Outcomes of hematopoietic stem-cell transplantation in infants with KMT2A-rearranged acute lymphoblastic leukemia in first remission: a systematic review and single-arm meta-analysis
DOI:
https://doi.org/10.46765/2675-374X.2025v7n1e345Keywords:
Acute lymphoblastic leukemia, Infant, Stem cell transplantation, KMT2AAbstract
Infants with acute lymphoblastic leukemia (ALL) frequently experience poor outcomes, with KMT2A gene rearrangements (KMT2A-r) serving as the most significant adverse prognostic factor. Allogeneic hematopoietic stem cell transplantation (HSCT) in first complete remission (CR1) has been utilized to improve survival, yet its definitive role remains a subject of intense debate. This systematic review and meta-analysis aimed to evaluate survival outcomes of HSCT in infants with KMT2A-r ALL in CR1. We systematically searched PubMed, Embase, and the Cochrane Library for randomized and observational studies reporting survival outcomes for infants with KMT2A-r ALL undergoing HSCT in CR1. Survival data were synthesized using parametric modeling (Weibull distribution) to estimate pooled outcomes. Mean survival across 1,000 iterations per study was calculated with 95%CI. Heterogeneity was assessed using IÇ statistics. All analyses were performed using R software (version 4.1.3). Seven studies comprising 371 patients were included. The pooled analysis demonstrated a 4-year overall survival (OS) of 65% (95%CI 58.7-72.1), a 4-year disease-free survival of 57.5% (95%CI 51.8-64.3), and a 3-year event-free survival (EFS) of 53.2% (95%CI 41.5-66.1). Our findings suggest that HSCT in CR1 is a viable therapeutic strategy for selected high-risk infants with KMT2A-r ALL, showing improved survival compared to historical benchmarks. While novel and safer therapies are currently under investigation, HSCT remains a reasonable option. Clinical decisions should carefully balance the curative potential of transplantation against the risk of severe treatment-related toxicities and long-term sequelae.
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Copyright (c) 2026 Bianca Lisa de Faria, Áurea Maria Salomão Simão, Pedro Henrique Aquino Gil de Freitas, Ana Beatriz Bertol, Amanda Silva Gomes, Barkhá Vijendra, Beatrice Araujo Duarte, Lilian Maria Cristofani
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