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.

Inclusion Criteria: * Patients with relapsed or refractory acute leukemia eligible for menin inhibitor treatment because of a KMT2A or NUP98 rearrangement or an NPM1 mutation. * Patients who received menin inhibitor treatment under a Temporary Authorization for Use (ATU). * Patients younger than 18 years at the time of acute leukemia diagnosis. * Patients who have not objected to the reuse of their data for scientific research. * Patients whose holders of parental authority have already provided authorization for the reuse of the child's data for scientific research. Exclusion Criteria: * Patients who received a Temporary Authorization for Use (ATU) but did not receive the treatment.