A clinical study of allogeneic hematopoietic stem cell transplantation for the treatment of severe aplastic anemia
Objective To investigate the efficacy of allogeneic hematopoietic stem cell transplantation(allo-HSCT)in the treatment of SAA/vSAA,and to compare the survival of haploidentical donor-hematopoietic stem cell transplantation(HID-HSCT)with matched sibling donor-hematopoietic stem cell transplantation(MSD-HSCT).Method The clinical data of 66 SAA/vSAA patients treated with allo-HSCT in the 940th Hospital of Joint Logistics Support Force of Chinese People's Liberation Army from January 2013 to December 2022 were retrospectively analyzed,and they were divided into 40 cases in HID-HSCT group and 26 cases in MSD-HSCT group according to the donor situation.Hematopoietic reconstruction,graft versus host disease(GVHD)and postoperative complications of 66 patients were analyzed and the survival of the two groups were compared.Result Among 66 SAA/vSAA patients,63 patients achieved hematopoietic reconstruction,the rate of hematopoietic reconstruction was 95.45%,and 3 patients who did not achieve hematopoietic reconstruction died of sepsis and gastrointestinal hemorrhage.The median implantation time of neutrophil was 15 days and the median implantation time of platelet was 16 days and 11 to 28 days after transplantation.Acute graft versus host disease(aGVHD)occurred in 31 patients,with a cumulative incidence of 47.0%,including 23 cases with grade Ⅰ/Ⅱ aGVHD and 8 cases with grade Ⅲ/Ⅳ aGVHD.There were 23 patients with upper respiratory tract infection,34 patients with different degrees of lung infection,4 patients with subcutaneous soft tissue infection,7 patients with urinary tract infection,7 patients with intestinal infection,6 patients with intracranial infection,14 patients with sepsis,14 patients with Epstein-Barr viremia,and 20 patients with cytomegaloviremia.3 patients developed severe intestinal GVHD combined with gastrointestinal hemorrhage after transplantation,1 patient died of sepsis and gastrointestinal hemorrhage after continuous non-recovery of blood image after transplantation,1 patient suffered intracranial hemorrhage after infection secondary to thrombocytopenia after hematopoietic reconstruction,and 2 patients suffered secondary cerebral hemorrhage due to severe infection and aGVHD after transplantation.9 patients developed(delayed)hemorrhagic cystitis after transplantation,3 patients developed Epstein-Barr virus associated lymphoproliferative disease after transplantation,3 patients developed thrombotic microangiopathy,1 patient developed thrombotic microangiopathy renal injury,and 6 patients developed secondary hypoimmunoglobulinemia.As of July 1,2023,48 patients were alive and 18 died,and the 5-year estimated overall survival rate was 76.1%.The 5-year estimated overall survival rate of the HID-HSCT group and the MSD-HSCT group were 73.5%and 80.0%,respectively,with no statistically significant difference(P=0.59).Conclusion Allo-HSCT is the effective way to cure severe aplastic anemia,with good sib matching effect and few transplant-related complications.As the first choice,HID-HSCT is preferred as an alternative treatment when there is no MSD-HSCT.
Severe aplastic anemiaAllogeneic hematopoietic stem cell transplantationHaploid transplantationHematopoietic reconstructionGraft versus host disease