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NEJM: Addition of Eltrombopag to Immunosuppressive Therapy for Severe Aplastic Anemia

 Last Update: 2022-01-22
 Source: Internet
 Author: User

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Acquired aplastic anemia is a blood disorder involving primary bone marrow failure manifesting as pancytopenia

immunity

Eltrombopag improves efficacy of standard immunosuppressive therapy including equine antithymocyte globulin [ATG] and cyclosporine in patients with severe aplastic anemia , a single-arm phase 1-2 study shows .

Eltrombopag improves the efficacy of standard immunosuppressive therapy, including equine antithymocyte globulin [ATG] and cyclosporine, in patients with severe aplastic anemia

New England published a prospective, investigator-led, open-label, multicenter, randomized phase 3 trial comparing equine ATG combined with cyclosporine ± eltrombopag as a previously untreated severe Efficacy and safety of first-line therapy in patients with aplastic anemia

Research design

research design research design

Hematologic complete remission rate at 3 months

The patients were randomized to receive immunosuppressive therapy (group A, 101 patients) or immunosuppressive therapy + eltrombopag (group B, 96 patients)

The complete remission rates at 3 months in Arms A and B were 10% and 22%, respectively The complete remission rates at 3 months in Arms A and B were 10% and 22%, respectively

Remission rate at 6 months

At 6 months, the overall response rates (including complete and partial responses) were 41% and 68% in arm A and arm B, respectively

The overall response rates (including complete and partial responses) were 41% and 68% in arm A and 68%, respectively.

event-free survival

The incidence of serious adverse events was similar in the two groups

The event-free survival rates were 34% and 46% , respectively The event-free survival rates were 34% and 46%, respectively

In conclusion, the addition of eltrombopag to standard immunosuppressive therapy improved hematologic response rate, speed of response, and depth of response in previously untreated patients with severe aplastic anemia without additional toxic effects

Addition of eltrombopag to standard immunosuppressive therapy improves hematologic response rate, speed of response, and depth of response in previously untreated patients with severe aplastic anemia without additional toxic effects Trombopag increases hematologic response rate, speed and depth of response in previously untreated patients with severe aplastic anemia without additional toxic effects

Original source:

Peffault de Latour Régis,Kulasekararaj Austin,Iacobelli Simona et al.

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