Adding eltrombopag to intensive immunosuppressive therapy helps adult patients catch up pediatric patients in severe aplastic anemia

Abstract

Direct comparison of immunosuppressive therapy (IST), especially after the addition of thrombopoietin receptor agonist (TPO-RA), between adult and paediatric patients with severe aplastic anaemia (SAA) has not been enunciated. The aim of this study was to evaluate differences in efficacy and survival between adults and children receiving IST ± eltrombopag (EPAG). Baseline characteristics, haematological response, overall survival (OS), event-free survival (EFS), relapse, and clonal evolution were compared in 366 patients (141 adults, 197males, median age 11 years). For IST alone, adults had a similar overall response rate (ORR) but a lower complete response rate (CRR) than did children at 12 months. Moreover, adults had lower OS and EFS. After adding EPAG, the ORR was greater in adults, and the CRR was similar between the two groups. OS and EFS in adults improved significantly and were similar to those in children. Adults had similar relapse rates but higher clonal evolution rates than children, with or without EPAG. Adults had greater clonal evolution, regardless of the addition of EPAG. Adding EPAG to intensive IST improved the ORR, CRR, OS and EFS in adults more than it did in children, bridged the gap previously existed between adult and paediatric patients.