| Comparison of Hematopoietic Stem Cell Transplantation Outcomes Using Matched Sibling Donors,Haploidentical Donors,and Immunosuppressive Therapy for Patients With Acquired Aplastic AnemiaObjectives To compare the outcomes of patients with acquired aplastic anemia(AA)who underwent hematopoietic stem cell transplantation(HSCT)with a fludarabine-based conditioning regimen from matched sibling donors(MSD)or haploidentical donors(HID)and immunosuppressive therapy(IST).Methods From 2014 and 2020,a total of 387 consecutively patients with acquired AA from Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College who received MSD-HSCT(n=108),HID-HSCT(n=91),or IST(n=188)were enrolled into this study.The primary objective of this study was to compare the overall survival(OS)of AA patients who received different procedures.Other major outcomes included infection,engraftment,acute graft versus host disease(aGVHD),chronic GVHD(cGVHD),therapy-related mortality(TRM),failure-free survival(FFS),and GVHD-free,FFS(GFFS).Results Compared with HID-HSCT,MSD-HSCT had a lower incidence of graft failure(1%vs.7%,P=0.062),grade II-IV aGVHD(16%vs.35%,P=0.001),and mild to severe cGVHD(8%vs.23%,P=0.007),but an equivalent incidence of gradeⅢ-Ⅳ aGVHD(8%vs.12%,P=0.237)and moderate to severe cGVHD(3%vs.9%,P=0.076).HSCT had superior blood count recovery at 3,6,and 12 months compared with IST(P<0.001).The estimated 5-year OS of the MSD,HID,and IST groups were 86%(95%confidence interval[CI],81-95),72%(95%CI,64-84),and 79%(95%CI,76-89)(P=0.02),respectively;accordingly,the FFS rates were 85%(95%CI,80-94),68%(95%CI,59-80),and 56%(95%CI,47-65),respectively(P<0.001).The estimated 5-year GFFS rate of MSD patients was 81%(95%CI,77-91)vs.65%(95%Cl,55-76)of HID patients(P=0.002).For patients aged<40 years,the OS rate was still significantly superior for MSD-HSCT recipients compared to HID-HSCT recipients(89%[95%CI,83-98]vs.76%[95%CI,67-88],P=0.024)while the HID-HSCT recipients showed similar OS(76%[95%CI,67-88]vs.78%[95%CI,71-88],P=0.166)but superior FFS(P=0.047)when follow-up was longer than 14.5 months in contrast to IST.Eleven patients(6%)in the IST group experienced MDS/AML evolution compared with none of patients in the transplant groups(P=0.002).As expected,during follow-up,we noticed that young women(≤40 years)receiving a FAC conditioning regimen were more likely to restore menstruation after transplantation(9 out of 10)compared with those receiving a BFAC conditioning regimen(3 out of 10 patients)(P=0.02).In a multivariate analysis,HID-HSCT and a conditioning regimen that included busulfan were adversely related to OS among patients who received allografts.We also performed propensity score matching to minimize effects of confounding donor factors(56 patients in the MSD and HID groups,respectively).After matching,the estimated 5-year OS,FFS,and GFFS rates of MSD patients were 92%(95%CI,88-100),92%(95%CI,88-100),and 87%(95%CI,80-100)vs.77%(95%CI,67-90)(P=0.012),75%(95%CI,65-88)(P=0.006),and 69%(95%CI,58-84)(P=0.026)of HID patients.Conclusions MSD-HSCT was the frontline choice for patients with severe AA aged≤40 years,while HID-HSCT was as effective as IST for patients without an MSD.Retrospective Comparison of Efficacy and Safety of Rabbit Anti-Thymocyte Globulin and Porcine Anti-Lymphocyte Globulin in Patients with Acquired Aplastic Anemia Undergoing Hematopoietic Stem Cell Transplantation from Matched Sibling DonorsObjectives To compare the efficacy and safety of porcine anti-lymphocyte globulin (pALG) and rabbit anti-thymocyte globulin (rATG) in patients with acquired aplastic anemia (AA) receiving hematopoietic stem cell transplantation(HSCT) from matched sibling donors(MSD).Methods From 2005 and 2020,a total of 226 patients with acquired AA who consecutively received MSD-HSCT from Institute of Hematology & Blood Diseases Hospital,Chinese Academy of Medical Sciences & Peking Union Medical College and Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology were enrolled in this study.140 patients received pALG while 86 patients received rATG.Engraftment,infection,graft versus host disease(GVHD),transplantation-related mortality(TRM),and survival among two groups were evaluated.Results The two groups had similar baseline characteristics except for a higher number of infused mononuclear cells(P<0.001) and a higher proportion of peripheral blood stem cells as graft sources(P=0.003) in the pALG group.The rates of neutrophil engraftment(P=1),platelet engraftment (P=0.228),bloodstream infection(P=0.867),invasive fUngal diseases(P=0.362),cytomegalovirus viremia(P=0.667),and graft rejection(P=0.147) were similar in the two groups.A higher cumulative incidence of grade II-IV acute GVHD (aGVHD) at 100 days occurred in the pALG group(19% vs.8%,P=0.035)while no significant differences in grade Ⅲ- Ⅳ aGVHD(P=0.572),mild to severe chronic GVHD(cGVHD)(P=0.181),and moderate to severe cGVHD(P=0.586) were observed.The actual 5-year overall survival(OS),failure-free survival(FFS),and GVHD-free,FFS rates of the pALG group were 87%(95% confidence interval [CI],82-93),85%(95%CI,80-92),and 78%(95%CI,72-92) versus 91%(95%CI,86-99)(P=0.33),88%(95%CI,82-97)(P=0.428),and 79%(95%CIS 72-90)(P=0.824) in the rATG group,respectively.A busulfan-containing conditioning regimen was the only adverse risk factor for OS and FFS in multivariate analysis.Conclusion pALG is an alternative to rATG in patients with severe AA receiving MSD-HSCT. |
PDF Full Text Request