美国食品药品监督管理局批准摘要：艾曲泊帕用于治疗儿童慢性免疫性（特发性）血小板减少症患者。

U.S. Food and Drug Administration approval summary: Eltrombopag for the treatment of pediatric patients with chronic immune (idiopathic) thrombocytopenia.

作者信息

Ehrlich Lori A, Kwitkowski Virginia E, Reaman Gregory, Ko Chia-Wen, Nie Lei, Pazdur Richard, Farrell Ann T

机构信息

Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.

Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.

出版信息

Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26657. Epub 2017 Jun 19.

DOI:10.1002/pbc.26657

PMID:28627134

Abstract

The U.S. Food and Drug Administration (FDA) approved eltrombopag for pediatric patients with chronic immune (idiopathic) thrombocytopenia (ITP) ages ≥6 on June 11, 2015, and ages ≥1 on August 24, 2015. Approval was based on the FDA review of two randomized trials that included 159 pediatric patients with chronic ITP who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This manuscript describes the basis for approval of these applications. The FDA concluded that eltrombopag has shown efficacy and a favorable benefit to risk profile for pediatric patients with chronic ITP.

摘要

2015年6月11日，美国食品药品监督管理局（FDA）批准艾曲泊帕用于年龄≥6岁的慢性免疫性（特发性）血小板减少症（ITP）儿科患者；2015年8月24日，批准用于年龄≥1岁的患者。该批准基于FDA对两项随机试验的审查，这两项试验纳入了159例对皮质类固醇、免疫球蛋白或脾切除术反应不足的慢性ITP儿科患者。本手稿描述了这些申请获批的依据。FDA得出结论，艾曲泊帕已显示出对慢性ITP儿科患者有效且获益风险比良好。