Groupe Francophone des Myélodysplasies (GFM), France.
Leuk Res. 2011 Nov;35(11):1444-8. doi: 10.1016/j.leukres.2011.05.034. Epub 2011 Jun 28.
We treated 95 RBC transfusion dependent lower risk MDS with del 5q with Lenalidomide (10mg/day, 3 weeks/4 weeks). Median age was 70.4, median interval from diagnosis 29 months. IPSS was low in 31% and intermediate-1 in 69% patients. Del 5q was isolated, with 1 additional and >1 additional abnormality in 79%, 14%, and 6% patients, respectively. 62 (65%) patients achieved transfusion independence (TI). The only significant factor predicting TI was baseline platelet count >150 G/L and platelet decrease by at least 50% during the first weeks of treatment (p=0.001). Grade III-IV neutropenia and thrombocytopenia were seen in 74% and 37.9% of the cases, respectively, and 3 deaths were attributed to cytopenias. Eight (8%) patients developed deep venous thrombosis (DVT). Platelet decrease by less than 50% predicted a higher risk of DVT. Only 6 patients (6.3%) patients progressed to AML, but median follow-up time was short (18 months). We confirm the high rate of TI with Lenalidomide in lower risk MDS with del 5q. Very close patient monitoring for cytopenias and DVT is mandatory, especially during the first weeks of treatment.
我们用来那度胺（每天 10 毫克，每 4 周的 3 周方案）治疗了 95 例依赖红细胞输注的低危 MDS 伴 del5q。中位年龄为 70.4 岁，从诊断到开始治疗的中位时间间隔为 29 个月。国际预后评分系统（IPSS）为低危的占 31%，中危-1 的占 69%。del5q 为孤立性异常，伴有 1 种以上额外异常的分别占 79%、14%和 6%。62 例（65%）患者达到输血独立性（TI）。唯一能显著预测 TI 的因素是基线血小板计数＞150G/L 和治疗最初几周血小板下降至少 50%（p=0.001）。分别有 74%和 37.9%的患者出现 3-4 级中性粒细胞减少和血小板减少，3 例死亡归因于血细胞减少。8 例（8%）患者发生深静脉血栓形成（DVT）。血小板下降＜50%预测 DVT 风险更高。仅 6 例（6.3%）患者进展为 AML，但中位随访时间较短（18 个月）。我们证实来那度胺治疗低危 MDS 伴 del5q 可获得较高的 TI 率。尤其是在治疗的最初几周，必须对血细胞减少和 DVT 进行密切监测。
