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

Authors: Nana Adjoa Ben-Crentsil, Eric Padron

Published: 2024-09-04

DOI: 10.1158/2159-8290.cd-24-0752

Source: Full article

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Abstract

Summary: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) .