Long-term results of a phase II trial of lenalidomide plus prednisone therapy for patients with myelofibrosis.
Long-term results of a phase II trial of lenalidomide plus prednisone therapy for patients with myelofibrosis.
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DOI：
10.1016/j.leukres.2016.06.007
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发表时间：
2016-09
影响因子：
2.7
通讯作者：
Verstovsek S
中科院分区：
文献类型：
--
作者：
Chihara D;Masarova L;Newberry KJ;Maeng H;Ravandi F;Garcia-Manero G;Ferrajoli A;Cortes J;Kantarjian H;Verstovsek S
来那度胺（联合或不联合泼尼松）是治疗骨髓纤维化 (MF) 患者的积极疗法。经过中位随访 9 年，我们提供了来那度胺加泼尼松治疗 MF 患者的 II 期研究的最新情况。四十名患者参加了这项研究，所有患者都可以进行反应评估。使用 2013 年发布的 IWG 反应标准重新评估了治疗反应：反应质量随着时间的推移而提高，总体反应率为 35%。 39% 的患者出现脾肿大缓解，32% 的患者出现贫血缓解。所有患者的中位治疗失败时间 (TTF) 为 8.2 个月，中位缓解持续时间为 34.6 个月。一些患者的反应非常持久：6 名患者 (15%) 的 TTF 治疗时间超过 60 个月（5 年），3 名患者仍在接受治疗超过 109 个月（9 年）。分别在 1 名和 5 名患者中观察到完全缓解和部分缓解，但实现更深入的缓解对于持久的缓解并不一定必要。需要新的临床研究来探索针对 MF 患者安全且耐受性良好的基于来那度胺的联合策略。
Lenalidomide, with or without prednisone, is an active therapy for patients with myelofibrosis (MF). We provide an update of a phase II study of lenalidomide plus prednisone in patients with MF, after median follow up of 9 years. Forty patients were enrolled in the study and all patients were evaluable for response. Response to the treatment was reevaluated using IWG response criteria published in 2013: quality of response improved over time and overall response rate was 35%. Response in splenomegaly was seen in 39% of patients and anemia response in 32%. The median time to treatment failure (TTF) in all patients was 8.2 months and the median duration of response was 34.6 months. Response was highly durable in some patients: six patients (15%) had TTF for more than 60 months (5 years) and three patients are still on the treatment beyond 109 months (9 years). Complete and partial responses were seen in one and five patients, respectively, but achieving deeper response was not necessary for the response to be durable. New clinical studies are needed to explore safe and well tolerated lenalidomide-based combination strategies for patients with MF.