loading page

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 Soler,
  • 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

Corresponding Author:juan.torres@salud.madrid.org

Author Profile
Cristina Ferreras
La Paz University Hospital Biomedical Research Foundation
Author Profile
Antonia Pascual
Hospital Regional Universitario Carlos Haya
Author Profile
Laura Alonso
Hospital Universitari Vall d'Hebron
Author Profile
Alexandra Regueiro
Hospital Clinico Universitario de Santiago de Compostela
Author Profile
Mercedes Plaza
Virgen de la Arrixaca University Hospital
Author Profile
JOSE MARIA PEREZ-HURTADO
HOSPITAL UNIVERSITARIO VIRGEN DEL ROCIO
Author Profile
Ana Benito
Hospital Universitario de Salamanca
Author Profile
Jose Couselo
University of Santiago Clinical Hospital
Author Profile
José Luis Fuster Soler
Hospital Clinico Universitario Virgen de la Arrixaca
Author Profile
Miguel Blanquer Blanquer
Hospital Clinico Universitario Virgen de la Arrixaca
Author Profile
Mariana Diaz-Almirón
Hospital Universitario La Paz
Author Profile
David Bueno
Hospital Universitario La Paz
Author Profile
Yasmina Mozo
Hospital Universitario La Paz
Author Profile
Alicia Gómez
Universidad Autonoma de Madrid
Author Profile
José Luis Vicario
Centro de Transfusión de Madrid
Author Profile
Antonio Balas
Centro de Transfusión de la Comunidad de Madrid
Author Profile
Luisa Sisinni
Hospital Universitario La Paz
Author Profile
Cristina Díaz de Heredia
Hospital Universitari Vall d'Hebron
Author Profile
Antonio Pérez-Martínez
HOSPITAL UNIVERSITARIO LA PAZ
Author Profile

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.
21 Oct 2020Published in European Journal of Haematology. 10.1111/ejh.13536