TY - JOUR
T1 - A phase 2 clinical trial of eltrombopag for treatment of patients with myelodysplastic syndromes after hypomethylating-agent failure
AU - Swaminathan, Mahesh
AU - Borthakur, Gautam
AU - Kadia, Tapan M.
AU - Ferrajoli, Alessandra
AU - Alvarado, Yesid
AU - Pemmaraju, Naveen
AU - Bodden, Kristy
AU - Yearby, Brittany
AU - Konopleva, Marina
AU - Khoury, Joseph
AU - Bueso-Ramos, Carlos
AU - Garcia-Manero, Guillermo
AU - DiNardo, Courtney D.
N1 - Funding Information:
This clinical trial was supported by Novartis.
Publisher Copyright:
© 2019, © 2019 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2019/7/29
Y1 - 2019/7/29
N2 - 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.
AB - 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.
KW - eltrombopag
KW - eltrombopag combination with HMA
KW - hypomethylating agent
KW - Myelodysplastic syndrome
KW - post-hypomethylating agent failure MDS
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U2 - 10.1080/10428194.2019.1576873
DO - 10.1080/10428194.2019.1576873
M3 - Article
C2 - 30773968
AN - SCOPUS:85072700892
SN - 1042-8194
VL - 60
SP - 2207
EP - 2213
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 9
ER -