如果你建造了它，罕见癌症患者就会前来：针对复发和难治性 JMML 的成功临床试验。

IF 29.7 1区医学 Q1 ONCOLOGY Cancer discovery Pub Date : 2024-09-04 DOI:10.1158/2159-8290.CD-24-0752

Nana Adjoa Ben-Crentsil, Eric Padron

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引用次数: 0

摘要

幼年髓单核细胞白血病（JMML）是一种罕见的儿童血液恶性肿瘤，复发率高，预后差，其特点是RAS通路突变。Stieglitz及其同事进行了一项II期临床试验，使用MEK抑制剂曲美替尼治疗复发和难治性幼年骨髓单核细胞白血病患者，观察到客观反应率为50%，4年后总生存率为80%。参见 Stieglitz 等人的相关文章，第 1590 页 (4) 。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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

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) .

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来源期刊

Cancer discovery ONCOLOGY-

CiteScore

22.90

自引率

1.40%

发文量

838

审稿时长

6-12 weeks

期刊介绍： Cancer Discovery publishes high-impact, peer-reviewed articles detailing significant advances in both research and clinical trials. Serving as a premier cancer information resource, the journal also features Review Articles, Perspectives, Commentaries, News stories, and Research Watch summaries to keep readers abreast of the latest findings in the field. Covering a wide range of topics, from laboratory research to clinical trials and epidemiologic studies, Cancer Discovery spans the entire spectrum of cancer research and medicine.