Relapsed or refractory pediatric acute lymphoblastic leukemia: current and emerging treatments
Related Experiment Video
Updated: May 19, 2026

Murine Model of Leukemia Relapse to Induction Chemotherapy for Acute Lymphoblastic Leukemia
Published on: October 17, 2025
Alissa Martin1, Elaine Morgan, Nobuko Hijiya
1Division of HematologyOncologyStem Cell Transplant, Ann Robert H. Lurie Childrens Hospital of Chicago, Chicago, IL 60611, USA.
Insights
Relapsed acute lymphoblastic leukemia (ALL) in children has poor outcomes despite treatment advances. Research focuses on understanding relapse biology and exploring new therapies, including novel agents and hematopoietic stem cell transplantation, to improve remission maintenance.
Area of Science:
- Pediatric Oncology
- Hematology
- Cancer Biology
Background:
- Relapsed acute lymphoblastic leukemia (ALL) is a significant cause of pediatric mortality.
- While survival for newly diagnosed ALL exceeds 85%, approximately 20% of patients relapse, facing poor prognoses.
- Maintaining remission after achieving a second complete remission in relapsed ALL remains a challenge.
Purpose of the Study:
- To review the current understanding of relapsed acute lymphoblastic leukemia (ALL) in pediatric patients.
- To discuss the underlying biology driving relapse and its impact on treatment strategies.
- To explore emerging therapeutic approaches and prognostic factors for relapsed ALL.
Main Methods:
- Literature review focusing on relapsed acute lymphoblastic leukemia (ALL) in pediatrics.
- Analysis of current treatment protocols, including chemotherapy, hematopoietic stem cell transplantation, and novel agents.
- Discussion of prognostic indicators and the biological mechanisms of ALL relapse.
Main Results:
- Outcomes for pediatric patients with relapsed ALL remain poor despite intensive chemotherapy.
- Hematopoietic stem cell transplantation is often considered for high-risk patients due to limited efficacy of conventional chemotherapy alone.
- New drug classes with minimal cross-resistance are under investigation for relapsed ALL treatment.
Conclusions:
- Relapsed ALL in children necessitates continued research into its biology and treatment.
- Novel therapeutic agents and a refined role for hematopoietic stem cell transplantation are crucial for improving outcomes.
- Understanding prognostic factors is essential for tailoring treatment strategies in relapsed pediatric ALL.
Abstract:
Relapsed acute lymphoblastic leukemia (ALL) represents a major cause of morbidity and mortality in pediatrics. With contemporary chemotherapy, >85% of patients with newly diagnosed ALL survive. Unfortunately, 20% of these patients will relapse and for these children, outcomes remain poor despite our best known chemotherapy protocols. Most of these children will achieve a second complete remission, but maintaining this remission remains difficult. Because relapsed ALL is such a significant cause of morbidity and mortality, it is the focus of much research interest. Efforts have been made and continue to focus on understanding the underlying biology that drives relapse. The role of hematopoietic stem cell transplantation in relapsed ALL remains unclear, but many clinicians still favor this for high-risk patients given the poor prognosis with current chemotherapy alone. It is important to use new drugs with little cross-resistance in the treatment of relapsed ALL. New classes of agents are currently being studied. We also discuss prognostic factors and the biology of relapsed ALL.
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