Abstract:

BACKGROUND: Hematopoietic stem cell (HSC) transplantation is an optimal therapeutic method of severe aplastic anemia. The transplantation of various sources-derived HSCs has been performed, including affinity haploid transplantation, unrelated transplantation. However, the reports on curative effects of HSCs combined with MSCs transplantation are individual case.
OBJECTIVE: To retrospectively compare and analyze the curative effect of HSC transplantation and non-transplantation in treatment of severe aplastic anemia.
METHODS: We studied 17 patients with severe aplastic anemia aged 3-53 years from April 2008 to April 2010. In them, 8 patients were subjected to HSC transplantation, and 9 patients were treated with non-transplantation. In the transplantation group, 4 patients received related HLA half-matched, 2 received HLA matched, and 2 received unrelated HSC transplantation. In all patients, 4 of them also received mesenchymal stem cell co-transplantation besides HSC infusion. The 9 patients in non-transplantation group received immunosuppressant and hematopoietic promotion treatment.
RESULTS AND CONCLUSION: One 45-years old patient in the transplantation group had received non-transplantation for 11 months, but no effect was determined; thus, the patient received transplantation when this patient was combined with renal failure and lung fungous infection, and died of transplantation complication. The chromosome and DNA fingerprint detection of the remaining 7 patients indicated that complete donor implantation and rapid recovery of hematopoietic functions following HSC transplantation. The mean time of neutrophil exceeded 0.5×10⁹/L and platelets recovery exceeded 20×10⁹/L were 12 and 14 days for all transplantation patients, respectively after transplantation, and 11.6 days (neutrophil exceeded 0.5×10⁹/L) and 11.6 days (platelets recovery exceeded 20×10⁹/L) in HSC co-transplants with MSC respectively after transplantation. Four patients received degrees Ⅰ and Ⅱ acute graft versus host disease, and four received limited chronic graft versus host disease. The quality of life of post-transplant patients was good, without blood products infusion or severe infection or bleeding. The hematopoietic function of patients in the non-transplantation group was not recovered to normal. One patient died of cerebral hemorrhage and infection. The remaining patients had low quality of life, and should be hospitalized for symptomatic treatment, long-term discontinuous blood products infusion. Following treatment, many kinds of severe complications occurred. Results suggested that HSC transplantation is highly effective method for treating severe aplastic anemia. The hematopoietic function can be rapidly recovered. Graft versus host disease can be prevented and controlled, showing high quality of life. The curative effect is obviously better compared with non-transplantation.