Abstract
Objective: To evaluate the efficacy and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in patients with acquired severe aplastic anemia (SAA), who lacked suitable related or unrelated HLA-matched donors.
Methods: 39 SAA patients underwent haplo-HSCT from Jul 2012 to Jun 2015 at our center. There were 23 males and 16 females at a median follow-up of 11 (range, from 0 to 36) months. The median time from diagnosis to transplantation was 1 (range, from 0.5 to 52) months. The median ages of SAA patients and related haploidentical donor were 23 years (range, 9 to 51years) and 45 (range, from 21 to 61) years, respectively. All patients were given BuCy plus ATG conditioning regimen. GVHD prophylaxis regimen consisted of cyclosporine A (CsA), mycophenolate motetil (MMF), and short-term methotrexate.
Results: Stem cells were collected from bone marrow in 23.08% (n=9) of patients, peripheral blood in 2.56% (n=1), bone marrow plus peripheral blood in 74.36% (n=29) patients. 36 patients received haplo-HSCT combined with the third part of cord blood transfusion 92.31%. The median stem cell dose transplanted was 9.76 (range, from 4.02 to 20.10)×108/kg for mononuclear cells, while 3.4 (range, from 1.05 to 8.60)×108/kg for CD34 cells. 36 patients achieved neutrophil engraftment at a median of 12 (range, from 9 to 28) , and 29 patients achieved platelet engraftment at a median of 29 (range, from 10 to 26) days. Cumulative incidence of III°~IV° acute graft versus host disease (aGVHD) was 8.9±4.9%. 6 patients died of transplant-related mortality (TRM), including 4 from severe infection, and 1 from TMA. The overal survival rate of all patients was 83.2%±6.4%
Conclusions: Haplo-HSCT is likely to be an option for SAA patients without suitable related or unrelated HLA-matched donors, in consideration of the acceptable TRM and severe GVHD incidences.
Disclosures
No relevant conflicts of interest to declare.
COMPARABLE OUTCOMES OF ALLOGENEIC PERIPHERAL BLOOD VERSUS BONE MARROW HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN
