Aim: To evaluate the differences of efficacy and survival between adults and children patients with severe aplastic anemia receiving intensive immunosuppressive therapy (IST) ± eltrombopag.

Methods: The clinical data of patients with SEVERE APLASTIC ANEMIA receiving IST ± eltrombopag between 2013 and 2022 were analyzed retrospectively. Baseline characteristics, hematological response, overall survival (OS), event-free survival (EFS), relapse, and clonal evolution in those treated with IST alone or IST+ eltrombopag were compared between adult (aged >18 years) and pediatric (aged ≤18 years) patients.

Results: A total of 366 patients were included, including 141 adults and 225 children. For IST alone, adult patients had a similar overall response rate (ORR, 76% vs.75%, P=1.000) but a lower complete response rate (CRR, 31% vs. 48%, P=0.048) than pediatric patients at 12 months. Adult patients had lower OS (84.9±3.9% vs. 96.9±2.2%, P=0.007) and EFS (66.4±5.1% vs. 79.7±5.3%, P=0.047) than pediatric patients. After adding eltrombopag, the ORR was higher in adult patients than pediatric patients (89% vs. 73%, P=0.027) and the CRR was similar in two groups (54% vs. 50%, P=0.614). The OS and EFS in adults improved significantly and were similar to those in pediatric patients (91.4±4.2% vs. 96.7±1.8%, P=0.081 and 75.1±6.3% vs. 72.3±4.7%, P=0.834, respectively). Adults had similar relapse rates as children, either with or without eltrombopag (P>0.05). Adult patients had significant higher clonal evolution rates than pediatric patients, either in IST alone group (9.2% vs. 1.4%, P=0.040) or in IST+ eltrombopag group (5.5% vs. 0%, P=0.017).

Conclusions: Adding eltrombopag to intensive IST improved the ORR, CRR, OS and EFS in adults more than it did in children. Adults had greater clonal evolution, regardless of the addition of eltrombopag.

Disclosures

No relevant conflicts of interest to declare.

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2024
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