Real-world use of thrombopoietin receptor agonists for the management of immune thrombocytopenia in adult patients in the United Kingdom: Results from the TRAIT study

Br J Haematol. 2024 Jun;204(6):2442-2452. doi: 10.1111/bjh.19345. Epub 2024 Mar 1.

Abstract

Few studies have reported the real-world use of both romiplostim and eltrombopag in immune thrombocytopenia (ITP). TRAIT was a retrospective observational study aimed to evaluate the platelet responses and adverse effects associated with the use of these thrombopoietin receptor agonists (TPO-RAs) in adult patients with ITP in the United Kingdom. Of 267 patients (median age at diagnosis, 48 years) with ITP (primary ITP [n = 218], secondary ITP [n = 49]) included in the study, 112 (42%) received eltrombopag and 155 (58%) received romiplostim as the first prescribed TPO-RA. A platelet count ≥30 × 109/L was achieved in 89% of patients with the first TPO-RA treatments, while 68% achieved a platelet count ≥100 × 109/L. Treatment-free response (TFR; platelet count ≥30 × 109/L, 3 months after discontinuing treatment) was achieved by 18% of the total patients. Overall, 61 patients (23%) switched TPO-RAs, most of whom achieved platelet counts ≥30 × 109/L with the second TPO-RA (23/25 who switched from eltrombopag to romiplostim [92%]; 28/36 who switched from romiplostim to eltrombopag [78%]). TFR was associated with secondary ITP, early TPO-RA initiation after diagnosis, the presence of comorbidity and no prior splenectomy or treatment with steroids or mycophenolate mofetil. Both TPO-RAs had similar efficacy and safety profiles to those reported in clinical studies.

Keywords: eltrombopag; immune thrombocytopenia (ITP); real‐world; romiplostim; thrombopoietin receptor agonist (TPO‐RA).

Publication types

  • Observational Study
  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Benzoates* / adverse effects
  • Benzoates* / therapeutic use
  • Female
  • Humans
  • Hydrazines* / adverse effects
  • Hydrazines* / therapeutic use
  • Male
  • Middle Aged
  • Platelet Count
  • Purpura, Thrombocytopenic, Idiopathic* / drug therapy
  • Pyrazoles* / adverse effects
  • Pyrazoles* / therapeutic use
  • Receptors, Fc* / therapeutic use
  • Receptors, Thrombopoietin* / agonists
  • Recombinant Fusion Proteins* / administration & dosage
  • Recombinant Fusion Proteins* / adverse effects
  • Recombinant Fusion Proteins* / therapeutic use
  • Retrospective Studies
  • Thrombopoietin* / adverse effects
  • Thrombopoietin* / therapeutic use
  • Treatment Outcome
  • United Kingdom
  • Young Adult

Substances

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