The following article features coverage from the 61st American Society of Hematology Annual Meeting and Exposition. Click here to read more of Hematology Advisor’s conference coverage.

Adding eltrombopag to immunosuppressive treatment does not improve outcomes among pediatric patients with severe aplastic anemia (SAA), according to research presented at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, Florida.1

SAA is a rare autoimmune disorder characterized by the bone marrow being targeted by immune cells. In the absence of a leukocyte antigen (HLA)-matched sibling, immunosuppression is the therapy of choice for patients younger than 40. Historically, horse antithymocyte globulin (h-ATG) and cyclosporine (CSA) have been good treatment options for pediatric patients, yielding 6-month overall response rates (OS) rates of approximately 74%.

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Previous data suggest that eltrombopag, an oral thrombopoietin-receptor agonist, improves ORR when used with h-ATG and CSA among treatment-naive patients. For this study, researchers evaluated whether adding eltrombopag to an immunosuppression treatment regimen improves ORR among pediatric patients with SAA.

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Data from 39 patients younger than 18 years treated with eltrombopag with h-ATG and CSA between 2012 and 2018 were evaluated. Comparators were adult patients treated with eltrombopag and immunosuppression and historical data from 87 pediatric patients treated between 1989 and 2010 with immunosuppression only. All patients in the comparator arms were treatment naive.

Median age in the pediatric eltrombopag group was 15 years compared with a median age of 11 years in the immunosuppression only group; 7 patients were removed from the study and underwent bone marrow transplant before the 6-month mark.

Seventy-two percent (28) of patients had a response at 6 months in the eltrombopag group compared with 74% of patients in the historical immunosuppression only group (P =.836). Among those who responded, the relapse rate was higher in the eltrombopag group (43%) than in the historical group (28%; P =.252).

No significant survival differences were noted between the pediatric eltrombopag group and either the historical pediatric group or the adult group.

“In our study, the addition of eltrombopag to [immunosuppression] did not provide any obvious therapeutic benefit to pediatric patients with [SAA],” the authors concluded.


1.     Groarke EM, Patel BA, Diamond C, et al. Outcomes in pediatric patients with severe aplastic anemia treated with standard immunosuppression and eltrombopag. Oral presentation at: 61st ASH Annual Meeting and Exposition; December 7-10, 2019; Orlando, FL. Abstract 454.