Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo‐controlled, double‐blind, parallel‐group study
Fostamatinib for the treatment of Japanese patients with primary immune thrombocytopenia: A phase 3, placebo‐controlled, double‐blind, parallel‐group study
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Fostamatinib 治疗日本原发性免疫性血小板减少症患者：一项 3 期、安慰剂对照、双盲、平行组研究
DOI：
10.1111/bjh.18582
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发表时间：
2022
影响因子：
6.5
通讯作者：
R788‐1301 Investigators
中科院分区：
文献类型：
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作者：
Kuwana Masataka;Ito Tomoki;Kowata Shugo;R788‐1301 Investigators
福他替尼是一种脾酪氨酸激酶抑制剂，已在美国、加拿大和一些欧洲国家获批用于治疗慢性原发性免疫性血小板减少症（ITP）。我们进行了一项III期、安慰剂对照、双盲、平行组研究，以评价福他替尼在日本原发性ITP患者中的疗效和安全性。34例患者随机接受福他替尼（n= 22）或安慰剂（n= 12）治疗，剂量为100-150 mg，每日两次，持续24周。在8例（36%）接受福他替尼治疗的患者中观察到稳定应答（第14 - 24周6次访视中≥4次访视时血小板≥50 000/μl），而在安慰剂组无患者中观察到稳定应答（p= 0.030）。在接受福他替尼治疗的10例（45%）患者中观察到总体缓解（第2周至第12周6次访视中≥1次访视时血小板≥50 000/μl），而在接受安慰剂治疗的患者中未观察到总体缓解（p= 0.006）。与安慰剂组患者相比，接受fostamatinib治疗的患者需要急救药物的频率更低，出血症状更少。观察到的不良事件为轻度或中度，可管理。在日本ITP患者中未发现新的安全性信号。
Fostamatinib, a spleen tyrosine kinase inhibitor, has been approved for the treatment of chronic primary immune thrombocytopenia (ITP) in the United States, Canada and some European countries. We conducted a phase 3, placebo‐controlled, double‐blind, parallel‐group study to evaluate the efficacy and safety of fostamatinib in Japanese patients with primary ITP. Thirty‐four patients were randomised to fostamatinib (n= 22) or placebo (n= 12) at 100–150 mg twice a day for 24 weeks. Stable responses (platelet ≥50 000/μl at ≥4 of the 6 visits from weeks 14 to 24) were observed in eight (36%) patients on fostamatinib and in none of the patients on placebo (p= 0.030). Overall responses (platelet ≥50 000/μl at ≥1 of the 6 visits from weeks 2 to 12) were seen in 10 (45%) patients on fostamatinib and in none of the patients on placebo (p= 0.006). Patients on fostamatinib required rescue medication less often and experienced fewer bleeding symptoms than patients on placebo. Adverse events observed were mild or moderate and were manageable. No new safety signals were identified in Japanese patients with ITP.