Blood Journal
Leading the way in experimental and clinical research in hematology

CME Article
Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug

  1. Ronan Desmond1,
  2. Danielle M. Townsley1,
  3. Bogdan Dumitriu1,
  4. Matthew J. Olnes2,
  5. Phillip Scheinberg3,
  6. Margaret Bevans4,
  7. Ankur R. Parikh1,
  8. Kinneret Broder1,
  9. Katherine R. Calvo5,
  10. Colin O. Wu6,
  11. Neal S. Young1, and
  12. Cynthia E. Dunbar1
  1. 1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;
  2. 2Hematology Department, Alaska Native Tribal Health Consortium, Anchorage, AK;
  3. 3Hospital Sao Jose – Hospital Beneficencia Portuguesa de Sao Paulo, Sao Paulo, Brazil;
  4. 4Nursing Research and Translational Science, National Institutes of Health, Bethesda, MD;
  5. 5Laboratory Medicine, National Institutes of Health, Bethesda, MD; and
  6. 6Office of Biostatistics Research, National Heart, Lung, and Blood Institute, Bethesda, MD

Key Points

  • Eltrombopag promotes hematopoiesis in patients with severe aplastic anemia by stimulating stem and progenitor cells.

  • Eltrombopag can be discontinued safely in robust responders with maintenance of hematopoiesis.


About a quarter of patients with severe aplastic anemia remain pancytopenic despite immunosuppressive therapy. We have previously demonstrated that eltrombopag has efficacy in this setting with 44% (11/25) of patients having clinically significant hematologic responses. We now report safety and efficacy data on a further 18 patients and long-term follow-up on the entire cohort of 43 patients. The overall response rate was 17 of 43 patients (40%) at 3 to 4 months, including tri- and bilineage responses. The majority of patients who remained on eltrombopag in an extension study (14/17) continued to show improvement, and 7 eventually had significant increases in neutrophil, red cell, and platelet lineages. Five patients with robust near-normalization of blood counts had drug discontinued at a median of 28.5 months after entry (range, 9-37 months), and all maintained stable counts a median of 13 months (range, 1-15 months) off eltrombopag. Eight patients, including 6 nonresponders and 2 responders, developed new cytogenetic abnormalities on eltrombopag, including 5 with chromosome 7 loss or partial deletion. None evolved to acute myeloid leukemia to date. Eltrombopag is efficacious in a subset of patients with aplastic anemia refractory to immunosuppressive therapy, with frequent multilineage responses and maintenance of normalized hematopoiesis off treatment. This study is registered at as #NCT00922883.

  • Submitted October 24, 2013.
  • Accepted December 7, 2013.
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