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. 2023 Feb 7:14:1103080.
doi: 10.3389/fimmu.2023.1103080. eCollection 2023.

Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience

Gianluca Dell'Orso  1 , Francesca Bagnasco  2 , Stefano Giardino  1 , Filomena Pierri  1 , Giulia Ferrando  3 , Daniela Di Martino  4 , Concetta Micalizzi  5 , Daniela Guardo  5 , Stefano Volpi  6 , Federica Sabatini  7 , Maurizio Miano  5 , Marco Gattorno  6 , Carlo Dufour  5 , Maura Faraci  1
Affiliations

Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience

Gianluca Dell'Orso et al. Front Immunol. .

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents an effective treatment for a variety of inborn errors of immunity (IEI). We report the experience of children affected by IEI who received allo-HSCT over a period of 32 years at IRCCS Istituto Giannina Gaslini, Genoa, Italy. HSCTs were performed in 67 children with IEI. Kaplan-Meier estimates of overall survival (OS) rate at 5 years in the whole group of patients was 83.4% after a median follow-up of 4 years. Median age at transplant was 2.5 years. Eight allo-HSCTs were complicated by either primary or secondary graft failure (GF), the overall incidence of this complication being 10.9%. Incidence of grade 3-4 acute GvHD (aGvHD) was 18.7%, significantly lower in the haploidentical transplant cohort (p = 0.005). Year of transplant (≤2006 vs. >2006) was the main factor influencing the outcome. In fact, a significant improvement in 5-year OS was demonstrated (92.5% >2006 vs. 65% ≤2006, p = 0.049). Frequency of severe aGvHD was significantly reduced in recent years (≤2006 61.5%, vs. >2006 20%, p = 0.027). A significant progress has been the introduction of the TCR αβ/CD19-depleted haploidentical platform, which was associated with the absence of severe aGvHD. However, it was associated with 23.5% incidence of GF. All but one patient experiencing GF in the this specific cohort were successfully retransplanted. In summary, allo-HSCT is confirmed to be an effective treatment for children with IEI, even in the absence of an HLA-matched donor.

Keywords: graft-versus-host disease; haploidentical TCR αβ/CD19+-depleted transplant; hematopoietic stem cell transplantation; inborn errors of immunity; primary immunodeficiency.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Overall survival.
Figure 2
Figure 2
Survival according to HSCT year (A), disease group (B), age at HSCT (C), and donor (D).
Figure 3
Figure 3
Survival according to age at HSCT by year ≤2006 (A) or >2006 (B) and according to type of donor by HSCT year ≤2006 (C) or >2006 (D).
See this image and copyright information in PMC

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