Document Detail

Eltrombopag and improved hematopoiesis in refractory aplastic anemia.
MedLine Citation:
PMID:  22762314     Owner:  NLM     Status:  MEDLINE    
BACKGROUND: Severe aplastic anemia, which is characterized by immune-mediated bone marrow hypoplasia and pancytopenia, can be treated effectively with immunosuppressive therapy or allogeneic transplantation. One third of patients have disease that is refractory to immunosuppression, with persistent, severe cytopenia and a profound deficit in hematopoietic stem cells and progenitor cells. Thrombopoietin may increase the number of hematopoietic stem cells and progenitor cells.
METHODS: We conducted a phase 2 study involving patients with aplastic anemia that was refractory to immunosuppression to determine whether the oral thrombopoietin mimetic eltrombopag (Promacta) can improve blood counts. Twenty-five patients received eltrombopag at a dose of 50 mg, which could be increased, as needed, to a maximum dose of 150 mg daily, for a total of 12 weeks. Primary end points were clinically significant changes in blood counts or transfusion independence. Patients with a response continued to receive eltrombopag.
RESULTS: Eleven of 25 patients (44%) had a hematologic response in at least one lineage at 12 weeks, with minimal toxic effects. Nine patients no longer needed platelet transfusions (median increase in platelet count, 44,000 per cubic millimeter). Six patients had improved hemoglobin levels (median increase, 4.4 g per deciliter); 3 of them were previously dependent on red-cell transfusions and no longer needed transfusions. Nine patients had increased neutrophil counts (median increase, 1350 per cubic millimeter). Serial bone marrow biopsies showed normalization of trilineage hematopoiesis in patients who had a response, without increased fibrosis. Monitoring of immune function revealed no consistent changes.
CONCLUSIONS: Treatment with eltrombopag was associated with multilineage clinical responses in some patients with refractory severe aplastic anemia. (Funded by the National Heart, Lung, and Blood Institute; number, NCT00922883.).
Matthew J Olnes; Phillip Scheinberg; Katherine R Calvo; Ronan Desmond; Yong Tang; Bogdan Dumitriu; Ankur R Parikh; Susan Soto; Angelique Biancotto; Xingmin Feng; Jay Lozier; Colin O Wu; Neal S Young; Cynthia E Dunbar
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Publication Detail:
Type:  Clinical Trial, Phase II; Journal Article    
Journal Detail:
Title:  The New England journal of medicine     Volume:  367     ISSN:  1533-4406     ISO Abbreviation:  N. Engl. J. Med.     Publication Date:  2012 Jul 
Date Detail:
Created Date:  2012-07-05     Completed Date:  2012-07-11     Revised Date:  2013-07-12    
Medline Journal Info:
Nlm Unique ID:  0255562     Medline TA:  N Engl J Med     Country:  United States    
Other Details:
Languages:  eng     Pagination:  11-9     Citation Subset:  AIM; IM    
Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
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MeSH Terms
Anemia, Aplastic / drug therapy*,  pathology
Benzoates / administration & dosage,  adverse effects,  therapeutic use*
Bone Marrow Cells / cytology
Chronic Disease
Drug Resistance
Hydrazines / administration & dosage,  adverse effects,  therapeutic use*
Middle Aged
Pyrazoles / administration & dosage,  adverse effects,  therapeutic use*
Young Adult
Grant Support
Reg. No./Substance:
0/Benzoates; 0/Hydrazines; 0/Pyrazoles; 0/eltrombopag
Comment In:
N Engl J Med. 2012 Sep 20;367(12):1162; author reply 1163   [PMID:  22992084 ]
N Engl J Med. 2012 Jul 5;367(1):74-5   [PMID:  22762322 ]
N Engl J Med. 2012 Sep 20;367(12):1162-3; author reply 1163   [PMID:  22992085 ]
Erratum In:
N Engl J Med. 2012 Jul 19;367(3):284

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