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Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children

Bone Marrow Transplantation volume 56, pages 1213–1216 (2021)Cite this article

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Abstract

Hematopoietic stem cell transplantation (HSCT) is currently the only curative option for hematological manifestations in patients with Fanconi anemia (FA). We report the outcome of 34 patients with FA inside a collaborative multicenter national study based on recommendations of Spanish Working Group for Bone Marrow Transplantation in Children (GETMON) between 2009 and 2016. Fludarabine-based conditioning regimen was carried out in all patients, with low dose total body irradiation in unrelated transplants. Disease status before HSCT was bone marrow failure (BMF) in 30 patients and myelodysplastic syndrome (MDS) in four. Donors were matched siblings donors (MSD) in 18, matched unrelated donors (MUD) in 15, and one haploidentical donor. All except one patient engrafted. Cumulative incidence of grades II-IV acute graft-versus-host disease (GVHD) was 29% and 11% for chronic GVHD. Median follow-up after HSCT was 6.5 years. Seven patients (21%) died due to transplant-related causes, two (6%) because of MDS relapse, and one (3%) after a squamous cell carcinoma. Overall survival (OS) was 73% at 5 years post-transplant, with no differences between MSD and MUD transplants. OS for patients with BMF was 80% while for MDS was 25%. Our data suggest HSCT can cure hematologic manifestations of most FA patients with BMF.

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Acknowledgements

We are grateful to Santiago Pérez-Hoyos for his patience performing the statistical analysis and Dr. Lucas Moreno for his contribution revising the manuscript.

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Authors and Affiliations

  1. Department of Pediatric Oncology and Hematology, Hospital Universitario Vall d’Hebron, Barcelona, Spain

    L. Murillo-Sanjuán, M. L. Uria Oficialdegui & C. Diaz-de-Heredia

  2. Department of Pediatric Hemato-Oncology, Hospital Infantil Universitario Niño Jesus, Madrid, Spain

    M. González-Vicent & J. Sevilla Navarro

  3. Pediatric Hematology Unit, Hospital Universitario y Politécnico La Fe, Valencia, Spain

    B. Argilés Aparicio

  4. Pediatric Hematopoietic Transplant Unit, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain

    I. Badell Serra

  5. Pediatric Hematology and Oncology Unit, Hospital Universitario Reina Sofía, Córdoba, Spain

    A. Rodríguez Villa

  6. Hematology Department, Hospital Universitario Marqués de Valdecilla, Santander, Spain

    M. López-Duarte

  7. Pediatric Oncology and Hematology Section, Hospital General Universitario Gregorio Marañón, Madrid, Spain

    C. Beléndez-Bieler

  8. Pediatric Hematology and Oncology Unit, Hospital Universitario La Paz, Madrid, Spain

    A. Sastre Urgelles

Authors
  1. L. Murillo-Sanjuán
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  2. M. González-Vicent
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  6. M. L. Uria Oficialdegui
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  9. A. Sastre Urgelles
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  10. J. Sevilla Navarro
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  11. C. Diaz-de-Heredia
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Consortia

from the Spanish Working Groups for Bone Marrow Transplantation in Children (GETMON), Hematopoietic Transplantation (GETH) and the Spanish Society of Pediatric Hematology and Oncology (SEHOP)

Contributions

LMS wrote the paper, collected and reviewed the data; CDH designed the research, wrote and reviewed the paper; JSN designed the research and reviewed the paper; all others authors reviewed the paper. All authors contributed equally in the data collection, each one for data related to patients from their own center.

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Correspondence to C. Diaz-de-Heredia.

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Murillo-Sanjuán, L., González-Vicent, M., Argilés Aparicio, B. et al. Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children. Bone Marrow Transplant 56, 1213–1216 (2021). https://doi.org/10.1038/s41409-020-01172-y

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