International Journal of Hematology

, Volume 106, Issue 6, pp 765–776 | Cite as

Eltrombopag for thrombocytopenia in patients with advanced solid tumors receiving gemcitabine-based chemotherapy: a randomized, placebo-controlled phase 2 study

  • Eric S. Winer
  • Howard Safran
  • Boguslawa Karaszewska
  • Sebastian Bauer
  • Dilawar Khan
  • Steffen Doerfel
  • Paul Burgess
  • Stacey Kalambakas
  • Yasser Mostafa Kamel
  • Frederic Forget
Original Article


In this phase 2 study, patients with solid tumors receiving gemcitabine monotherapy or gemcitabine plus cisplatin/carboplatin were randomized 2:1 to eltrombopag 100 mg (n = 52) or placebo (n = 23) for 5 days before and after chemotherapy was started. The primary endpoint was prechemotherapy (Day 1) platelet count across ≤6 cycles. Prechemotherapy platelet counts were numerically higher with eltrombopag than placebo. Frequencies of grades 3/4 thrombocytopenia were lower with eltrombopag in both the combination therapy (77 vs. 100%) and monotherapy (36 vs. 42%) groups. Proportionately fewer eltrombopag-treated patients had platelet counts <100 × 109/L at nadir. Among patients receiving combination chemotherapy, mean time to recovery from platelet nadir was 8 days with eltrombopag vs. 15 days with placebo. Eltrombopag-treated patients had fewer dose delays/reductions or missed doses due to thrombocytopenia in both the combination therapy (77 vs. 91%) and monotherapy (62 vs. 83%) groups. Adverse events and serious adverse events were less frequent with eltrombopag in both chemotherapy groups, with reduced rates of anemia, neutropenia, and thrombocytopenia in patients receiving combination chemotherapy. In conclusion, eltrombopag treatment shortened the time to recovery from platelet nadir in patients treated with gemcitabine-based chemotherapy and reduced dose delays/reductions due to thrombocytopenia.


Blood platelets Cancer chemotherapy drugs Eltrombopag Thrombocytopenia Gemcitabine 



Funding for this study (NCT01147809 available from was provided by GlaxoSmithKline; however, as of March 2, 2015, eltrombopag is an asset of Novartis AG. We thank Vassilios Aslanis, PharmD, of Novartis Pharmaceuticals Corporation, for his critical review of the manuscript and the pharmacology data. We also thank the patients and principal investigators and their institutions for their contributions to the study. All listed authors meet the criteria for authorship set forth by the International Committee for Medical Journal Editors. Editorial support (assembling tables and figures, collating author comments, copyediting, fact checking, and referencing) and graphic services were provided by Elizabeth Rosenberg, PhD, and Nancy E. Price, PhD, of AOI Communications, L.P., and were funded by Novartis Pharmaceuticals Corporation.

Compliance with ethical standards

Conflict of interest

EW, HS, BK, and SD have nothing to disclose. SB has provided consultancy for Blueprint Medicine, Novartis, and Pfizer. DK received honoraria from and provided consultancy for Incyte, Pfizer, and Pharmacyclics. PB held stock in and was an employee of GSK during the time of the study conduct and is now an employee of Novartis Pharma AG. SK was an employee of Novartis Pharmaceuticals Corporation and holds stock in Novartis. YMK was an employee of GlaxoSmithKline (GSK) during the time of study conduct and Novartis Pharmaceuticals Corporation, held GSK stock, and received travel accommodations and expenses from GSK. FF has received research funding from GSK.


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Copyright information

© The Japanese Society of Hematology 2017

Authors and Affiliations

  • Eric S. Winer
    • 1
  • Howard Safran
    • 2
  • Boguslawa Karaszewska
    • 3
  • Sebastian Bauer
    • 4
  • Dilawar Khan
    • 5
  • Steffen Doerfel
    • 6
  • Paul Burgess
    • 7
  • Stacey Kalambakas
    • 8
  • Yasser Mostafa Kamel
    • 8
  • Frederic Forget
    • 9
  1. 1.Dana Farber Cancer InstituteBostonUSA
  2. 2.Brown University Oncology Research GroupProvidenceUSA
  3. 3.Komed Branch Medical CenterKoninPoland
  4. 4.Department of Medical OncologyUniversity Hospital Essen, University of Duisburg-EssenEssenGermany
  5. 5.Harbin ClinicRomeUSA
  6. 6.Onkozentrum DresdenDresdenGermany
  7. 7.Novartis Pharma AGBaselSwitzerland
  8. 8.Novartis Pharmaceuticals CorporationEast HanoverUSA
  9. 9.Center Hospital of the ArdenneLibramontBelgium

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