Citation

Yang Y, Ji J, Tang Z, Han B. Front. Oncol. 2021; 11.

Copyright

(Copyright © 2021, Frontiers Research Foundation)

DOI

10.3389/fonc.2021.614965

PMID

unavailable

Abstract

Background and Aims: This study aimed at comparing the efficacy and safety of eltrombopag (EPAG) plus immunosuppressive therapies (ISTs) and haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in the frontline treatment for severe aplastic anemia (SAA) patients.

METHODS: Four electronic databases and Clinicaltrials.gov were comprehensively searched from January 2010 to August 2020. Studies that aimed at evaluating the efficacy and safety of EPAG+IST or haplo-HSCT in SAA patients were included. One-/2-year overall survival (OS), complete response (CR), and overall response rates (ORRs) were indirectly compared between EPAG+IST and haplo-HSCT.

RESULTS: A total of 447 patients involved in 10 cohort studies were found to be eligible for this study. A narrative synthesis was performed due to lack of data directly comparing the outcome of EPAG+IST and haplo-HSCT. Consistent with the analysis results in the whole population, subgroup analyses in the age-matched population showed that there was no significant difference in ORR between EPAG+IST and haplo-HSCT groups. However, the CR rate was lower in the EPAG+IST group when compared with the haplo-HSCT group. The incidence rate of clonal evolution/SAA relapse ranged at 8-14 and 19-31% in the EPAG+IST group but not reported in the haplo-HSCT group. The incidence rate for acute graft vs. host disease (aGVHD) and chronic graft vs. host disease (cGVHD) ranged at 52-57 and 12-67%, respectively, for the haplo-HSCT group. The main causes of deaths were infections in the EPAG+IST group, and GVHD and infections in the haplo-HSCT group.

CONCLUSION: EPAG+IST has a comparable ORR and 1-/2-year OS but lower CR rate when indirectly compared with haplo-HSCT in the frontline treatment of patients with SAA. Patients treated with haplo-HSCT may exhibit a high incidence of GVHD, whereas patients treated with EPAG+IST may experience more relapses or clone evolution. © Copyright © 2021 Yang, Ji, Tang and Han.

Language: en

Keywords

human; suicide; systematic review; Review; survival; graft versus host reaction; glucocorticoid; infection; brain disease; antibiotic agent; aciclovir; cotrimoxazole; immunosuppressive treatment; aplastic anemia; immunosuppressive agent; thymocyte antibody; methotrexate; cyclophosphamide; overall survival; data extraction; leukocyte antigen; fluconazole; cardiogenic shock; chronic graft versus host disease; hematopoietic stem cell transplantation; busulfan; antifungal agent; graft failure; whole body radiation; antibiotic prophylaxis; eltrombopag; mycophenolate mofetil; mortality rate; acute graft versus host disease; fludarabine; all cause mortality; cyclosporine; overall response rate; data quality assessment; haploidentical hematopoietic stem cell transplantation; haploidentical transplantation; immunosuppression therapy; posttransplant lymphoproliferative disease; severe aplastic anemia