Haploidentical transplantation in high‐risk pediatric leukemia: A retrospective comparative analysis on behalf of the Spanish working Group for bone marrow transplantation in children (GETMON) and the Spanish Grupo for hematopoietic transplantation (GETH)

Abstract

A total of 192 pediatric patients, median age 8.6 years, with high-risk hematological malignancies, underwent haploidentical stem cell transplantation (haplo-HSCT) using post-transplantation cyclophosphamide (PT-Cy), or ex vivo T cell-depleted (TCD) graft platforms, from January 1999 to December 2016 in 10 centers in Spain. Some 41 patients received an unmanipulated graft followed by PT-Cy for graft-vs-host disease (GvHD) prophylaxis. A total of 151 patients were transplanted with CD3-depleted peripheral blood stem cells (PBSCs) by either CD34 + selection, CD3 + CD19 + depletion, TCRαβ + CD19 + depletion or CD45RA + depletion, added to CD34 + selection for GvHD prophylaxis. The PBSCs were the only source in patients

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

“…This study reports the accumulated experience of all Spanish centers performing haplo‐HSCTs for the pediatric population with NMDs, which corresponds to 13.9% of the cumulative activity of haplo‐HSCTs 30 . In our study, the 2‐year OS of 44.9% was lower than that in our reported series on malignant disorders (55.1%) 26 and that recently reported from various groups in NMDs, which also differed according to the diagnosis 2,12,28,29,36‐39 .…”

“…Our group (the Spanish Working Group for Hematopoietic Transplantation) reported consistent results from the largest analysis of clinical outcomes in children when comparing the two platforms in pediatric hematologic malignancies. Our study showed 2‐year relapse‐free and GvHD‐free rates of 49.2% and 40.4%, respectively, with relapse the main cause of haplo‐HSCT failure 30 . Based on the results of this study, we conducted a retrospective and multicenter study aimed at analyzing the feasibility of haplo‐HSCT, reporting the outcomes of a Spanish cohort of children with NMDs who underwent the procedure, and proposing a shared future strategy for haplo‐HSCT.…”

“…Our study showed 2-year relapse-free and GvHD-free rates of 49.2% and 40.4%, respectively, with relapse the main cause of haplo-HSCT failure. 30 Based on the results of this study, we conducted a retrospective and multicenter study aimed at analyzing the feasibility of haplo-HSCT, reporting the outcomes of a Spanish cohort of children with NMDs who underwent the procedure, and proposing a shared future strategy for haplo-HSCT.…”

Influence of KIR and NK Cell Reconstitution in the Outcomes of Hematopoietic Stem Cell Transplantation

“…Cooley et al observed that patients with AML with KIR-B/x donors experienced a 30% improvement in RFS compared with those with A/A donors ( 40 ). Subsequently, many further investigations confirmed this beneficial effect of the KIR-B haplotype on relapse and survival in patients with hematological malignancies ( 50 , 51 , 57 , 67 , 72 , 76 , 79 , 81 , 85 , 88 – 92 , 96 , 98 , 101 – 104 ). Five of these studies reported that the protection effects mainly existed in the KIR Cen-B locus ( 67 , 88 , 91 , 92 , 96 ).…”

“…In addition to the technique of adoptive transfer, many studies have analyzed the effects of innate donor-recipient NK cell alloreactivity on GVHD in a clinical setting. The majority of studies did not report a significant association between these parameters ( 41 – 44 , 46 , 47 , 50 , 51 , 54 – 56 , 59 , 65 , 66 , 79 , 81 , 83 , 87 – 89 , 91 – 93 , 97 , 98 , 102 , 104 ), while some reported a protective effect ( 70 , 74 , 76 ). Moreover, several studies found that KIR ligand mismatch or receptor-ligand mismatch increased the risk of GVHD ( 45 , 57 , 60 , 64 , 68 , 80 ).…”