Effect of NAC on the Hematopoietic Reconstitution After Haploidentical Hematopoietic Stem Cell Transplantation
Status:
Completed
Trial end date:
2018-12-01
Target enrollment:
Participant gender:
Summary
The aim of the study is to evaluate the efficacy of the prophylactic administration of
N-acetyl-L-cysteine (NAC) in acute leukemia patients with complete remission pre- and
post-allotransplant on the occurrence of poor graft function (PGF) and prolonged isolated
thrombocytopenia (PT) after haploidentical hematopoietic stem cell transplantation.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment of
malignant hematopoietic diseases. However, the delayed hematopoietic reconstitution,
including PGF and PT, remain serious complication after allo-HSCT, and the effective
therapeutic strategies are limited. In murine studies, endothelial cells have been identified
as a key cellular component supporting hematopoietic stem cells in the bone marrow
microenvironment. Our previous prospective nested case-control study suggested that the
frequency of bone marrow endothelial cells was markedly reduced in patients with PGF or PT.
Moreover, our recent study further identified reduced bone marrow endothelial cells (<0.1%)
pre-allotransplant was associated with significant higher incidences of PGF or PT after
allo-HSCT. In addition, NAC treatment in vitro could quantitatively and functionally improve
bone marrow endothelial cells derived from the patients with PGF or PT. Therefore, bone
marrow endothelial cells (<0.1%) pre-allotransplant can be used to identify patients with a
higher incidence of PGF or PT to provide timely prophylactic intervention of NAC to prevent
the occurrence of delayed hematopoietic reconstitution post-transplant. The study hypothesis:
Prophylactic intervention of NAC pre- and post-allotransplant could reduce the incidence of
PGF and PT in acute leukemia patients after haploidentical hematopoietic stem cell
transplantation.