Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia

Br J Haematol. 2021 Feb;192(3):605-614. doi: 10.1111/bjh.17232. Epub 2021 Jan 7.


Acquired severe aplastic anaemia (SAA) has an immune pathogenesis, and immunosuppressive therapy (IST) with anti-thymocyte globulin and cyclosporine is effective therapy. Eltrombopag (EPAG) added to standard IST was associated with higher overall and complete response rates in patients with treatment-naïve SAA compared to a historical IST cohort. We performed a paediatric subgroup analysis of this trial including all patients aged <18 years who received EPAG plus standard IST (n = 40 patients) compared to a historical cohort (n = 87) who received IST alone. Response, relapse, clonal evolution, event-free survival (EFS), and overall survival were assessed. There was no significant difference in either the overall response rate (ORR) or complete response rate at 6 months (ORR 70% in EPAG group, 72% in historical group, P = 0·78). Adults (≥18 years) had a significantly improved ORR of 82% with EPAG compared to 58% historically (P < 0·001). Younger children had lower response rates than did adolescents. The trend towards relapse was higher and EFS significantly lower in children who received EPAG compared to IST alone. Addition of EPAG added to standard IST did not improve outcomes in children with treatment-naïve SAA. EPAG in the paediatric population should not automatically be considered standard of care. Registration: clinicaltrials.gov (NCT01623167).

Keywords: aplastic anaemia; marrow failure; paediatric aplastic anaemia; paediatric haematology.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Anemia, Aplastic / drug therapy*
  • Anemia, Aplastic / immunology
  • Antilymphocyte Serum / therapeutic use*
  • Benzoates / therapeutic use*
  • Child
  • Cyclosporine / therapeutic use*
  • Female
  • Humans
  • Hydrazines / therapeutic use*
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Pyrazoles / therapeutic use*
  • Treatment Outcome


  • Antilymphocyte Serum
  • Benzoates
  • Hydrazines
  • Immunosuppressive Agents
  • Pyrazoles
  • Cyclosporine
  • eltrombopag

Associated data

  • ClinicalTrials.gov/NCT01623167