Fleischmann Maximilian, Jentzsch Madlen, Brioli Annamaria, Eisele Florian, Frietsch Jochen J, Eigendorff Farina, Tober Romy, Schrenk Karin G, Hammersen Jakob Friedrich, Yomade Olaposi, Hilgendorf Inken, Hochhaus Andreas, Scholl Sebastian, Schnetzke Ulf
Klinik für Innere Medizin II, Abteilung für Hämatologie Und Internistische Onkologie, Universitätsklinikum Jena, Jena, Germany.
Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany.
Ann Hematol. 2025 Jan;104(1):285-294. doi: 10.1007/s00277-024-06048-5. Epub 2024 Oct 25.
The combination of venetoclax with hypomethylating agents is currently the standard of care for elderly patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy. Despite its favorable efficacy, clinical use is often associated with post-remission cytopenia, frequently necessitating treatment delays and dose modifications. This study aims to evaluate the efficacy and safety of shortened venetoclax treatment durations. A multicenter analysis was conducted involving 20 adult AML patients receiving venetoclax (7 or 14 days with 9 and 11 patients, respectively) combined with 5-azacitidine (5-7 days) between 2021 and 2024. The cohort included patients from four German academic centers all treated in first line. Outcome measures included bone marrow response, transfusion dependence, overall survival (OS) and progression-free survival (PFS). Median age was 73.5 years, with 70% of patients having secondary AML. Adverse molecular risk was observed in 75% of patients. The overall response rate (ORR) was 100%, with a composite complete remission rate of 78%. No significant differences in response rates were observed between the 7-day and 14-day venetoclax regimens. Median OS for the cohort was 15 months. Infection-related complications were observed in 55% of patients, with severe sepsis in 20% of cases. In this cohort, shortened venetoclax regimens demonstrated efficacy comparable to standard treatment protocols, with a potential reduction in hematologic toxicity. These findings support the individualization of treatment regimens to optimize clinical outcomes while potentially minimizing adverse effects.
维奈克拉与低甲基化药物联合使用目前是不适用于强化化疗的老年急性髓系白血病（AML）患者的标准治疗方案。尽管其疗效良好，但临床使用常与缓解后血细胞减少相关，这常常需要延迟治疗和调整剂量。本研究旨在评估缩短维奈克拉治疗疗程的疗效和安全性。2021年至2024年期间，对20例接受维奈克拉（分别为7天和14天，各9例和11例）联合5-阿扎胞苷（5-7天）治疗的成年AML患者进行了多中心分析。该队列包括来自德国四个学术中心的所有一线治疗患者。观察指标包括骨髓反应、输血依赖、总生存期（OS）和无进展生存期（PFS）。中位年龄为73.5岁，70%的患者患有继发性AML。75%的患者存在不良分子风险。总缓解率（ORR）为100%，综合完全缓解率为78%。7天和14天维奈克拉治疗方案之间的缓解率无显著差异。该队列的中位OS为15个月。55%的患者出现感染相关并发症，20%的病例出现严重脓毒症。在该队列中，缩短的维奈克拉治疗方案显示出与标准治疗方案相当的疗效，且血液学毒性可能降低。这些发现支持治疗方案个体化，以优化临床结局，同时可能将不良反应降至最低。
