Kuo-Kai Chin, Andriy Derkach, Christopher Famulare, Gaurav K Gupta, P Dayand Borge, Mark B Geyer, Aaron D Goldberg, Tamanna Haque, Jae H Park, Lindsey E Roeker, Martin S Tallman, Maximilian Stahl, Eytan M Stein
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引用次数: 0
摘要
低甲基化药物（HMA）和venetoclax（VEN）常用于不符合诱导化疗条件的IDH突变（IDHm）急性髓性白血病（AML）患者。虽然之前的研究表明，HMA/VEN 在 IDHm AML 中的反应率和存活率都很高，但治疗方法的调整在现实世界中的影响尚不明确。我们回顾性研究了2018年1月至2023年6月期间接受HMA/VEN治疗的89例IDHm AML患者。新诊断（ND）患者的CR/CRi率为76%，复发/难治（R/R）患者的CR/CRi率为55%，中位总生存期分别为29.2个月（ND）和17.1个月（R/R）。治疗调整很常见。早期减少VEN与较低的反应率有关，但并不影响生存。延长周期与较差的反应率或生存率无关。在CR/CRi前后，显著的中性粒细胞减少和急诊就诊或计划外住院的情况相当多，但之后发热性中性粒细胞减少的情况有所减少。HMA/VEN疗效显著，治疗方法的调整不会影响生存率，但长期毒性反应明显。
HMA/VEN treatment modifications and associated outcomes in IDH-mutant AML.
Hypomethylating agents (HMA) and venetoclax (VEN) are commonly used in patients with IDH-mutated (IDHm) acute myeloid leukemia (AML) ineligible for induction chemotherapy. While prior studies demonstrated high response and survival rates with HMA/VEN in IDHm AML, the impact of treatment modifications in real-world settings is unclear. We retrospectively reviewed 89 IDHm AML patients treated with HMA/VEN from January 2018 to June 2023. CR/CRi rates were 76% in newly diagnosed (ND) and 55% in relapsed/refractory (R/R) patients, and median overall survival was 29.2 months (ND) and 17.1 months (R/R), respectively. Treatment modifications were common. Early VEN reductions were associated with lower response rates but not worse survival. Prolonged cycles were not associated with worse response rates or survival. Significant neutropenia and ED visits or unplanned hospitalizations were considerable before and after CR/CRi, though febrile neutropenia decreased afterward. HMA/VEN is efficacious, with treatment modifications not affecting survival, though long-term toxicities are notable.
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Leukemia & Lymphoma in its fourth decade continues to provide an international forum for publication of high quality clinical, translational, and basic science research, and original observations relating to all aspects of hematological malignancies. The scope ranges from clinical and clinico-pathological investigations to fundamental research in disease biology, mechanisms of action of novel agents, development of combination chemotherapy, pharmacology and pharmacogenomics as well as ethics and epidemiology. Submissions of unique clinical observations or confirmatory studies are considered and published as Letters to the Editor
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