Real-world use of thrombopoietic agents in treatment-naïve children with severe aplastic anemia: a multicenter retrospective study

Abstract

Severe aplastic anemia (SAA) in children is a life-threatening disease. While hematopoietic stem cell transplantation (HSCT) remains the standard of care, many SAA patients lack a suitable donor. Thrombopoietin receptor agonists (TPO-RAs) have emerged as adjuncts to immunosuppressive therapy (IST). The objective of this study was to report a multicenter experience of TPO-RAs use in treatment-naïve children with SAA. We included 57 children, with a median age of 7 years (range 1.2-17), who received TPO-RA monotherapy (n = 13) or TPO-RA + cyclosporine A (CsA) (n = 44). Median hemoglobin, platelet counts, and absolute neutrophilic count all increased significantly in both groups (p < 0.001). Overall, 68.4% of patients achieved remission, and none reported severe adverse events related to the TPO-RA. The rate of remission was higher in the TPO-RA monotherapy group (92.3%) vs TPO-RA + CsA (59.1%), (p = 0.074), with a shorter median time to remission (6 vs. 12 months; p = 0.025). Median observation period was 24 months (range 4-108) with no significant difference between the two treatment groups. Treatment with TPO-RA monotherapy or TPO-RA + CsA resulted in clinically meaningful hematologic responses. These findings support prospective evaluation of TPO-RAs in frontline therapy for SAA when HSCT is not readily available, potentially changing management of pediatric SAA in specific contexts.