Phase 2 Study of Risk-Based Treatment for Newly Diagnosed Infant Leukemia Using Chemotherapy and Stem Cell Transplantation Based on Genetic and Disease Response Factors

What this Trial Is About

This clinical trial is a phase 2, open-label, multicenter study focused on infants diagnosed with leukemia. It aims to improve survival rates by tailoring the use of chemotherapy and hematopoietic stem cell transplantation based on genetic features at diagnosis and minimal residual disease MRD levels after treatment. The study also seeks to clarify which patient groups need stem cell transplantation and to understand long-term side effects and outcomes related to treatment in infants. Participants are divided into low, intermediate, and high risk groups based on genetic markers and MRD results. Each group receives a specific treatment plan low risk patients undergo induction chemotherapy followed by several consolidation chemotherapy phases and maintenance therapy intermediate risk patients receive induction and high-risk consolidation chemotherapy followed by maintenance high risk patients receive induction, high-risk consolidation chemotherapy, and then hematopoietic stem cell transplantation. Chemotherapy regimens include various drugs such as prednisolone, dexamethasone, vincristine, daunorubicin, and others, delivered over several weeks, with maintenance chemotherapy lasting about two years. During the study, participants will be closely monitored with assessments of overall survival at three years as the primary outcome, along with secondary measures including event-free survival, transplantation rates, recurrence, and treatment-related death rates up to five years. Side effects and long-term effects will be prospectively collected during and after treatment. The total participation period extends up to five years, allowing for comprehensive follow-up to evaluate treatment impact and safety.

Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia