Menin Inhibitors in Pediatric Relapsed or Refractory Acute Leukemia

Acute leukemia is the most common childhood cancer. Despite advances in targeted therapies and immunotherapies, treatment remains challenging for children with relapsed or refractory acute leukemia, particularly those with KMT2A rearrangements, NPM1 mutations, or NUP98 rearrangements. Menin inhibitors have shown promising antileukemic activity in these molecular subgroups. This French national, multicenter, retrospective observational study will describe the pediatric population treated with menin inhibitors for relapsed or refractory acute leukemia. Clinical and biological data will be collected from the SACHA database for patients treated between February 1, 2020, and July 31, 2026. Approximately 10 patients are expected to be included. The study will describe patient and disease characteristics, treatment administration, safety, and efficacy, including remission status, minimal residual disease, relapse, and overall survival.