Investigational treatment options in phase I and phase II trials for relapsed or refractory acute lymphoblastic leukemia in pediatric patients.

: Upfront treatment of pediatric patients with B-cell acute lymphoblastic leukemia (B-ALL) and T-cell acute lymphoblastic leukemia (T-ALL) results in cure rates of 60-95%, depending on risk factors. However, patients with refractory or relapsed B-ALL or T-ALL have much worse outcomes with conventional chemotherapy, hence treatment of these cohorts with novel agents is a priority.: This paper reviews early phase clinical trials in pediatric leukemia.