Study design and patients
cerebral thrombosis, death, QT syndrome, convulsion, paralysis, coma
CEREBRAL THROMBOSIS, INTRACRANIAL HEMORRHAGE, ADVERSE EVENTS, CARDIAC EVENTS, CENTRAL NERVOUS SYSTEM LEUKEMIA, MAY, PEDIATRIC APL, COMA, EVENTS, LEUKOCYTOSIS
The SCCLG-APL study was a multicenter, prospective, randomized, non-inferiority trial comparing the efficacy and safety of oral RIF and intravenous ATO in the treatment of pediatric APL. The study received institutional review board approval. Patients’ families and/or patients provided informed consent in accordance with the Declaration of Helsinki. The trial was registered at Patients were recruited from 14 participating hospitals from September 2011 to July 2020. Eligible patients were 16 years old or younger with newly diagnosed APL with confirmation of PML-RARa by reverse-transcription PCR (RT-PCR) assay and (or) fluorescence in situ hybridization. Those were excluded if they had one of the following events occurring before randomization: death from any cause, or coma, convulsion, paralysis due to intracranial hemorrhage, cerebral thrombosis or central nervous system leukemia; or who had prolonged QT syndrome because of the risk of QT interval prolongation during arsenic therapy; or who did not accept randomization.In this open-label trial, patients were randomly assigned 1:1 by computer-generated codes to the ATO or RIF group. The last follow-up time was May 31, 2023. The primary endpoint was 5-year EFS. Secondary end-points were the number of cumulated hospital days and adverse events including incidences of leukocytosis, DS, and cardiac events during induction and consolidation treatment.