Department of Hematology, Medipol University, Istanbul, Turkey.
Department of Hematology, Uludağ University, Bursa, Turkey.
Clin Lymphoma Myeloma Leuk. 2021 Aug;21(8):e686-e692. doi: 10.1016/j.clml.2021.04.004. Epub 2021 Apr 20.
Venetoclax is a selective B-cell lymphoma 2 (BCL2) inhibitor, which is approved to treat elderly patients with newly diagnosed acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) in combination with either low-dose cytarabine (ARA-C) or hypomethylating agents. We aimed to collect and share data among the efficacy and safety of venetoclax both as a monotherapy or in combination with other drugs used to treat high-risk MDS or AML.
A total of 60 patients with a median age of 67 (30-83) years from 14 different centers were included in the final analysis. Thirty (50%) of the patients were women; 6 (10%) of the 60 patients were diagnosed with high-risk MDS and the remaining were diagnosed with AML.
The best objective response rate (complete remission [CR], complete remission with incomplete hematological recovery (CRi), morphological leukemia-free state [MLFS], partial response [PR]) was 35% in the entire cohort. Best responses achieved during venetoclax per patient number were as follows: 7 CR, 1 CRi, 8 MLFS, 5 PR, and stable disease. Median overall survival achieved with venetoclax was 5 months in patients who relapsed and not achieved in patients who were initially treated with venetoclax. Nearly all patients (86.7%) had experienced a grade 2 or more hematologic toxicity. Some 36.7% of these patients had received granulocyte colony stimulating factor (GCSF) support. Infection, mainly pneumonia (26.7%), was the leading nonhematologic toxicity, and fatigue, diarrhea, and skin reactions were the others reported.
Our real-life data support the use of venetoclax in patients with both newly diagnosed and relapsed high-risk MDS and AML.
维奈托克是一种选择性 B 细胞淋巴瘤 2（BCL2）抑制剂，已被批准与低剂量阿糖胞苷（Ara-C）或低甲基化剂联合用于治疗新诊断的老年急性髓系白血病（AML）和高危骨髓增生异常综合征（MDS）患者。我们旨在收集和分享维奈托克单药或与其他用于治疗高危 MDS 或 AML 的药物联合使用的疗效和安全性数据。
共有 14 个不同中心的 60 名中位年龄为 67 岁（30-83 岁）的患者纳入最终分析。30 名（50%）患者为女性；60 名患者中 6 名（10%）诊断为高危 MDS，其余患者诊断为 AML。
整个队列的最佳客观缓解率（完全缓解[CR]、不完全血液学恢复的完全缓解[CRi]、形态学无白血病状态[MLFS]、部分缓解[PR]）为 35%。维奈托克治疗每位患者的最佳反应如下：7 例 CR、1 例 CRi、8 例 MLFS、5 例 PR 和疾病稳定。复发患者的维奈托克中位总生存期为 5 个月，初治患者未达到该生存期。几乎所有患者（86.7%）均出现 2 级或更高级别的血液学毒性。其中 36.7%的患者接受了粒细胞集落刺激因子（GCSF）支持。感染，主要为肺炎（26.7%），是主要的非血液学毒性，其他还包括疲劳、腹泻和皮肤反应。
我们的真实数据支持维奈托克用于新诊断和复发的高危 MDS 和 AML 患者。
