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

• Published in: Leukemia & lymphoma Vol. 60; no. 9; pp. 2207 - 2213
• Main Authors: Swaminathan, Mahesh, Borthakur, Gautam, Kadia, Tapan M., Ferrajoli, Alessandra, Alvarado, Yesid, Pemmaraju, Naveen, Bodden, Kristy, Yearby, Brittany, Konopleva, Marina, Khoury, Joseph, Bueso-Ramos, Carlos, Garcia-Manero, Guillermo, DiNardo, Courtney D.
• Format: Journal Article
• Language: English
• Published: United States Taylor & Francis 29.07.2019
• Subjects: eltrombopag; eltrombopag combination with HMA; hypomethylating agent; Myelodysplastic syndrome; post-hypomethylating agent failure MDS; eltrombopag combination with HMA; hypomethylating agent; eltrombopag; Myelodysplastic syndrome; post-hypomethylating agent failure MDS
• ISSN: 1042-8194; 1029-2403
• DOI: 10.1080/10428194.2019.1576873

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. Trial registration: ClinicalTrials.gov identifier: NCT01893372.