Aplastic anemia

What is the long-term outlook for aplastic anemia?

A small number of people with aplastic anemia may spontaneously recover with supportive care; however, for most individuals, the condition worsens without identification and treatment of the underlying cause and/or treatment of the disease. Bone marrow transplant may cure the disease in children and young patients and has a 10 year survival rate of approximately 73%. For many, bone marrow transplant is not an option due to the risks and potential long-term side effects.

Last updated on 05-01-20

How might aplastic anemia be treated?

Treatment for aplastic anemia varies depending on the severity. While some individuals with mild to moderate aplastic anemia may not require treatment, for others, treatment may include:

  • Blood transfusions to keep blood cell counts at acceptable levels
  • Blood and marrow stem cell transplants to replace damaged stem cells with health ones from a donor (another person)
  • Medications to stimulate the bone marrow, suppress the immune system, and prevent and treat infections

Blood and marrow stem cell transplants may cure aplastic anemia in some instances. This treatment option works best in children and young adults with severe aplastic anemia who are otherwise in good health.

For patients with severe aplastic anemia who are under the age of 20 years, and those ages 20 to 50 years who are otherwise in good health the first option is the transplant when a sibling donor is available. For those who do not have an available sibling donor, the medication eltrombopag or eltrombopag plus immunesuppression therapy can be used.

For patients over 50 years of age, the decision is based on the patient’s overall health, and preferences, and treatment may include eltrombopag or eltrombopag plus immunesuppression therapy (horse anti-thymocyte globulin (ATG), cyclosporin A (CSA), and glucocorticoids). People older than 50 years old have more risks of having rejection with the transplant and have greater risks of treatment toxicity and early mortality.

Last updated on 05-01-20

Name: American Cancer Society 250 Williams Street NW
Atlanta, GA, 30329, United States
Toll Free: 1-800-227-2345 Url: https://www.cancer.org
Name: Aplastic Anemia and MDS International Foundation 4330 East-West Highway, Suite 230
Bethesda, MD, 20814 , United States
Phone: +1-301-279-7202 Toll Free: 1-800-747-2820 Option 2 (Helpline) Fax : +1-301-279-7205 Email: help@aamds.org Url: https://www.aamds.org
Name: Aplastic Anemia and Myelodysplasia Association of Canada AAMAC 11181 Yonge Street Suite 321
Richmond Hill, ON, Canada
Phone: 905-780-0698 Toll Free: 1-888-840-0039 Fax : 905-780-1648 Email: info@aamac.ca Url: http://www.aamac.ca/

Note, these links are external searches against the National Laboratory of Medicine's drug database. You may need to adjust the search if there are no results found.

Drug Name Generic Name
Promacta Promacta

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