Abstract
Haploidentical hematopoietic cell transplantation (HCT) is a valuable curative option for children with non-malignant diseases. Haploidentical HCT using post-transplant cyclophosphamide (PTCy) is a readily available option in the absence of an HLA-matched donor. We conducted a retrospective single-center study on the outcome of haploidentical HCT in children with non-malignant diseases. We gathered data from 44 patients underwent HCT in the period 2015 to 2020. The indications for HCT were bone marrow failure, primary immunodeficiency, metabolic disorders, and hemoglobinopathy. Median age at HCT was 4years (range 0.7-20). The conditioning regimens were myeloablative (n = 17) or reduced intensity (n = 27). After a median follow-up of 20months (range 4-71), 2-year overall survival was 89% and 2-year GvHD-free relapse-free survival (GRFS) was 66%. Incidence of primary graft failure was 13.6%. Cumulative incidence of grade II-IV acute and moderate/severe chronic GvHD were 20% and 6.4%, respectively. Younger age at HCT (< 4years) and primary immunodeficiency were significantly associated with better GRFS (p < 0.05). In conclusion, haploidentical HCT using PTCy is feasible and curative in children with non-malignant diseases lacking an HLA-matched donor. Early diagnosis and referral in addition to timely treatment can further improve outcomes.
Highlights
Allogeneic hematopoietic cell transplantation (HCT) is curative for a variety of non-malignant diseases, including primary immunodeficiency (PID), bone marrow failure (BMF), and metabolic disorders.[1,2,3,4,5] finding a donor can be a barrier for HCT; as many patients do not have a suitable HLA-matched related donor
Haploidentical HCT has become an alternative donor source for patients who do not have an HLAmatched donor that is readily available for almost all patients
Bone marrow was used as stem cell source in 18/44 (41%) patients and peripheral blood in 26/44 (59%) patients
Summary
Allogeneic hematopoietic cell transplantation (HCT) is curative for a variety of non-malignant diseases, including primary immunodeficiency (PID), bone marrow failure (BMF), and metabolic disorders.[1,2,3,4,5] finding a donor can be a barrier for HCT; as many patients do not have a suitable HLA-matched related donor. Haploidentical HCT has become an alternative donor source for patients who do not have an HLAmatched donor that is readily available for almost all patients. Graft failure (GF) and graftversus-host-disease (GvHD) are the main barriers for haploidentical HCT.[7,8] In more recent years, T-cell replete haploidentical HCT using post-transplant cyclophosphamide (PTCy) has emerged as a promising strategy, and is being used more frequently due to availability, safety profile, and low cost of the procedure.[9] This approach relies on cyclophosphamide to eliminate both donor and host alloreactive Tcells to reduce the risk of both GF and GvHD.[10,11] hematopoietic stem cells are protected from the cytotoxic effects of PTCy due to higher amounts of aldehyde dehydrogenase, an enzyme responsible for metabolizing the drug.[12,13]
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