Related Experiment Video
Updated: Mar 26, 2026

Murine Model of Leukemia Relapse to Induction Chemotherapy for Acute Lymphoblastic Leukemia
Published on: October 17, 2025
[Infant acute leukemia]
Benoît Brethon1, Hélène Cavé2, Mony Fahd1
1Assistance publique-Hôpitaux de Paris, hôpital Robert-Debré, hématologie et immunologie pédiatrique, 48, boulevard Sérurier, 75019 Paris, France.
Insights
Infant acute leukemia, particularly acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), presents unique challenges due to immature cells and treatment toxicities. Novel targeted therapies are urgently needed as conventional treatments show limited success and high risks in infants.
Area of Science:
- Pediatric Oncology
- Hematology
- Cancer Genetics
Context:
- Acute leukemia is rare in infants (<1 year), with similar frequencies of acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML).
- Infant leukemia often features high hyperleukocytosis, significant tumor burden, and extramedullary involvement (CNS, skin).
- Leukemic cells in infants are typically immature, with Mixed Lineage Leukemia (MLL) gene rearrangements being a common hallmark.
Purpose:
- To review the unique characteristics of infant acute leukemia, including its presentation, genetic landscape, and treatment challenges.
- To discuss the limitations of conventional therapies and the associated toxicities in this age group.
- To highlight the urgent need for alternative treatment strategies, such as targeted therapies.
Summary:
- Infant leukemia, characterized by immature cells and MLL rearrangements, exhibits resistance to conventional chemotherapy and corticosteroids.
- Treatment is limited by drug dose-intensity intolerance, risks of radiotherapy, and high rates of infectious complications.
- Survival rates for infant ALL and AML are significantly lower than in older children, necessitating new therapeutic approaches.
Impact:
- Current treatment strategies for infant acute leukemia yield poor outcomes, with survival rates for ALL being notably limited compared to older children.
- Allogeneic stem cell transplantation is reserved for high-risk subgroups but carries significant morbidity and mortality.
- The ethical considerations surrounding treatment toxicity and dismal prognosis underscore the critical need for innovative targeted therapies in infant leukemia.
Abstract:
If acute leukemia is the most frequent cancer in childhood (33%), it remains a very rare diagnosis in infants less than one year old, e.g. less than 5% of cases. At this age, the frequency of acute lymphoblastic leukemia (ALL) (almost all of B-lineage) is quite similar to the one of myeloblastic forms (AML). Infant leukemia frequently presents with high hyperleucocytosis, major tumoral burden and numerous extra-hematological features, especially in central nervous system and skin. Whatever the lineage, the leukemic cell is often very immature cytologically and immunologically. Rearrangements of the Mixed Lineage Leukemia (MLL) gene, located on band 11q23, are the hallmark of these immature leukemias and confer a particular resistance to conventional approaches, corticosteroids and chemotherapy. The immaturity of infants less than 1-year-old is associated to a decrease of the tolerable dose-intensity of some drugs (anthracyclines, alkylating agents) or asks questions about some procedures like radiotherapy or high dose conditioning regimen, responsible of inacceptable acute and late toxicities. The high level of severe infectious diseases and other high-grade side effects limits also the capacity to cure these infants. The survival of infants less than 1-year-old with AML is only 50% but similar to older children. On the other hand, survival of those with ALL is the same, then quite limited comparing the 80% survival in children over one year. Allogeneic stem cell transplantations are indicated in high-risk subgroups of infant ALL (age below 6 months, high hyperleucocytosis >300.10(9)/L, MLL-rearrangement, initial poor prednisone response). However, morbidity and mortality remain very important and these approaches cannot be extended to all cases. During the neonatal period, the dismal prognosis linked to the high number of primary failures or very early relapses and uncertainties about the late toxicities question physicians about ethics. It is an emergency to propose different strategies (targeted therapies) to these infants with acute leukemia as conventional trials failed to improve outcome.
Related Concept Videos
Disorders of Leukocytes
Leukopenia may result from bone marrow disorders, autoimmune diseases, and infectious diseases. For example, conditions such as multiple myeloma and aplastic anemia can impair the bone marrow's ability to produce adequate leukocytes. Similarly, autoimmune diseases like lupus and viral infections such as HIV can prompt the immune...
Acute Pyelonephritis I: Introduction
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
Development of Immunocompetence
The initial cells that migrate from the fetal thymus settle within the skin and epithelial tissues lining the mouth, digestive tract, and in females, the uterus and vagina. These cells, including skin-based dendritic cells, serve as antigen-presenting cells, playing a key role in T cell activation.
Subsequent T...
Pharmacokinetics in Pediatric Patients: Drug Excretion
Immunodeficiency Diseases
There are three main causes of immunodeficiency...

