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Outcomes of hematopoietic stem cell transplantation in pediatric acute lymphoblastic leukemia: a multicenter Brazilian cohort study

Bone Marrow Transplantation (2025)Cite this article

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Abstract

Acute leukemias are prevalent among children and adolescents, with survival rates exceeding 90%, but high-risk cases often require hematopoietic stem cell transplantation (HSCT). Limited data are available on HSCT outcomes for pediatric acute lymphoblastic leukemia (ALL) in low- and middle-income countries. This Brazilian multicenter retrospective cohort study analyzed outcomes of ALL patients aged up to 18 years who underwent HSCT from January 2010 to June 2020. A total of 439 patients were included (69% male, 78.9% B-ALL). Unrelated donor transplants accounted for 49.9%, and haploidentical transplants for 18.9%. Bone marrow (82.6%) was the predominant stem cell source. Transplants occurred in the first or second complete remission (CR) in 38.7% and 44.2% of cases, respectively. The 100-day cumulative incidence of acute GvHD was 46.1%, and chronic GvHD after 100 days occurred in 25.5%. At five years, overall survival and event-free survival were 52.4% and 51.7%. By day +100, sepsis was the leading cause of mortality (42.3%), whereas relapse accounted for 76.3% of deaths thereafter. Findings align with HSCT literature, demonstrating similar outcomes despite challenges in developing countries and providing a basis for further research.

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Fig. 1: Overall survival curve of the 439 participants enrolled in the study.
Fig. 2: Cumulative incidence of relapse-related mortality (RM) and non-relapse-related mortality (NRM).
Fig. 3: Overall survival (OS) and event-free survival (EFS) analyzed by donor type, disease status, and measurable residual disease (MRD) status.

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Data availability

The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

We are grateful to individuals who participated in the study. This research was supported by Fundo de Incentivo à Pesquisa do Hospital de Clínicas de Porto Alegre (FIPE-HCPA, grant number: 2021–0041).

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Authors and Affiliations

  1. Programa de Pós Graduação em Saúde da Criança e do Adolescente, Departamento de Pediatria, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

    Daniela Morales, Mario Bernardes Wagner & Liane Esteves Daudt

  2. Laboratório de Pediatria Translacional, Centro de Pesquisa Experimental, Hospital de Clínicas de Porto Alegre, Porto Alegre, Rio Grande do Sul, Brazil

    Daniela Morales, Nathalia Kersting, Ciliana Rechenmacher, Yasmine Monteiro, Mariana Bohns Michalowski & Liane Esteves Daudt

  3. Instituto de Pesquisa Pelé Pequeno Príncipe/Hospital Pequeno Príncipe, Curitiba, Brazil

    Gisele Loth, Adriana Mello Rodrigues, Polliany Roberta Dorini Pelegrina, Cilmara Cristina Kuwahara & Carmem Bonfim

  4. Hospital de Clínicas no Paraná, Universidade Federal do Paraná, Curitiba, Brazil

    Gisele Loth, Adriana Mello Rodrigues & Carmem Bonfim

  5. Hospital Amaral Carvalho, Jaú, São Paulo, Brazil

    Vergílio Antonio Rensi Colturato

  6. Instituto Nacional do Câncer (INCA), Rio de Janeiro, Brazil

    Rita Barbosa Tavares & Simone Lermontov

  7. Hospital de Clínicas de Porto Alegre, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

    Priscila de Oliveira da Silva, Mariana Bohns Michalowski & Liane Esteves Daudt

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  1. Daniela Morales
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Contributions

DM was responsible for writing, selecting, and reviewing articles, as well as editing the database, interpreting results, and formatting. GL, VARC, RBT, SL, AMR, PRDP, CCK, and POS contributed to the construction of the database. NK played a role in editing the database, reviewing the statistical analysis, interpreting results, and formatting. CR was in charge of submitting the work to the ethics committee. YM was involved in building and editing the database. MBW performed and reviewed the statistical analysis and organized the database. MBM, CB, and LED contributed to the study design, project supervision, and final review.

Corresponding author

Correspondence to Liane Esteves Daudt.

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The authors declare no competing interests.

Ethics approval and consent to participate

This study was reviewed and approved by the Local Research Ethics Committee of the Hospital de Clínicas de Porto Alegre and other participating centers (CAAE: 42464621.5.1001.5327, #2021-0035). All methods were performed in accordance with relevant guidelines and regulations. Due to the retrospective nature of this study, the requirement for informed consent was waived by the Ethics Committees of the participating institutions.

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Morales, D., Loth, G., Colturato, V.A.R. et al. Outcomes of hematopoietic stem cell transplantation in pediatric acute lymphoblastic leukemia: a multicenter Brazilian cohort study. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02676-1

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