Adès Lionel, Boehrer Simone, Prebet Thomas, Beyne-Rauzy Odile, Legros Laurence, Ravoet Christophe, Dreyfus François, Stamatoullas Aspasia, Chaury Marie Pierre, Delaunay Jacques, Laurent Guy, Vey Norbert, Burcheri Sara, Mbida Rose-Marie, Hoarau Natacha, Gardin Claude, Fenaux Pierre
Hôpital Avicenne, Université Paris, Paris, France.
Blood. 2009 Apr 23;113(17):3947-52. doi: 10.1182/blood-2008-08-175778. Epub 2008 Nov 5.
Higher-risk MDS with del5q carry a poor prognosis. In this phase 2 trial, 47 patients with higher-risk MDS received lenalidomide 10 mg/day. International Prognostic Scoring System was high in 60%, intermediate-2 risk in 40%. del 5q was isolated, with one additional and more than one additional abnormality in 19%, 23%, and 58% patients, respectively. Thirteen (27%) patients achieved hematologic response, including 7 hematologic complete remission (CR) (with complete [4] or partial [3] cytogenetic response), 2 marrow CR and 4 hematologic improvement erythroid, and 12 became red blood cell (RBC) transfusion independent, for a median duration of 6.5 months. Median CR duration was 11.5 months. Six of 9 (67%) patients with isolated del 5q achieved CR, versus 1 of 11 and none of 27 patients with one or more than one additional abnormality, respectively (P < .001). Seven of 20 (35%) with initial platelets more than 100,000/mm(3) obtained CR, compared with none of the 27 with lower platelet counts less than 100,000/mm(3) (P = .001). Our data support a potential role of lenalidomide in higher-risk MDS with isolated del 5q.
伴有5号染色体长臂缺失的高危骨髓增生异常综合征（MDS）预后较差。在这项2期试验中，47例高危MDS患者接受了来那度胺10毫克/天的治疗。国际预后评分系统显示，60%为高危，40%为中-2级风险。5号染色体长臂缺失单独存在、伴有一种额外异常和伴有一种以上额外异常的患者分别占19%、23%和58%。13例（27%）患者获得血液学缓解，包括7例血液学完全缓解（CR）（伴有完全[4例]或部分[3例]细胞遗传学缓解）、2例骨髓CR和4例红系血液学改善，12例患者不再依赖红细胞（RBC）输血，中位持续时间为6.5个月。CR的中位持续时间为11.5个月。9例单独存在5号染色体长臂缺失的患者中有6例（67%）达到CR，而伴有一种额外异常的11例患者中有1例达到CR，伴有一种以上额外异常的27例患者中无1例达到CR（P< .001）。初始血小板计数超过100,000/mm³的20例患者中有7例（35%）获得CR，而血小板计数低于100,000/mm³的27例患者中无1例获得CR（P = .001）。我们的数据支持来那度胺在单独存在5号染色体长臂缺失的高危MDS中的潜在作用。
