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Haploidentical transplantation in pediatric non-malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH)
  • +17
  • juan torres canizales,
  • Cristina Ferreras,
  • Antonia Pascual,
  • Laura Alonso,
  • Alexandra Regueiro,
  • Mercedes Plaza,
  • JOSE MARIA PEREZ-HURTADO,
  • Ana Benito,
  • Jose Couselo,
  • José Luis Fuster SolerOrcid,
  • Miguel Blanquer Blanquer,
  • Mariana Diaz-Almirón,
  • David Bueno,
  • Yasmina Mozo,
  • Alicia Gómez,
  • José Luis Vicario,
  • Antonio Balas,
  • Luisa Sisinni,
  • Cristina Díaz de Heredia,
  • Antonio Pérez-Martínez
juan torres canizales
hospital la paz
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Cristina Ferreras
La Paz University Hospital Biomedical Research Foundation
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Antonia Pascual
Hospital Regional Universitario Carlos Haya
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Laura Alonso
Hospital Universitari Vall d'Hebron
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Alexandra Regueiro
Hospital Clinico Universitario de Santiago de Compostela
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Mercedes Plaza
Virgen de la Arrixaca University Hospital
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JOSE MARIA PEREZ-HURTADO
HOSPITAL UNIVERSITARIO VIRGEN DEL ROCIO
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Ana Benito
Hospital Universitario de Salamanca
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Jose Couselo
University of Santiago Clinical Hospital
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José Luis Fuster Soler
Orcid
Hospital Clinico Universitario Virgen de la Arrixaca
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Miguel Blanquer Blanquer
Hospital Clinico Universitario Virgen de la Arrixaca
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Mariana Diaz-Almirón
Hospital Universitario La Paz
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David Bueno
Hospital Universitario La Paz
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Yasmina Mozo
Hospital Universitario La Paz
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Alicia Gómez
Universidad Autonoma de Madrid
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José Luis Vicario
Centro de Transfusión de Madrid
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Antonio Balas
Centro de Transfusión de la Comunidad de Madrid
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Luisa Sisinni
Hospital Universitario La Paz
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Cristina Díaz de Heredia
Hospital Universitari Vall d'Hebron
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Antonio Pérez-Martínez
HOSPITAL UNIVERSITARIO LA PAZ
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Abstract

Background: We retrospectively analyzed the data of children with non-malignant diseases who have received a haploidentical hematopoietic stem cell transplant (haplo-HSCT). A total of 31 haplo-HSCT were performed in 26 pediatric patients using ex vivo T cell-depleted (TCD) graft platforms or post-transplantation cyclophosphamide (PT-Cy) from January 2001 to December 2016 in 7 Spanish centres. Procedure: A total of five cases were unmanipulated PT-Cy haplo-HSCT, sixteen received highly purified CD34+ cells, ten were ex vivo TCD graft manipulated either with CD3+CD19+ depletion (n= 1), TCΡαβ+CD19+ selection (n= 7) or naive CD45RA+ T cells depletion (n=2). Peripheral blood stem cells were the only source in patients following TCD haplo-HSCT, and bone marrow was the source for one PT-Cy haplo-HSCT. The most common indications for transplant were primary immune deficiency disorders (PIDs) 18, severe aplastic anemia (SAA) 4, osteopetrosis 2 and thalassemia 2. Results: The 1-year cumulative incidence of graft failure was 27.4 %. The 1-year III-IV acute graft versus host disease (aGvHD) and 1-year chronic graft versus host disease (cGvHD) were 34.6% and 16.7% respectively. Besides, the 2-year overall survival (OS) and the 2-year GvHD-free and relapse-free survival (GRFS) were 44.9% for PIDS and 37.6% for the other NMDs. The TRM at day 100 was 30.8%. Conclusions : These results are discouraged and need to be improved to offer a guaranteed treatment for these patients. Improvements will come if procedures are centralized in centres of expertise. The decision between T-cell depletion platforms will depend on the patients’ underlying diseases, comorbidities, and conditioning regimens.