Thrombopoietin receptor agonist switch in children with persistent and chronic severe immune thrombocytopenia: A retrospective analysis in a large tertiary center

Pediatr Blood Cancer. 2019 Jun;66(6):e27704. doi: 10.1002/pbc.27704. Epub 2019 Mar 11.

Abstract

We retrospectively analyzed sequential therapy with romiplostim and eltrombopag in 23 children with immune thrombocytopenia: switching from romiplostim to eltrombopag (10 patients) or vice versa (13 patients). The median age of patients at enrollment in the study was 5.6 years (2-15 years). Switching from romiplostim to eltrombopag was effective in eight (80%) patients, whereas switching from eltrombopag to romiplostim was effective in eight (62%) patients. The response rate was similar in patients failing the first thrombopoietin receptor agonist and those who had previous response. To date, all responders continue to maintain platelets over 50 × 109 /L at 13-39 months after switching.

Keywords: children; eltrombopag; immune thrombocytopenia; romiplostim; thrombopoietin receptor agonists.

MeSH terms

  • Adolescent
  • Benzoates / administration & dosage*
  • Child
  • Child, Preschool
  • Chronic Disease
  • Female
  • Follow-Up Studies
  • Humans
  • Hydrazines / administration & dosage*
  • Male
  • Platelet Count
  • Prognosis
  • Purpura, Thrombocytopenic, Idiopathic / blood
  • Purpura, Thrombocytopenic, Idiopathic / classification
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Pyrazoles / administration & dosage*
  • Receptors, Fc / administration & dosage*
  • Receptors, Thrombopoietin / agonists*
  • Recombinant Fusion Proteins / administration & dosage*
  • Retrospective Studies
  • Severity of Illness Index
  • Tertiary Care Centers / standards*
  • Thrombopoietin / administration & dosage*

Substances

  • Benzoates
  • Hydrazines
  • Pyrazoles
  • Receptors, Fc
  • Receptors, Thrombopoietin
  • Recombinant Fusion Proteins
  • Thrombopoietin
  • romiplostim
  • eltrombopag