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      Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia

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      New England Journal of Medicine
      Massachusetts Medical Society

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          Aplastic Anemia

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            Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia.

            In patients with acquired aplastic anemia, destruction of hematopoietic cells by the immune system leads to pancytopenia. Patients have a response to immunosuppressive therapy, but myelodysplastic syndromes and acute myeloid leukemia develop in about 15% of the patients, usually many months to years after the diagnosis of aplastic anemia.
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              Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

              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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                Author and article information

                Contributors
                (View ORCID Profile)
                Journal
                New England Journal of Medicine
                N Engl J Med
                Massachusetts Medical Society
                0028-4793
                1533-4406
                January 06 2022
                January 06 2022
                : 386
                : 1
                : 11-23
                Affiliations
                [1 ]From the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital and Université de Paris (R.P.L., F.S.F., C.F., G.S.), and the Adolescent and Young Adult Hematology Unit, Saint-Louis Hospital (F.R.), Paris, the Hematology Department, Centre Hospitalier Universitaire de Toulouse, Institut Universitaire du Cancer de Toulouse Oncopole, Toulouse (C.R., S.T.), Centre Hospitalier Universitaire Lyon Sud, Lyon (F.B., A.P.), Centre Hospitalier Universitaire de...
                Article
                10.1056/NEJMoa2109965
                34986284
                5c179002-e5af-4e03-b4f3-aa9536238e42
                © 2022

                http://www.nejmgroup.org/legal/terms-of-use.htm

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