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Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia

The New England journal of medicine, 2017-04, Vol.376 (16), p.1540-1550 [Peer Reviewed Journal]

Copyright © 2017 Massachusetts Medical Society. All rights reserved. ;ISSN: 0028-4793 ;EISSN: 1533-4406 ;DOI: 10.1056/NEJMoa1613878 ;PMID: 28423296

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Title:
Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia
Author: Townsley, Danielle M ; Scheinberg, Phillip ; Winkler, Thomas ; Desmond, Ronan ; Dumitriu, Bogdan ; Rios, Olga ; Weinstein, Barbara ; Valdez, Janet ; Lotter, Jennifer ; Feng, Xingmin ; Desierto, Marie ; Leuva, Harshraj ; Bevans, Margaret ; Wu, Colin ; Larochelle, Andre ; Calvo, Katherine R ; Dunbar, Cynthia E ; Young, Neal S
Subjects: Adolescent ; Adult ; Age ; Aged ; Analysis ; Anemia ; Anemia, Aplastic - drug therapy ; Antigens, CD34 ; Antilymphocyte Serum - therapeutic use ; Aplastic anemia ; Benzoates - adverse effects ; Benzoates - therapeutic use ; Bone marrow ; Cancer ; CD34 antigen ; Cell Count ; Cell number ; Cyclosporine - therapeutic use ; Dosage and administration ; Drug therapy ; Drug Therapy, Combination ; Eltrombopag ; Female ; Hematologic Agents - adverse effects ; Hematologic Agents - therapeutic use ; Hemopoiesis ; Humans ; Hydrazines - adverse effects ; Hydrazines - therapeutic use ; Immunosuppression ; Immunosuppressive agents ; Immunosuppressive Agents - therapeutic use ; Male ; Middle Aged ; Patient outcomes ; Prospective Studies ; Pyrazoles - adverse effects ; Pyrazoles - therapeutic use ; R&D ; Receptors, Thrombopoietin - agonists ; Research & development ; Thrombopoietin ; Treatment outcome ; Young Adult
Is Part Of: The New England journal of medicine, 2017-04, Vol.376 (16), p.1540-1550
Description: In a phase 2 clinical trial, eltrombopag plus standard immunosuppression resulted in a 6-month complete-response rate of 58% among patients receiving eltrombopag for 6 months. Immunosuppression alone has induced complete responses in approximately 10% of patients historically. In patients with aplastic anemia, the “empty” bone marrow does not produce blood cells, and when pancytopenia is severe and untreated, almost all patients die within 1 year after diagnosis. 1 Acquired aplastic anemia has immune pathophysiological features, as inferred from numerous laboratory studies and animal models. 2 Patients are cured by the replacement of both the hematopoietic and immune systems in a stem-cell transplant, but immunosuppressive therapy alone is also effective. Approximately two thirds of patients have increased blood-cell production after a single course of horse antithymocyte globulin and cyclosporine. 3 – 5 Hematologic response is strongly correlated with long-term survival. 6 Many efforts . . .
Publisher: United States: Massachusetts Medical Society
Language: English
Identifier: ISSN: 0028-4793
EISSN: 1533-4406
DOI: 10.1056/NEJMoa1613878
PMID: 28423296
Source: ProQuest One Psychology
MEDLINE
ProQuest Central

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Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia

Copyright © 2017 Massachusetts Medical Society. All rights reserved. ;ISSN: 0028-4793 ;EISSN: 1533-4406 ;DOI: 10.1056/NEJMoa1613878 ;PMID: 28423296

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