Burgstaller Sonja, Stauder Reinhard, Kuehr Thomas, Lang Alois, Machherndl-Spandl Sigrid, Mayrbaeurl Beate, Noesslinger Thomas, Petzer Andreas, Valent Peter, Greil Richard, Thaler Josef
a Department of Internal Medicine IV , Klinikum Wels-Grieskirchen , Wels , Austria.
b Department of Internal Medicine V (Hematology and Oncology) , Innsbruck Medical University , Innsbruck , Austria.
Leuk Lymphoma. 2018 May;59(5):1121-1126. doi: 10.1080/10428194.2017.1369070. Epub 2017 Aug 30.
According to former classification systems chronic myelomonocytic leukemia (CMML) is listed within myelodysplastic syndromes (MDS). Therefore data regarding treatment of CMML is largely derived from MDS trials. Two published studies suggest efficacy of lenalidomide in a proportion of patients with CMML, but the number of patients included was very low. We initiated this phase I trial with lenalidomide in patients with CMML. Primary objective of the current study was to determine the maximum tolerated dose (MTD). Secondary objectives were safety, tolerability and response. Twenty patients were enrolled and received lenalidomide using a classical 3 + 3 design. Lenalidomide 5 mg daily was confirmed as MTD. Best response achieved was partial remission in one patient and stable disease in nine patients. Lack of response with primary progressive disease was seen in three patients. Lenalidomide had a good toxicity profile in this study with thrombocytopenia being the main toxicity observed. Efficacy needs to be confirmed in further trials.
根据以往的分类系统，慢性粒单核细胞白血病（CMML）被归类于骨髓增生异常综合征（MDS）。因此，关于CMML治疗的数据在很大程度上来自MDS试验。两项已发表的研究表明来那度胺对一部分CMML患者有效，但纳入的患者数量非常少。我们启动了这项针对CMML患者的来那度胺I期试验。本研究的主要目的是确定最大耐受剂量（MTD）。次要目的是安全性、耐受性和反应。20名患者入组，并采用经典的3+3设计接受来那度胺治疗。来那度胺每日5mg被确认为MTD。最佳反应为1例患者部分缓解，9例患者病情稳定。3例患者出现原发性进行性疾病无反应。在本研究中来那度胺具有良好的毒性特征，主要观察到的毒性是血小板减少。疗效需要在进一步试验中得到证实。
