Hypomethylating agents (HMA) are indicated in the treatment of higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The combination of hypomethylating agents with venetoclax (Ven) has demonstrated promising results in these diseases, although randomized clinical trials are needed for validation. In this retrospective study, we compared two matched cohorts of patients with MDS or CMML: one receiving oral decitabine-cedazuridine (DEC-C, n = 73) and one receiving DEC-C and Ven (DEC-C-Ven, n = 51), in three contemporary clinical trials. The aim is to determine the impact of the addition of Ven to HMA in MDS and CMML. Individuals were matched using a propensity score approach that was based on the IPSS-M score and age. All patients had excess blasts; 84% were diagnosed with MDS and 16% with CMML. Most patients had high- or very high-risk disease, according to the revised IPSS-R. The overall response rate was superior in the DEC-C-Ven cohort (90% vs 64%, P = 0.002). The median times to best response were 1.1 and 2.7 months for the DEC-C-Ven and DEC-C cohorts, respectively (P < 0.001). More patients underwent hematopoietic stem cell transplantation in the DEC-C-Ven cohort (47%) than in the DEC-C cohort (16%, P < 0.001). The 4- and 8-week mortality did not significantly differ between the DEC-C and DEC-C-Ven cohorts. Patients in the DEC-C-Ven cohort had a more profound neutropenia at days 15 and 21 of the first cycle. The median overall survival was 24 and 19 months for the DEC-C-Ven and DEC-C cohorts, respectively (P = 0.89), and the median event-free survival durations were 18 and 10 months (P = 0.026). In conclusion, the addition of Ven resulted in improved response rates and outcomes in specific subgroups; prospective clinical trials are needed to confirm these findings.
低甲基化剂（HMA）适用于治疗高风险骨髓增生异常综合征（MDS）和慢性粒-单核细胞白血病（CMML）。低甲基化剂与维奈托克（Ven）的联合疗法在这些疾病中显示出有希望的结果，尽管需要随机临床试验进行验证。在这项回顾性研究中，我们比较了三个当代临床试验中两个匹配的MDS或CMML患者队列：一组接受口服地西他滨-cedazuridine（DEC-C，n = 73），另一组接受DEC-C和Ven（DEC-C-Ven，n = 51）。目的是确定在HMA基础上添加Ven对MDS和CMML的影响。个体匹配采用基于IPSS-M评分和年龄的倾向评分方法。所有患者均有原始细胞增多；84%被诊断为MDS，16%为CMML。根据修订的IPSS-R，大多数患者患有高风险或极高风险疾病。DEC-C-Ven队列的总体缓解率更优（90% vs 64%，P = 0.002）。DEC-C-Ven和DEC-C队列达到最佳缓解的中位时间分别为1.1和2.7个月（P < 0.001）。DEC-C-Ven队列中接受造血干细胞移植的患者更多（47%） than in the DEC-C cohort（16%，P < 0.001）。DEC-C和DEC-C-Ven队列之间的4周和8周死亡率无显著差异。DEC-C-Ven队列患者在第一个周期的第15天和21天出现更严重的中性粒细胞减少。DEC-C-Ven和DEC-C队列的中位总生存期分别为24和19个月（P = 0.89），中位无事件生存期分别为18和10个月（P = 0.026）。总之，添加Ven改善了特定亚组的缓解率和结局；需要前瞻性临床试验来确认这些发现。
肿瘤(癌症)患者之家