Eltrombopag for immune thrombocytopenia secondary to chronic lymphoproliferative disorders: a phase 2 multicenter study

Blood. 2019 Nov 14;134(20):1708-1711. doi: 10.1182/blood.2019001617.

Abstract

Immune thrombocytopenia (ITP) secondary to chronic lymphoproliferative disorders (LPDs) is poorly responsive to conventional treatments. We conducted a multicenter phase 2 prospective 24-week study in 18 patients with ITP secondary to LPDs to assess the safety and efficacy of eltrombopag. Responsive patients entered an extension study for up to 5 years. For inclusion, patients should not require cytotoxic treatment and should have a platelet count <30 × 109/L or have symptoms of bleeding. Eltrombopag was initiated at 50 mg/day, with a maximum of 150 mg/day. The primary end point was platelet response after 4 weeks. Median age was 70 years (range, 43-83 years), and 14 patients had chronic lymphocytic leukemia, 2 had classic Hodgkin lymphoma, and 2 had Waldenström macroglobulinemia. All patients had received previous ITP treatments. Response rate at week 4 was 78% (95% confidence interval [CI], 58%-97%), with 50% of patients having a complete response (CR) (95% CI, 43%-57%); respective results at week 24 were 59% (95% CI, 36%-82%) with 30% reaching a CR (95% CI, 8%-52%). Median exposure to eltrombopag was 16 months; median dose at week 4 was 50 mg/day (range, 25-100 mg/day), and at week 24, it was 50 mg/day (range, 25-150 mg/day). No grade >2 adverse events were reported. Eltrombopag is active and well tolerated in ITP secondary to LPDs. This trial was registered at www.clinicaltrials.gov as #NCT01610180.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Blood Platelets / drug effects
  • Chronic Disease
  • Female
  • Humans
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use*
  • Lymphoproliferative Disorders / complications*
  • Male
  • Middle Aged
  • Prospective Studies
  • Purpura, Thrombocytopenic, Idiopathic / complications*
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use*

Substances

  • Benzoates
  • Hydrazines
  • Pyrazoles
  • eltrombopag

Associated data

  • ClinicalTrials.gov/NCT01610180
  • ClinicalTrials.gov/NCT01610180