Publication Only: Stem cell transplantation - Clinical
In recent years, haploid hematopoietic stem cell transplantation has made great progress in the treatment of acquired aplastic anemia, but the incidence of transplant related complications and transplant related mortality are still high, and the conditioning reginem needs to be further optimized.
To investigate the safety and efficacy of haploidentical hematopoietic stem cell transplantation with different intensity conditioning regimen in the treatment of aplastic anemia (AA).
27 AA patients who underwent haploidentical transplantation in Affiliated Hospital of Shandong University of Traditional Chinese Medicine from July 2017 to July 2018 were enrolled. All patients received bone marrow plus peripheral blood stem cells.According to the dosage of cyclophosphamide in the conditioning regimen, 26 patients excluding one other conditioning regimens were divided into high-dosage group (regimen 1, 16 cases) and low-dosage group (regimen 2, 10 cases). The data of Engraftment, graft-vs-host disease (GVHD), hematopoietic reconstitution, relapse, infection, overall survival (OS) were analyzed. The comparison between the two groups was tested by χ(2) test.
ALL patients achieved primary engraftment. The activation rate of CMV and EBV was 63 % (17/27). Post-transplant lymphocyte disease (PTLD) in 0 case. The cumulative incidences of acute GVHD grade I-IV and chronic GVHD were 22.2% (6/27) and 18.5% (5/27) respectively and the incidence of extensive chronic GVHD was 0%. The median follow-up time was 6.5 (0.5-12.5) months, the OS was 96.3% (26/27). All survived patients were no longer dependent on blood transfusion and none of them had recurrence. Comparing the rates of overall survival(94%(15/16) vs.100%(10/10)) and rates of chronic GVHD(18.8%(3/16) vs. 20%(2/10)) in regimen 1 and regimen 2 group, there were no significant difference (P> 0.05). Significant difference was found at the incidence of I-IV acute GVHD (12.5% (2/16) vs. 30% (3/10), P < 0.05).
Haploidentical hematopoietic stem cell transplantation is effective and safe in the treatment of acquired aplastic anemia. It is suitable for patients who are not eligible for matched donor transplantation. Application of low dose cyclophosphamide preconditioning in haploid transplantation is worth exploring.