Assessment of Arsenic Trioxide and All-trans Retinoic Acid for the Treatment of Pediatric Acute Promyelocytic Leukemia: A Report From the Children’s Oncology Group AAML1331 Trial

Mené sur 154 patients pédiatriques atteints d'une leucémie promyélocytaire aiguë à risque standard ou à haut risque de récidive (âge médian : 14,4 ans), cet essai non randomisé évalue l'efficacité, du point de vue de la survie sans événement 2 ans après le diagnostic, et la toxicité d'un traitement à base d'acide tout-trans rétinoïque (ATRA) et de trioxyde d'arsenic

Résumé en anglais

Importance : All-trans retinoic acid (ATRA) and arsenic trioxide therapy without the use of maintenance therapy has been found to be beneficial for the treatment of adults with standard-risk acute promyelocytic leukemia (APL). However, it is unclear whether similar regimens are safe and beneficial for the treatment of high-risk APL or pediatric patients with standard-risk APL.

Objective: To assess whether treatment with an ATRA and arsenic trioxide–based regimen is safe and allows for the elimination or substantial reduction of chemotherapy use among pediatric patients with standard-risk or high-risk APL, respectively.

Design, Setting, and Participants : The Children’s Oncology Group AAML1331 study is a nonrandomized, noninferiority trial that examined survival outcomes among 154 pediatric patients with APL compared with a historical control group of patients with APL from the AAML0631 study. Patients aged 1 to 21 years were enrolled at 85 pediatric oncology centers (members of the Children’s Oncology Group) in Australia, Canada, and the US from June 29, 2015, to May 7, 2019, with follow-up until October 31, 2020. All patients had newly diagnosed APL and were stratified into standard-risk APL (white blood cell count <10 000/