Contributions
Abstract: EP593
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Eltrombopag (ELT) is a thrombopoietin agonist which has been recently applied with immunosuppressive therapy (IST) for untreated severe aplastic anemia (SAA) patients. This new combination therapy was reported to be effective, yet no study has compared ELT+IST with matched sibling donor bone marrow transplantation (MSD-BMT), the traditional first-line therapy for SAA.
Aims
This meta-analysis aimed to compare the efficacy and survival benefit of ELT+IST and MSD-BMT for newly-diagnosed SAA patients.
Methods
Databases of Pubmed, Embase, Cochrane Library and Web of Science were searched. Studies published in recent 10 years, with a topic of ELT+IST and/or MSD-BMT and a record of patient overall response (OR) rate/myeloid engraftment rate, 1, 2 and 2.5-year overall survival (OS) rate and causes of death were included. Patients were separated into ELT+IST group and MSD-BMT group for data analysis. Baseline characteristic-compatible subgroup were selected for further analysis. OR rate, OS rate, failure-free survival (FFS) rate and the proportion of adverse events (AE) were pooled and compared.
Results
8 clinical trials or cohort studies with 1004 patients (113 patients receiving ELT+IST, and 891 receiving MSD-BMT) were selected. The pooled age of patients receiving ELT+IST was significantly older than that of MSD-BMT group (45.2±20.5 vs 21.6±15.6 years old, P=0.000). For ELT-IST group, the pooled OR rate was significantly lower (86% vs 94%, P=0.002), yet the 1, 2 and 2.5-year OS rate was significantly higher (99% to 99% vs 92% to 90% from 1 to 2.5-year OS, P<0.05). No significant difference was observed in 1 and 2-year FFS rate between 2 therapy groups. The major cause of death was infection for MSD-BMT, while no treatment-related death was recorded in ELT+IST group. The pooled relapse rate was 16%, and 8.4% for pooled clonal evolution rate for ELT+IST, while no record of relapse or clonal evolution was available for MSD-BMT. In the subgroup with compatible patient baseline characteristics (mean age: 33 years old), 92 patients were included in ELT+IST group and 213 patients were in MSD-BMT group. The pooled OR rate was lower (87% vs 95%, P=0.017), and the pooled 1, 2 and 2.5-year OS rate was higher (P<0.05) for ELT+IST. For ELT+IST, the relapse rate was 14%, and the clonal evolution rate was 7.6%, while there was no relevant data for MSD-BMT group.
Conclusion
ELT+IST had potential advantage in survival benefit but lower OR rate for newly-diagnosed SAA patients when compared with MSD-BMT during 2.5 years of follow-up. Treatment-related death and relapse/clonal evolution needed to be balanced when ELT+IST was considered for SAA patients.
Keyword(s): Aplastic anemia, Clinical outcome, Immunosuppressive therapy, Transplant
Abstract: EP593
Type: E-Poster Presentation
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Eltrombopag (ELT) is a thrombopoietin agonist which has been recently applied with immunosuppressive therapy (IST) for untreated severe aplastic anemia (SAA) patients. This new combination therapy was reported to be effective, yet no study has compared ELT+IST with matched sibling donor bone marrow transplantation (MSD-BMT), the traditional first-line therapy for SAA.
Aims
This meta-analysis aimed to compare the efficacy and survival benefit of ELT+IST and MSD-BMT for newly-diagnosed SAA patients.
Methods
Databases of Pubmed, Embase, Cochrane Library and Web of Science were searched. Studies published in recent 10 years, with a topic of ELT+IST and/or MSD-BMT and a record of patient overall response (OR) rate/myeloid engraftment rate, 1, 2 and 2.5-year overall survival (OS) rate and causes of death were included. Patients were separated into ELT+IST group and MSD-BMT group for data analysis. Baseline characteristic-compatible subgroup were selected for further analysis. OR rate, OS rate, failure-free survival (FFS) rate and the proportion of adverse events (AE) were pooled and compared.
Results
8 clinical trials or cohort studies with 1004 patients (113 patients receiving ELT+IST, and 891 receiving MSD-BMT) were selected. The pooled age of patients receiving ELT+IST was significantly older than that of MSD-BMT group (45.2±20.5 vs 21.6±15.6 years old, P=0.000). For ELT-IST group, the pooled OR rate was significantly lower (86% vs 94%, P=0.002), yet the 1, 2 and 2.5-year OS rate was significantly higher (99% to 99% vs 92% to 90% from 1 to 2.5-year OS, P<0.05). No significant difference was observed in 1 and 2-year FFS rate between 2 therapy groups. The major cause of death was infection for MSD-BMT, while no treatment-related death was recorded in ELT+IST group. The pooled relapse rate was 16%, and 8.4% for pooled clonal evolution rate for ELT+IST, while no record of relapse or clonal evolution was available for MSD-BMT. In the subgroup with compatible patient baseline characteristics (mean age: 33 years old), 92 patients were included in ELT+IST group and 213 patients were in MSD-BMT group. The pooled OR rate was lower (87% vs 95%, P=0.017), and the pooled 1, 2 and 2.5-year OS rate was higher (P<0.05) for ELT+IST. For ELT+IST, the relapse rate was 14%, and the clonal evolution rate was 7.6%, while there was no relevant data for MSD-BMT group.
Conclusion
ELT+IST had potential advantage in survival benefit but lower OR rate for newly-diagnosed SAA patients when compared with MSD-BMT during 2.5 years of follow-up. Treatment-related death and relapse/clonal evolution needed to be balanced when ELT+IST was considered for SAA patients.
Keyword(s): Aplastic anemia, Clinical outcome, Immunosuppressive therapy, Transplant