Aschauer Gregor, Greil Richard, Linkesch Werner, Nösslinger Thomas, Stauder Richard, Burgstaller Sonja, Fiegl Michael, Fridrik Michael, Girschikofsky Michael, Keil Felix, Petzer Andreas
Internal Medicine I, Medical Oncology, Hematology and Gastroenterology, Bamherzige Schwestern Hospital, Linz, Austria.
Department of Internal Medicine III, Hospital Salzburg-Paracelsus Medical University, Salzburg, Austria.
Clin Lymphoma Myeloma Leuk. 2015 Nov;15(11):e143-9. doi: 10.1016/j.clml.2015.07.645. Epub 2015 Aug 5.
Lenalidomide has demonstrated remarkable efficacy for therapy of lower-risk myelodysplastic syndromes (MDS) associated with 5q(-). The present evaluation aimed to describe the characteristics and outcomes of low-risk MDS patients treated with lenalidomide in Austria.
For this retrospective, multicenter, observational analysis of MDS patients who received lenalidomide, data were collected at various hospitals in Austria over a period of 3 years. MDS classification, previous and current MDS therapies, and outcome and safety of lenalidomide were evaluated.
Forty-six percent of the patients (n = 23) had a 5q(-) syndrome, while 12% (n = 6) exhibited 5q(-) plus additional aberrations or isolated 5q(-) but ≥ 5% blasts in the bone marrow (10%, n = 5). The remaining 32% of patients (n = 16) had MDS with other World Health Organization classifications. Seventy percent belonged to lower International Prognostic Scoring System risk classes. Sixteen centers participated, involving a total of 50 patients. Most frequently used lenalidomide doses were 10 mg and 5 mg on days 1 to 21 of a 28-day cycle. Seventy-five percent of the patients received 11 months of treatment, with a median therapy period of 3.5 months; median follow-up was 3.9 months (range, 0-26 months). Response rate, defined as transfusion independence during the 2 months after lenalidomide therapy, was 64%. Median overall survival was not reached.
Lenalidomide was well tolerated and is an effective and well-tolerated option for therapy of patients with 5q(-) syndrome but also lower-risk MDS patients with other World Health Organization classifications in clinical practice.
来那度胺已被证明对治疗与5q(-)相关的低危骨髓增生异常综合征（MDS）具有显著疗效。本评估旨在描述奥地利接受来那度胺治疗的低危MDS患者的特征和结局。
对于这项接受来那度胺治疗的MDS患者的回顾性、多中心、观察性分析，在奥地利各医院收集了3年期间的数据。评估了MDS分类、既往和当前的MDS治疗方法以及来那度胺的结局和安全性。
46%的患者（n = 23）患有5q(-)综合征，而12%（n = 6）表现为5q(-)加其他异常或孤立的5q(-)但骨髓中原始细胞≥5%（10%，n = 5）。其余32%的患者（n = 16）患有世界卫生组织其他分类的MDS。70%属于较低的国际预后评分系统风险类别。16个中心参与，共涉及50名患者。28天周期的第1至21天最常使用的来那度胺剂量为10 mg和5 mg。75%的患者接受了11个月的治疗，中位治疗期为3.5个月；中位随访时间为3.9个月（范围0 - 26个月）。来那度胺治疗后2个月内定义为无需输血的缓解率为64%。中位总生存期未达到。
来那度胺耐受性良好，对于5q(-)综合征患者以及临床实践中世界卫生组织其他分类的低危MDS患者而言，是一种有效且耐受性良好的治疗选择。
