Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
Gianluca Dell’Orso, Francesca Bagnasco, Stefano Giardino, Filomena Pierri, Giulia Ferrando, Daniela Di Martino, Concetta Micalizzi, Daniela Guardo, Stefano Volpi, Federica Sabatini, Maurizio Miano, Marco Gattorno, Carlo Dufour, Maura Faraci
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.