Trametinib Safe, Effective for Juvenile Myelomonocytic Leukemia

TUESDAY, June 18, 2024 (HealthDay News) -- For children with relapsed or refractory juvenile myelomonocytic leukemia (JMML), trametinib is safe and effective, according to a study published online June 12 in Cancer Discovery.

Noting that patients with relapsed or refractory (advanced) JMML have poor outcomes, Elliot Stieglitz, M.D., from the University of California San Francisco, and colleagues examined the safety and efficacy of trametinib, an oral MEK1/2 inhibitor, in a phase 2 trial involving 10 patients with advanced JMML.

The researchers found that the objective response rate was 50 percent. After receiving trametinib, four patients with refractory disease proceeded to hematopoietic stem cell transplantation (HSCT). Three additional patients completed all 12 cycles of trametinib permitted in the study and then received trametinib off-protocol, without undergoing HSCT. The remaining three patients had progressive disease; by the end of cycle 2, two of these patients had molecular evolution.

"Our trial offered an option for parents who did not want to subject their children to a repeat HSCT and, in some cases, helped patients avoid HSCT entirely," Stieglitz said in a statement. "The findings suggest that trametinib may be a less toxic alternative to HSCT for select patients."

Two authors disclosed ties to the biopharmaceutical industry; Novartis provided trametinib for the study.

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