Ivan, Lancashire Jonathan, Patel Jesal, Barg Assaf Arie, Avishai Einat, Dardik Rima, Barazani Tami Brutman, Livnat Tami, Motwani Jayashree, Kenet Gili
Аннотация
BACKGROUND: Hemophilia A (HA) is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. Prophylactic FVIII replacement therapy is essential for preventing bleeds, but it carries a risk of inhibitor development, especially in previously untreated and minimally treated patients (PUPs and MTPs, respectively). Emicizumab, a bispecific monoclonal antibody that mimics FVIII function, offers a promising alternative for HA prophylaxis due to its subcutaneous administration and favorable safety profile.
METHODS: This study evaluated the real-world safety and efficacy of emicizumab prophylaxis in two international hemophilia treatment centers (HTC). The first HTC included a cohort of 22 MTPs with severe HA (FVIII <1%) and fewer than five prior FVIII exposure days, while the second HTC enrolled 19 PUPs with severe HA.
RESULTS: The median age at emicizumab prophylaxis initiation was 5 months for the MTPs and 8 months for the PUPs. Patients were followed for a median of 27 and 29 months, respectively. The median time to first bleed was 13 months for MTPs, with a significantly longer time to first bleed noted in the PUPs cohort. Safety outcomes were favorable, with neither intracranial hemorrhage nor anti-emicizumab antibodies reported. Four patients, 18% of all MTPs, developed FVIII inhibitors, all associated with high-risk genetic mutations and prior bleeding events.
CONCLUSIONS: Our findings support the early use of emicizumab as a safe and effective prophylactic strategy in infants with severe HA. However, the observed inhibitor rate underscores the need for ongoing monitoring and research to optimize care, particularly in vulnerable populations.
METHODS: This study evaluated the real-world safety and efficacy of emicizumab prophylaxis in two international hemophilia treatment centers (HTC). The first HTC included a cohort of 22 MTPs with severe HA (FVIII <1%) and fewer than five prior FVIII exposure days, while the second HTC enrolled 19 PUPs with severe HA.
RESULTS: The median age at emicizumab prophylaxis initiation was 5 months for the MTPs and 8 months for the PUPs. Patients were followed for a median of 27 and 29 months, respectively. The median time to first bleed was 13 months for MTPs, with a significantly longer time to first bleed noted in the PUPs cohort. Safety outcomes were favorable, with neither intracranial hemorrhage nor anti-emicizumab antibodies reported. Four patients, 18% of all MTPs, developed FVIII inhibitors, all associated with high-risk genetic mutations and prior bleeding events.
CONCLUSIONS: Our findings support the early use of emicizumab as a safe and effective prophylactic strategy in infants with severe HA. However, the observed inhibitor rate underscores the need for ongoing monitoring and research to optimize care, particularly in vulnerable populations.