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Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia
Author(s) -
Danielle M. Townsley,
Phillip Scheinberg,
Thomas Winkler,
Ronan Desmond,
Bogdan Dumitriu,
Olga Rios,
Barbara Weinstein,
Janet Valdez,
Jennifer Lotter,
Xingmin Feng,
Marie J. Desierto,
Harshraj Leuva,
Margaret Bevans,
Colin O. Wu,
André Larochelle,
Katherine R. Calvo,
Cynthia E. Dunbar,
Neal S. Young
Publication year - 2017
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1613878
Subject(s) - eltrombopag , medicine , cohort , aplastic anemia , regimen , immunosuppression , thrombopoietin receptor , gastroenterology , surgery , thrombopoietin , bone marrow , haematopoiesis , immune thrombocytopenia , stem cell , platelet , genetics , biology
Acquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.

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