← 返回前沿论文

新型靶向治疗与细胞免疫治疗使复发/难治性急性髓系白血病患者能够接受异基因造血干细胞移植

英文原题:Novel targeted therapy and cellular immunotherapy enabling allogeneic hematopoietic stem cell transplantation for relapsed/refractory acute myeloid leukemia.

PubMed 2026/08/28(内容时间) Chin Med J (Engl) Q1 · IF 9.1(JCR 2025)

研究概要

复发或难治性(R/R)急性髓系白血病(AML)患者的预后极差。

中文摘要

复发或难治性(R/R)急性髓系白血病(AML)患者预后极差。对于大多数 R/R AML 患者而言,异基因造血干细胞移植(HSCT)是唯一的治愈性选择。当疾病复发时,再次进行突变分析以识别可被新型治疗靶向的新遗传学异常十分重要。作为预处理方案的组成部分或移植后维持治疗,新型靶向药物以多种形式支持 R/R AML 患者的 HSCT,包括联合化疗以提高 HSCT 前的完全缓解(CR)。对于伴 FMS 相关酪氨酸激酶 3(FLT3)突变的 R/R AML 患者,索拉非尼和奎扎替尼无论是联合化疗桥接移植,还是作为 HSCT 后的维持治疗,均显示出令人鼓舞的疗效。分别靶向突变型异柠檬酸脱氢酶(IDH)1 和 2 的抑制剂艾伏尼布和恩西地平,已获美国食品药品监督管理局(FDA)批准用于治疗 IDH1/IDH2 突变的 R/R AML。维奈克拉是一种 B 细胞淋巴瘤-2(BCL2)抑制剂,广泛用于 R/R AML 的挽救治疗,其疗效优于传统化疗且药物毒性可控。此外,嵌合抗原受体(CAR)T 细胞免疫治疗(靶向 CD33、CD123 和 CLL1)在 R/R AML 的 I 期和 II 期临床试验中取得了令人鼓舞的临床缓解率,后续桥接 HSCT 显著改善了患者的生存。

展开英文摘要原文

Patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) are characterized by a disastrous prognosis. For most patients with R/R AML, allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option. It is important to conduct mutational analysis again when the disease relapses to identify any new genetic abnormalities that can be targeted with novel treatments. As part of a conditioning regimen or post-transplant maintenance therapy, new targeted agents support HSCT in patients with R/R AML in various forms, including combination chemotherapy to improve complete remission (CR) prior to HSCT. For R/R AML patients with mutated FMS-related tyrosine kinase 3 (FLT3), sorafenib and quizartinib have shown encouraging therapeutic effects either in combination with chemotherapy for bridging to transplantation or as maintenance therapy after HSCT. Ivosidenib and enasidenib, which are inhibitors that target mutated isocitrate dehydrogenase (IDH) 1 and 2, respectively, have been approved by the US Food and Drug Administration (FDA) for the treatment of IDH1/IDH2-mutated R/R AML. Venetoclax, an inhibitor of B-cell lymphoma-2 (BCL2), is widely used in the salvage treatment of R/R AML and has better therapeutic effects and controllable drug toxicity than traditional chemotherapy. In addition, chimeric antigen receptor (CAR) T-cell immunotherapy (targeting CD33, CD123, and CLL1) has achieved encouraging clinical response rates in phase I and phase II clinical trials for R/R-AML, and subsequent bridging HSCT has significantly improved patient survival.

论文信息

作者
Cao L、Hu D、Cui L、Zhang X
单位
Peking University People's Hospital, Peking University Institute of Hematology, Department of Hematology, Beijing 100000, China.China
期刊
Chinese medical journal2026 Aug 28
原文标识
PubMed 42665995 · DOI 10.1097/CM9.0000000000004212