如果建好平台，罕见癌症患者就会来：复发/难治性 JMML 的成功临床试验。

If You Build It, Patients with Rare Cancers Will Come: A Successful Clinical Trial in Relapsed and Refractory JMML.

机构信息

Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, Florida.

出版信息

Cancer Discov. 2024 Sep 4;14(9):1574-1576. doi: 10.1158/2159-8290.CD-24-0752.

Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare pediatric hematologic malignancy with a high relapse rate and a poor prognosis hallmarked by RAS pathway mutations. Stieglitz and colleagues conducted a phase II clinical trial using the MEK inhibitor trametinib to treat patients with relapsed and refractory juvenile myelomonocytic leukemia and observed an objective response rate of 50% and an overall survival of 80% after 4 years. See related article by Stieglitz et al., p. 1590 (4) .

摘要

幼年髓单核细胞白血病（JMML）是一种罕见的儿科血液系统恶性肿瘤，具有高复发率和不良预后的特点，其特征是 RAS 通路突变。Stieglitz 及其同事进行了一项 II 期临床试验，使用 MEK 抑制剂 trametinib 治疗复发和难治性幼年髓单核细胞白血病患者，观察到 4 年后客观缓解率为 50%，总生存率为 80%。见 Stieglitz 等人的相关文章，第 1590 页（4）。