A phase 2 clinical trial of eltrombopag for treatment of patients with myelodysplastic syndromes after hypomethylating-agent failure. Issue 9 (29th July 2019)

• Record Type: Journal Article
• 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.
• Is Part Of: Leukemia & lymphoma. Volume 60:Issue 9(2019)
• Page Start: 2207
• Page End: 2213
• Publication Date: 2019-07-29
• Subjects: Myelodysplastic syndrome -- eltrombopag -- hypomethylating agent -- post-hypomethylating agent failure MDS -- eltrombopag combination with HMA; Leukemia -- Periodicals; Lymphomas -- Periodicals; 616.99419
• DOI: 10.1080/10428194.2019.1576873
• Languages: English
• ISSNs: 1042-8194

Abstract

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. Trial registration: ClinicalTrials.gov identifier: NCT01893372.