Abstract
Eltrombopag has been added to first-line treatment of immune aplastic anaemia (AA), resulting in higher responses. We analysed marrow samples of AA patients who responded to immunosuppressive therapy (IST) alone or in combination with eltrombopag for the composition of the haematopoietic stem and progenitor cell (HSPC) compartment. The number of CD34+ cells and multipotent progenitors was higher in patients treated with eltrombopag (P < 0·005; P < 0·05; respectively), but not the number of stem cells. No aberrant phenotype was observed. These results indicate that eltrombopag augments CD34+ cells in vivo and preferentially expands multipotent progenitors, but not stem cells.
Keywords:
aplastic anaemia; eltrombopag; flow cytometry; haematopoietic stem cells; thrombopoietin receptor agonist.
© 2020 British Society for Haematology and John Wiley & Sons Ltd.
Publication types
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Comparative Study
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Research Support, Non-U.S. Gov't
MeSH terms
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Adolescent
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Adult
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Anemia, Aplastic / drug therapy*
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Antigens, CD34 / drug effects
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Benzoates / administration & dosage
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Benzoates / pharmacology*
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Biopsy, Needle / methods
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Bone Marrow Cells / cytology
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Bone Marrow Cells / drug effects
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Bone Marrow Cells / pathology
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Brazil / epidemiology
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Female
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Flow Cytometry / methods
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Hematopoietic Stem Cells / cytology
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Hematopoietic Stem Cells / drug effects*
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Humans
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Hydrazines / administration & dosage
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Hydrazines / pharmacology*
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Immunosuppressive Agents / therapeutic use
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Male
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Middle Aged
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Multipotent Stem Cells / cytology
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Multipotent Stem Cells / drug effects*
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Pyrazoles / administration & dosage
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Pyrazoles / pharmacology*
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Receptors, Thrombopoietin / agonists*
Substances
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Antigens, CD34
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Benzoates
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Hydrazines
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Immunosuppressive Agents
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Pyrazoles
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Receptors, Thrombopoietin
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MPL protein, human
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eltrombopag