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Single unrelated umbilical cord blood versus unmanipulated haploidentical HCT using PTCy in pediatric AML: a retrospective study on behalf of the EBMT PDWP and CTIWP

Bone Marrow Transplantation volume 60, pages 1635–1641 (2025)Cite this article

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Abstract

Hematopoietic stem cell transplantation (HCT) is the standard consolidation therapy for children with high-risk or relapsed acute myeloid leukemia (AML). In the absence of an human leukocyte antigen (HLA)-matched donor, both unrelated cord blood transplantation (UCBT) and haploidentical HCT with post-transplant cyclophosphamide (haplo-PTCy) serve as viable alternatives. This study analyzed outcomes of 254 pediatric AML patients (CR1/CR2) who underwent either single-unit UCBT (N = 127) without serotherapy or haplo-PTCy (N = 127) between 2011 and 2021. Propensity score weighting was applied to minimize baseline differences. With a median follow-up of 2.9 years, no significant differences were observed in overall survival, leukemia-free survival, relapse incidence, non-relapse mortality (NRM), or graft-versus-host disease (GVHD)-free relapse-free survival. However, UCBT recipients had a lower risk of chronic GVHD (7.1% vs. 19.8%; P = 0.019). These findings confirm comparable survival outcomes between UCBT and Haplo-PTCy. Given the higher risk of cGVHD in haplo-PTCy, further research is needed to refine GVHD prevention strategies while maintaining relapse-free survival. Both approaches remain safe and readily available alternatives for pediatric AML patients lacking matched donors.

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Fig. 1: Weighted survival curves.
Fig. 2: Weighted platelet recovery curves.
Fig. 3: Weighted chronic GVHD curves.

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Author information

Author notes

  1. These authors contributed equally: Krzysztof Kalwak, Annalisa Ruggeri.

Authors and Affiliations

  1. Department of Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

    Benedetta Elena Di Majo, Giovanna Lucchini & Adriana Balduzzi

  2. School of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy

    Benedetta Elena Di Majo & Adriana Balduzzi

  3. EBMT Paris Study Unit, Saint Antoine Hospital, INSERM UMR-S 938, Sorbonne University, Paris, France

    Mouad Abouqateb, Jacques-Emmanuel Galimard & Arnaud Dalissier

  4. Princess Maxima Center/ University Hospital for Children (WKZ), Stem cell transplantation, Utrecht, Netherlands

    Birgitta Versluys

  5. RM Gorbacheva Research Institute, Pavlov University, Saint Petersburg, Russian Federation

    Ludmila Zubarovskaya

  6. Bristol Royal Hospital for Children Dept. of Paediatric Haematology/Oncology/BMT, Bristol, UK

    Oana Mirci-Danicar

  7. CHU Bordeaux Groupe Hospitalier Pellegrin-Enfants, Bordeaux, France

    Charlotte Jubert

  8. Royal Marsden Hospital, Leukaemia Myeloma Units, London, UK

    Sanjay Tewari

  9. Royal Manchester Children’s Hospital, Department of Paediatric Haematology, Bone Marrow Unit, Manchester, UK

    Robert Wynn

  10. Birmingham Children’s Hospital Department of Haematology, Birmingham, UK

    Sarah Lawson

  11. Hopital de la Timone, Département Hématologie Oncologie Pédiatrique, Marseille, France

    Gerard Michel

  12. Service Hématologie Greffe de moelle osseuse, Centre Pierre et Marie Curie, Algiers, Algeria

    Rose-Marie Hamladji

  13. Hôpital A De Villeneuve, Onco-Hématologie pédiatrique, Montpellier, France

    Anne Sirvent

  14. King Faisal Specialist Hospital & Research Centre, Department of Pediatric Hematology/Oncology, Riyadh, Saudi Arabia

    Ali Al-Ahmari

  15. CHU Nantes, Departement D’Hematologie, Nantes, France

    Fanny Rialland

  16. HSCT Unit, Department of Hematology and Oncology, IRCCS Institute G. Gaslini, Genoa, Italy

    Maura Faraci

  17. Department of Women, Child and Adolescent, Onco-Hematology Unit, Geneva University Hospital, Geneva, Switzerland

    Marc Ansari

  18. Cansearch research platform for paediatric oncology and haematology, Department of Paediatrics, Gynaecology and Obstetrics, Faculty of Medicine, University of Geneva, Geneva, Switzerland

    Marc Ansari

  19. Pediatric Immunology and Hematology Department and CRMR aplasies médullaires, Robert Debré Hospital, Groupe Hospitalier Universitaire, Assistance Publique-Hôpitaux de Paris Nord, Université de Paris Cité, Paris, France

    Jean-Hugues Dalle & Charlotte Calvo

  20. TIGITH team, INSERM UMR-1342, Institut de Recherche Saint-Louis, Paris, France

    Charlotte Calvo

  21. Hospital de Clinicas, Hematology, Transfusion and Cell Therapy Service, University of São Paulo, Sao Paulo, Brazil

    Vanderson Rocha

  22. Eurocord APHP, Hospital Saint Louis, Paris, France

    Eliane Gluckman

  23. Monacord Centre Scientifique de Monaco, Monaco, Monaco

    Eliane Gluckman

  24. Department of Pediatric Oncology, Hematology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany

    Katharina Kleinschmidt & Selim Corbacioglu

  25. Department of Pediatric Hematology, Oncology and BMT, Wroclaw Medical University, Wroclaw, Poland

    Krzysztof Kalwak

  26. Haematology and Bone Marrow Transplant Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy

    Annalisa Ruggeri

Authors
  1. Benedetta Elena Di Majo
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  2. Mouad Abouqateb
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  3. Jacques-Emmanuel Galimard
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  4. Arnaud Dalissier
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  5. Giovanna Lucchini
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  6. Birgitta Versluys
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  7. Ludmila Zubarovskaya
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  8. Oana Mirci-Danicar
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  9. Charlotte Jubert
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  10. Sanjay Tewari
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  11. Robert Wynn
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  12. Sarah Lawson
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  13. Gerard Michel
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  14. Adriana Balduzzi
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  15. Rose-Marie Hamladji
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  16. Anne Sirvent
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  17. Ali Al-Ahmari
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  18. Fanny Rialland
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  19. Maura Faraci
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  20. Marc Ansari
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  21. Jean-Hugues Dalle
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  22. Charlotte Calvo
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  23. Vanderson Rocha
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  25. Katharina Kleinschmidt
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  26. Selim Corbacioglu
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  27. Krzysztof Kalwak
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  28. Annalisa Ruggeri
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Contributions

AR and KK designed the study. BEDM wrote the manuscript. MA and JEG performed the statistical analysis. GL critically reviewed the paper. AD, BV, LZ, OMD, CJ, ST, RW, SL, GM, AB, RMH, AS, AAA, FR, MF, MA, JHD, CC, VR, EG, KaK and SC provided cases for the study. All authors edited and approved the manuscript. Full acknowledgement list of participating Centers is available in the Supplementary material.

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Correspondence to Annalisa Ruggeri.

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Di Majo, B.E., Abouqateb, M., Galimard, JE. et al. Single unrelated umbilical cord blood versus unmanipulated haploidentical HCT using PTCy in pediatric AML: a retrospective study on behalf of the EBMT PDWP and CTIWP. Bone Marrow Transplant 60, 1635–1641 (2025). https://doi.org/10.1038/s41409-025-02720-0

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