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.
Chazli et al. (Mon,) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: