A phase 2 clinical trial of eltrombopag for treatment of patients with myelodysplastic syndromes after hypomethylating-agent failure

Mahesh Swaminathan, Gautam Borthakur, Tapan M. Kadia, Alessandra Ferrajoli, Yesid Alvarado, Naveen Pemmaraju, Kristy Bodden, Brittany Yearby, Marina Konopleva, Joseph Khoury, Carlos Bueso-Ramos, Guillermo Garcia-Manero, Courtney D. DiNardo

Research output: Contribution to journalArticlepeer-review

8 Scopus citations

Abstract

Hypomethylating agents (HMA) are the standard of care for treatment of myelodysplastic syndromes (MDS). HMA-failure MDS has extremely poor prognosis. This study was designed to explore the utility of eltrombopag in post-HMA failure MDS patients. Patients were treated in one of two arms: eltrombopag as monotherapy (Arm A), or with continuation of HMA (Arm B). The starting eltrombopag dose was 200 mg orally daily. Twenty-nine patients with a median age of 72 years (42–84) were enrolled. The median number of prior treatment was 1 (1–5). Seven (24%) patients were enrolled in cohort A and 22 (76%) in cohort B. One early death (<30 days) occurred in cohort B due to infection/sepsis. Of 28 evaluable patients, 3 (11%) in cohort B experienced platelet improvement. Median overall survival was 12 months. This study demonstrated modest platelet improvement in some, without evidently increased toxicity or increased risk of leukemia progression.

Original languageEnglish (US)
Pages (from-to)2207-2213
Number of pages7
JournalLeukemia and Lymphoma
Volume60
Issue number9
DOIs
StatePublished - Jul 29 2019
Externally publishedYes

Keywords

  • eltrombopag
  • eltrombopag combination with HMA
  • hypomethylating agent
  • Myelodysplastic syndrome
  • post-hypomethylating agent failure MDS

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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