Duke University School of Medicine, Durham, NC, 27708, USA.
J Hematol Oncol. 2023 Mar 3;16(1):17. doi: 10.1186/s13045-023-01411-x.
Developments in investigational agents and novel regimens in acute myeloid leukemia (AML) were reported in the 2022 American Society of Hematology (ASH) annual meeting. Encouraging efficacy data were presented from first-in-human studies of two investigational menin inhibitors, SNDX-5613 and KO-539, in relapsed and refractory (R/R) acute myeloid leukemia (AML) with KMT2A rearrangement or mutant NPM1, with overall response rates (ORR) of 53% (32/60) and 40% (8/20), respectively. The addition of the novel drug pivekimab sunirine, a first-in-class antibody-drug conjugate targeting CD123, to azacitidine and venetoclax in R/R AML resulted in an ORR of 45% (41/91), which rose to 53% in those who were venetoclax naïve. Additional novel triplet treatment combinations included the addition of magrolimab, an anti-CD47 antibody, to azacitidine and venetoclax, with an ORR of 81% (35/43) in newly diagnosed AML, including an ORR of 74% (20/27) in TP53 mutated AML. The addition of the FLT3 inhibitor gilteritinib to azacitidine/venetoclax was also featured, with an ORR of 100% (27/27) in newly diagnosed AML and an ORR of 70% (14/20) in R/R AML.
在 2022 年美国血液学会（ASH）年会上报告了急性髓系白血病（AML）中研究药物和新方案的进展。首次人体研究中，两种新型 MENIN 抑制剂 SNDX-5613 和 KO-539 在伴有 KMT2A 重排或突变 NPM1 的复发/难治性（R/R）急性髓系白血病（AML）中显示出令人鼓舞的疗效数据，总体缓解率（ORR）分别为 53%（32/60）和 40%（8/20）。新型药物 pivekimab sunirine（一种针对 CD123 的首创抗体药物偶联物）联合阿扎胞苷和 venetoclax 用于 R/R AML，ORR 为 45%（41/91），在 venetoclax 初治患者中升至 53%。其他新型三联治疗组合包括将抗 CD47 抗体 magrolimab 联合阿扎胞苷和 venetoclax，新诊断的 AML 的 ORR 为 81%（35/43），包括 TP53 突变 AML 的 ORR 为 74%（20/27）。还介绍了 FLT3 抑制剂 gilteritinib 联合阿扎胞苷/venetoclax 的疗效，新诊断的 AML 的 ORR 为 100%（27/27），R/R AML 的 ORR 为 70%（14/20）。
