BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.

IF 2.3 3区医学 Q2 HEMATOLOGY

European Journal of Haematology Pub Date : 2024-12-12 DOI:10.1111/ejh.14362

Rolf Ljung, Anthony K C Chan, Sanjay P Ahuja, Maria Elisa Mancuso, Jose Francisco Cabre Marquez, Florian Volk, Victor Blanchette, Bryce A Kerlin, Sonata Saulyte Trakymiene, Heidi Glosli, Gili Kenet

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引用次数: 0

Abstract

Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.

Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).

Results: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0-1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0-0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was 'good'/'excellent' in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%).

Conclusion: Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.

Trial registration: ClinicalTrials.gov identifier: NCT01311648.

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目的：报告 BAY 81-8973 在 LEOPOLD 儿童推广阶段的长期安全性和有效性：报告 LEOPOLD Kids 延长阶段中 BAY 81-8973 的长期安全性和有效性：患者接受 BAY 81-8973（25-50 IU/kg）治疗，每周至少两次。主要终点是安全性，对所有进入延长阶段的患者（n = 82）进行评估。疗效终点在未使用高滴度抑制剂/免疫耐受诱导的患者（n = 67）中进行评估：结果：儿童患者（n = 82）接受 BAY 81-8973 治疗的时间中位数为 3.1 年/人，接触天数中位数为 405 天/人。BAY 81-8973的长期耐受性良好，在延长阶段没有出现新的抑制剂。所有出血（中位数[IQR]，0.7 [0-1.9]；平均值，1.4 [SD, 2.1]）和关节出血（中位数[IQR]，0 [0-0.7]；平均值，0.5 [SD, 1.1]）在预防后 48 小时内的年化出血率 (ABRs) 均较低（n = 67）。67例患者中有21例（31.3%）在预防性治疗48小时内没有出血；87.9%的出血患者治疗反应为 "良好"/"极佳"，大多数出血在输注BAY 81-8973少于2次后（83.5%）得到缓解：结论：BAY 81-8973的长期治疗耐受性良好，对重症血友病A患儿的所有出血和关节出血均能维持较低的ABR：试验注册：ClinicalTrials.gov identifier：NCT01311648。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

European Journal of Haematology 医学-血液学

CiteScore

5.50

自引率

0.00%

发文量

168

审稿时长

4-8 weeks

期刊介绍： European Journal of Haematology is an international journal for communication of basic and clinical research in haematology. The journal welcomes manuscripts on molecular, cellular and clinical research on diseases of the blood, vascular and lymphatic tissue, and on basic molecular and cellular research related to normal development and function of the blood, vascular and lymphatic tissue. The journal also welcomes reviews on clinical haematology and basic research, case reports, and clinical pictures.