Improvement in the treatment of autoimmune aplastic anemia

  • The addition of Eltrombopag to conventional treatment with immunosuppressants increases the effectiveness and speed of treatment.
  • The study, published in the New England Journal of Medicine, is a phase 3 clinical trial involving IDIBELL and ICO.
  • Aplastic anemia is a bone marrow disorder that prevents the formation of blood cells and causes fatigue, susceptibility to infections and bleeding.
NP02 - Sureda_NEJM - Imatge

Aplastic anemia is a condition that occurs when the production of blood cells in the bone marrow is insufficient. This deficiency causes fatigue, susceptibility to infections, and may even be the trigger of uncontrolled bleeding. There are many factors that can lead to this type of anemia: chemotherapy treatments, exposure to toxic substances, viral infections, among others; but the most common ones are autoimmune disorders that impair proper bone marrow function.

Treatment with immunosuppressants is the standard therapy to treat aplastic anemia, as it lowers the attacks of the immune system to the bone marrow itself. However, the effectiveness of these drugs in reducing anemia is limited, and in between 10 and 15% of cases it can be the cause of some type of myeloid cancer.

Now, a phase 3 clinical trial has been published in the New England Journal of Medicine which shows that the addition of Eltrombopag to immunosuppressive therapy significantly improves the effectiveness of the treatment for anemia. The multicenter study had the participation of Dr. Anna Sureda, group leader at the Bellvitge Biomedical Research Institute (IDIBELL) and head of the Clinical Hematology Service at the Catalan Institute of Oncology (ICO) of L’Hospitalet.

“While immunosuppressive drugs prevent the immune system from attacking the bone marrow and allow it to work, Eltrombopag stimulates the proliferation and differentiation of blood cells,” says Dr. Sureda.

The results of the study show that the combination of drugs increases the percentage of patients who respond to therapy: from 41% with only immunosuppressants to 68% when adding Eltrombopag. It also improves the response time to treatment from almost nine months to three. All this without showing any additional side effects.

As indicated by Dr. Sureda: “In the last 30 years none of the efforts to improve therapy against aplastic anemia have been successful, this work is the first to show a significant improvement in the quality and speed of hematological recovery.”



The Bellvitge Biomedical Research Institute (IDIBELL) is a biomedical research center created in 2004. It is participated by the Bellvitge University Hospital and the Viladecans Hospital of the Catalan Institute of Health, the Catalan Institute of Oncology, the University of Barcelona and the City Council of L’Hospitalet de Llobregat.

IDIBELL is a member of the Campus of International Excellence of the University of Barcelona HUBc and is part of the CERCA institution of the Generalitat de Catalunya. In 2009 it became one of the first five Spanish research centers accredited as a health research institute by the Carlos III Health Institute. In addition, it is part of the “HR Excellence in Research” program of the European Union and is a member of EATRIS and REGIC. Since 2018, IDIBELL has been an Accredited Center of the AECC Scientific Foundation (FCAECC).

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