Although the addition of eltrombopag to a standard immunosuppressive therapy regimen has been approved by the US Food and Drug Administration (FDA) in patients with acquired severe aplastic anemia who are 2 years of age and older, a new subgroup analysis does not support the addition of eltrombopag to immunosuppressive therapy in children with treatment-naive severe aplastic anemia. This finding was published in the British Journal of Haematology.1

The new data come from an ongoing, prospective, nonrandomized phase 1/2 study of eltrombopag added to standard immunosuppressive therapy for patients with treatment-naive severe aplastic anemia ( Identifier: NCT01623167). The study compared these new data (n=40; study period, 2012-2019) with data from an historical pediatric cohort treated with standard immunosuppressive therapy alone in 4 different clinical trials (n=87; study period, 1989-2010; Identifier: NCT00061360, NCT00001964, and NCT00260689 and no NCT number available: Rosenfeld et al, 20032) and data from comparator adult patients who received eltrombopag in the ongoing study (n=131) and immunosuppressive therapy in the historical study (n=286). Patients were assessed for overall response rate (ORR) at 3 and 6 months, complete response (CR) rate, relapse, clonal evolution, event-free survival (EFS), and overall survival (OS).1,2

The pediatric eltrombopag and historical groups showed no significant differences in 6-month ORR (70% vs 72%, respectively; P = .78) or CR rate (30% vs 23%; P = .42). However, the adult eltrombopag group had significantly improved ORR compared with the historical group (82% vs 58%; respectively; P < .001).1

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Subgroup analysis revealed that younger children (<12 years) had lower CR rates with the addition of eltrombopag than with immunosuppressive therapy alone (6% vs 24%; P = .049); however, the difference between adolescents (>12 years) in the 2 groups was not significant (46% vs 21%; P = .052).1

A nonsignificant trend toward higher probability of relapse (43% vs 27%; likelihood ratio P = .0661) and significantly lower EFS was documented in children who received eltrombopag compared with those who received immunosuppressive therapy alone (at 1432.5 days, 57% vs 69%; likelihood ratio P = .049).1

“Addition of [eltrombopag] added to standard [immunosuppressive therapy] did not improve outcomes in children with treatment-naive [severe aplastic anemia]. [Eltrombopag] in the pediatric population should not automatically be considered standard of care,” concluded the authors.

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


  1. Groarke EM, Patel BA, Gutierrez‐Rodrigues F, et al. Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia. Br J Haematol. January 2021:bjh.17232. doi:10.1111/bjh.17232
  2. Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289(9):1130-1135. doi:10.1001/jama.289.9.1130