Treatment characteristics, efficacy and safety of thrombopoietin analogues in routine management of primary immune thrombocytopenia

Blood Coagul Fibrinolysis. 2018 Jun;29(4):374-380. doi: 10.1097/MBC.0000000000000726.

Abstract

: Thrombopoietin receptor agonist (TPO-RAs) have demonstrated good efficacy and tolerance in clinical trials in refractory chronic primary immune thrombocytopenia (ITP) or chronic ITP with contraindication for splenectomy. No head-to-head study is available, and differences in trials design do not allow comparisons. Information on the use of TPO-RAs in nonchronic ITP is scant. We described our experience with TPO-RAs in ITP (chronic, persistent and newly diagnosed ITP) in routine clinical practice. Retrospective series of 100 adult ITP patients was analysed; 41 treated with eltrombopag, 37 with romiplostim and 22 with both. Response-related and safety variables were evaluated. With a median follow-up of 86.5 weeks (interquartile range, 34.3-128 weeks), no differences were found in response rate, time to response, stability of response or response duration based on the type of TPO-RA used. Of all, 25% of patients with newly diagnosed or persistent ITP and 7.2% with chronic responded and maintained their response when TPO-RAs were stopped. Regarding safety, two developed bone marrow fibrosis grade 3, with loss of response to both drugs. Incidence of vascular events was 7%. Both TPO-RAs may be useful in all types of ITP, not only chronic but also persistent and newly diagnosed. Similar results were noted in efficacy and safety variables for both drugs.

MeSH terms

  • Adult
  • Aged
  • Benzoates / therapeutic use
  • Disease Management
  • Female
  • Humans
  • Hydrazines / therapeutic use
  • Male
  • Middle Aged
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Pyrazoles / therapeutic use
  • Receptors, Fc / therapeutic use
  • Receptors, Thrombopoietin / agonists
  • Recombinant Fusion Proteins / therapeutic use
  • Retrospective Studies
  • Thrombopoietin / therapeutic use*
  • Treatment Outcome

Substances

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