Adding recombinant human thrombopoietin to cyclosporine A and hetrombopag enhances the early hematologic response in transfusion-dependent non-severe aplastic anemia: a retrospective study
Year: 2025
Type: article
Study design: Observational Study
Abstract: Objectives Transfusion-dependent non-severe aplastic anemia (TD-NSAA) is a bone marrow failure disorder with high progression risk and transfusion complications, necessitating rapidly effective therapies. Recombinant human thrombopoietin (rhTPO) stimulates megakaryocyte proliferation through a mechanism distinct from that of oral TPO receptor agonists (TPO-RAs), suggesting a potential synergistic effect in accelerating hematopoietic recovery. This study evaluated the efficacy and safety of rhTPO plus cyclosporine A (CsA) and hetrombopag in patients with TD-NSAA.Methods In this retrospective study, data were collected from patients with newly diagnosed TD-NSAA treated with CsA + hetrombopag + rhTPO or CsA + hetrombopag alone between July 2022 and December 2023 at our center.Results Twenty-nine patients in the rhTPO group and 28 in the control group were enrolled. The overall response rates (ORRs) at 1 and 2 months were significantly higher in the rhTPO group (34.5% vs 10.7%, P = 0.033; 55.2% vs 28.6%, P = 0.042, respectively). Time to achieve OR was shorter (P = 0.035), and platelet transfusion independence rates at 2 and 3 months were higher in the rhTPO group (52.6% vs. 18.8%, P = 0.039; 63.2% vs. 25.0%, P = 0.024, respectively). No significant difference was observed between the two groups in 3/6 months ORRs or complete response rates. Adverse events were comparable between groups.Conclusion Adding rhTPO to CsA + hetrombopag improves platelet recovery and early hematologic responses, supporting its potential role in optimizing the initial management of patients with TD-NSAA.
Source: Hematology
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,557
Awards: G3132876688, G4633788826, G5175395690
OpenAlex ID: W4414783187