A novel recombinant human thrombopoietin therapy for the management of immune thrombocytopenia in pregnancy

Blood. 2017 Aug 31;130(9):1097-1103. doi: 10.1182/blood-2017-01-761262. Epub 2017 Jun 19.

Abstract

The aim of this study was to determine the safety and efficacy of recombinant human thrombopoietin (rhTPO) for the management of immune thrombocytopenia (ITP) during pregnancy. Pregnant patients with ITP were enrolled in the study if they had a platelet count less than 30 × 109/L, were experiencing bleeding manifestations, had failed to respond to corticosteroids and/or intravenous immunoglobulin (IVIG), and had developed refractoriness to platelet transfusion. Thirty-one patients received rhTPO at an initial dose of 300 U/kg once daily for 14 days. Twenty-three patients responded (74.2%), including 10 complete responders (>100 × 109/L) and 13 responders (30-100 × 109/L). It appears that rhTPO ameliorated the bleeding symptoms remarkably, even in the nonresponders. rhTPO was well tolerated. Dizziness, fatigue, and pain at an injection site were reported in 1 patient each. No congenital disease or developmental delays were observed in the infants in a median follow-up of 53 (range, 39-68) weeks. In conclusion, rhTPO is a potentially safe and effective treatment choice for patients with ITP during pregnancy. Our work has paved the way for further study on the clinical application of rhTPO and other thrombopoietic agents for the management of ITP during pregnancy. This study is registered at www.clinicaltrials.gov as NCT02391272.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Female
  • Hemorrhage / drug therapy
  • Humans
  • Infant, Newborn
  • Platelet Count
  • Pregnancy
  • Pregnancy Complications, Hematologic / blood
  • Pregnancy Complications, Hematologic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / blood
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use*
  • Thrombopoietin / adverse effects
  • Thrombopoietin / blood
  • Thrombopoietin / therapeutic use*

Substances

  • Recombinant Proteins
  • Thrombopoietin

Associated data

  • ClinicalTrials.gov/NCT02391272