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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.

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.

Fecha de publicación: 01/05/2021 Fecha Ahead of Print: 10/12/2020

Autores de IIS La Fe

Bienvenida Argiles Aparicio

Autor

Participantes ajenos a IIS La Fe

Murillo-Sanjuán L
González-Vicent M
Badell Serra I
Rodríguez Villa A
Uria Oficialdegui ML
López-Duarte M
Beléndez-Bieler C
Sastre Urgelles A
Sevilla Navarro J
Diaz-de-Heredia C

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.

Datos de la publicación

ISSN/ISSNe:: 0268-3369, 1476-5365
Tipo:: Article
Páginas:: 1213-1216
DOI:: 10.1038/s41409-020-01172-y
PubMed:: 33303901
Factor de Impacto:: 0,998 SCImago ℠
Cuartil:: Q2 SCImago ℠

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Filiaciones

Murillo-Sanjuán L:: Department of Pediatric Oncology and Hematology, Hospital Universitario Vall d'Hebron, Barcelona, Spain
González-Vicent M:: Department of Pediatric Hemato-Oncology, Hospital Infantil Universitario Niño Jesus, Madrid, Spain
Argilés Aparicio B:: Pediatric Hematology Unit, Hospital Universitario y Politécnico La Fe, Valencia, Spain
Badell Serra I:: Pediatric Hematopoietic Transplant Unit, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
Rodríguez Villa A:: Pediatric Hematology and Oncology Unit, Hospital Universitario Reina Sofía, Córdoba, Spain
Uria Oficialdegui ML:: Department of Pediatric Oncology and Hematology, Hospital Universitario Vall d'Hebron, Barcelona, Spain
López-Duarte M:: Hematology Department, Hospital Universitario Marqués de Valdecilla, Santander, Spain
Beléndez-Bieler C:: Pediatric Oncology and Hematology Section, Hospital General Universitario Gregorio Marañón, Madrid, Spain
Sastre Urgelles A:: Pediatric Hematology and Oncology Unit, Hospital Universitario La Paz, Madrid, Spain
Sevilla Navarro J:: Department of Pediatric Hemato-Oncology, Hospital Infantil Universitario Niño Jesus, Madrid, Spain
Diaz-de-Heredia C:: Department of Pediatric Oncology and Hematology, Hospital Universitario Vall d'Hebron, Barcelona, Spain.