Eltrombopag Therapy in Children With Rare Disorders Associated With Thrombocytopenia

J Pediatr Hematol Oncol. 2020 Mar;42(2):113-117. doi: 10.1097/MPH.0000000000001528.

Abstract

Eltrombopag (ELT) is a thrombopoietin receptor activator that has shown efficacy in chronic immune thrombocytopenia. We report the outcome of ELT therapy in 4 children who were treated for rare hematologic disorders, including Pearson syndrome, DiGeorge syndrome, posttransplant allogeneic poor graft function (PGF), and Wiskott-Aldrich syndrome. The ELT tolerance in the analyzed group was good, with the exception of the child with Pearson syndrome, who experienced an exacerbation of cataracts and had to discontinue treatment. Thromboembolic events were observed in one child, who continued ELT therapy despite achieving normalized platelet counts. Independence from PLT transfusions was observed at the 4-week timepoint of therapy in patients with DiGeorge syndrome and PGF who responded to ELT. Discontinuation of therapy was successful in one child, who sustained the normal CBC values afterward. In 2 patients, an increase in neutrophil counts was observed during ELT therapy without additional intervention, and a positive correlation between neutrophil and platelet values during ELT therapy was observed in the child with PGF. ELT is effective in rare pediatric disorders, but response patterns are determined by the underlying disease. ELT shows promising results in patients, but constitutional hematopoiesis defects reduce the chances of a response.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acyl-CoA Dehydrogenase, Long-Chain / deficiency*
  • Adolescent
  • Benzoates / therapeutic use*
  • Child
  • Child, Preschool
  • Congenital Bone Marrow Failure Syndromes / complications
  • Congenital Bone Marrow Failure Syndromes / drug therapy*
  • Congenital Bone Marrow Failure Syndromes / pathology
  • DiGeorge Syndrome / complications
  • DiGeorge Syndrome / drug therapy*
  • DiGeorge Syndrome / pathology
  • Female
  • Graft Rejection / drug therapy*
  • Graft Rejection / etiology
  • Graft Rejection / pathology
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Humans
  • Hydrazines / therapeutic use*
  • Lipid Metabolism, Inborn Errors / complications
  • Lipid Metabolism, Inborn Errors / drug therapy*
  • Lipid Metabolism, Inborn Errors / pathology
  • Male
  • Mitochondrial Diseases / complications
  • Mitochondrial Diseases / drug therapy*
  • Mitochondrial Diseases / pathology
  • Muscular Diseases / complications
  • Muscular Diseases / drug therapy*
  • Muscular Diseases / pathology
  • Prognosis
  • Pyrazoles / therapeutic use*
  • Receptors, Thrombopoietin / agonists*
  • Thrombocytopenia / complications
  • Thrombocytopenia / drug therapy*
  • Thrombocytopenia / pathology
  • Wiskott-Aldrich Syndrome / complications
  • Wiskott-Aldrich Syndrome / drug therapy*
  • Wiskott-Aldrich Syndrome / pathology

Substances

  • Benzoates
  • Hydrazines
  • Pyrazoles
  • Receptors, Thrombopoietin
  • Acyl-CoA Dehydrogenase, Long-Chain
  • eltrombopag

Supplementary concepts

  • VLCAD deficiency