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1
Leniolisib来尼利西布
2
A randomised, placebo-controlled, phase III trial of leniolisib in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS): Adolescent and adult subgroup analysis.来那利西布治疗活化磷脂酰肌醇3-激酶δ(PI3Kδ)综合征(APDS)的随机、安慰剂对照III期试验:青少年和成人亚组分析
Clin Immunol. 2025 Jan;270:110400. doi: 10.1016/j.clim.2024.110400. Epub 2024 Nov 17.
3
Activated PI3K Delta Syndrome活化磷脂酰肌醇-3激酶δ综合征
4
Leniolisib: First Approval.利尼利昔布:首次获批
Drugs. 2023 Jul;83(10):943-948. doi: 10.1007/s40265-023-01895-4.
5
Interim analysis: Open-label extension study of leniolisib for patients with APDS.临时分析:APDS 患者用 leniolisib 的开放标签扩展研究。
J Allergy Clin Immunol. 2024 Jan;153(1):265-274.e9. doi: 10.1016/j.jaci.2023.09.032. Epub 2023 Oct 4.
6
Comparative efficacy of leniolisib (CDZ173) versus standard of care on rates of respiratory tract infection and serum immunoglobulin M (IgM) levels among individuals with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS): an externally controlled study.来那利西布(CDZ173)与标准治疗对活化磷脂酰肌醇3-激酶δ(PI3Kδ)综合征(APDS)患者呼吸道感染率和血清免疫球蛋白M(IgM)水平的疗效比较:一项外部对照研究。
Clin Exp Immunol. 2025 Jan 21;219(1). doi: 10.1093/cei/uxae107.
7
Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA).使用多标准决策分析(MCDA)评估来那利西布在西班牙治疗活化磷脂酰肌醇-3激酶δ综合征(APDS)中的价值贡献。
Glob Reg Health Technol Assess. 2025 Jan 27;12:9-15. doi: 10.33393/grhta.2025.3199. eCollection 2025 Jan-Dec.
8
Effective "activated PI3Kδ syndrome"-targeted therapy with the PI3Kδ inhibitor leniolisib.使用PI3Kδ抑制剂来那度胺进行有效的“活化PI3Kδ综合征”靶向治疗。
Blood. 2017 Nov 23;130(21):2307-2316. doi: 10.1182/blood-2017-08-801191. Epub 2017 Sep 29.
9
APDS patients with immune-complex vasculitis and resolution with leniolisib.患有免疫复合物性血管炎且使用来尼利西布后病情缓解的活化磷脂酰肌醇3激酶δ综合征(APDS)患者。
Clin Immunol. 2024 May;262:110176. doi: 10.1016/j.clim.2024.110176. Epub 2024 Mar 8.
10
Expert insights on Hodgkin's lymphoma development in an activated PI3K delta syndrome patient undergoing leniolisib treatment.对一名接受来尼利西布治疗的活化磷脂酰肌醇-3-激酶δ综合征患者霍奇金淋巴瘤发展情况的专家见解。
Front Immunol. 2025 Jan 13;15:1517543. doi: 10.3389/fimmu.2024.1517543. eCollection 2024.

来尼利西布

Leniolisib

PMID:39083630
Abstract

Leniolisib is an orally administered kinase inhibitor that is used to treat adults and children 12 years of age or older with Activated Phosphoinositide-3-kinase-delta Syndrome (APDS), an extremely rare, inherited immunodeficiency disease. Leniolisib has had very limited clinical use but has not been reported to cause elevations in liver enzymes and has not been linked to episodes of clinically apparent liver injury.

摘要

来尼洛西布是一种口服激酶抑制剂,用于治疗患有活化磷脂酰肌醇-3-激酶δ综合征(APDS)的12岁及以上成人和儿童,APDS是一种极其罕见的遗传性免疫缺陷疾病。来尼洛西布的临床应用非常有限,但尚未有报道称其会导致肝酶升高,也未发现与明显的临床肝损伤发作有关。