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. 2021 Feb;106(2):196-204.
doi: 10.1111/ejh.13536. Epub 2020 Nov 17.

Haploidentical transplantation in pediatric non-malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH)

Juan Torres Canizales  1   2 , Cristina Ferreras  1   2 , Antonia Pascual  3 , Laura Alonso  4 , Alexandra Regueiro  5 , Mercedes Plaza  6 , José María Pérez Hurtado  7 , Ana Benito  8 , José M Couselo  5 , José L Fuster  6 , Mariana Díaz-Almirón  1 , David Bueno  1 , Yasmina Mozo  1 , Alicia Gómez López  9 , José L Vicario  10 , Antonio Balas  10 , Luisa Sisinni  1 , Cristina Díaz de Heredia  4 , Antonio Pérez-Martínez  1   2   9
Affiliations

Haploidentical transplantation in pediatric non-malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH)

Juan Torres Canizales et al. Eur J Haematol. 2021 Feb.

Abstract

Objective: Describe the GETH haploidentical stem cell transplantation (haplo-HSCT) activity in non-malignant disease (NMDs).

Methods: We retrospectively analyzed data from children with NMDs who underwent haplo-HSCT.

Results: From January 2001 to December 2016, 26 pediatric patients underwent 31 haplo-HSCT through ex vivo T cell-depleted (TCD) graft platforms or post-transplantation cyclophosphamide (PT-Cy) at 7 Spanish centers. Five cases employed unmanipulated PT-Cy haplo-HSCT, 16 employed highly purified CD34+ cells, and 10 employed ex vivo TCD grafts manipulated either with CD3+ CD19+ depletion, TCRαβ+ CD19+ selection or naive CD45RA+ T-cell depletion. Peripheral blood stem cells were the sole source for patients following TCD haplo-HSCT, and bone marrow was the source for one PT-Cy haplo-HSCT. The most common indications for transplantation were primary immunodeficiency disorders (PIDs), severe aplastic anemia, osteopetrosis, and thalassemia. The 1-year cumulative incidence of graft failure was 27.4%. The 1-year III-IV acute graft-versus-host disease (GvHD) and 1-year chronic GvHD rates were 34.6% and 16.7%, respectively. The 2-year overall survival was 44.9% for PIDs, and the 2-year graft-versus-host disease-free and relapse-free survival rate was 37.6% for the other NMDs. The transplantation-related mortality at day 100 was 30.8%.

Conclusion: Although these results are discouraging, improvements will come if procedures are centralized in centers of expertise.

Keywords: T cell-depleted graft; haploidentical hematopoietic stem cell transplantation; high-dose post-transplantation cyclophosphamide; non-malignant diseases; pediatric.

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References

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