Your browser does not support javascript:   Search for gard hereSearch for news-and-events here.

Diseases

Genetic and Rare Diseases Information Center (GARD)

Print friendly version

Congenital dyserythropoietic anemia type 2


Other Names for this Disease
  • Anemia, dyserythropoietic, congenital type 2
  • CDA
  • CDA II
  • CDA type 2
  • CDA type II
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

Newline Maker

How might congenital dyserythropoietic anemia (CDA) type 2 be treated?

The goal of CDA type 2 treatment is to address and prevent complications from anemia and iron overload.[1] Most people with CDA type 2 develop iron overload, for some this is as early as in their 20's.[1]  If a person with CDA type 2 has mild anemia, but evidence of iron loading, treatment may involve phlebotomy.[1] An alternative treatment is chelation therapy.[1][2] In particular, chelation therapy is preferred for people with iron (ferritin) levels greater than 1000 mg/L.[1] The Iron Disorders Institute provides information on chelation therapy through their Web site at:
http://www.irondisorders.org/chelation-therapy

Many people with CDA-2 maintain hemoglobin levels just above the threshold for symptoms.[2] Mild anemia may not need treatment, as long as it doesn't worsen.  Less commonly CDA-2 causes severe anemia. Treatment of severe anemia may involve blood transfusions. Blood transfusions can raise iron levels so, careful monitoring and treatment for iron overload is required.

The National Heart, Lung, and Blood Institute offers tips for living with hemolytic anemia at the following link:
http://www.nhlbi.nih.gov/health/health-topics/topics/ha/livingwith

Splenectomy is considered for people with CDA-2 and severe anemia. Splenectomy can cause a consistent rise in hemoglobin values.[1] The spleen, however, is important in fighting infection.  People, particularly children, who have had a splenectomy are more likely to contract a serious and possibly life-threatening infection (sepsis). This risk must be carefully weighed. Splenectomy does not affect iron overload.

Lastly, people with very severe CDA-2 may be candidates for hematopoietic stem cell transplantation (HSCT). Currently this is the only available curative treatment for CDA-2.[1][2][3][4]
Last updated: 6/25/2015

References
  1. Renella R, Wood WG. The congenital dyserythropoietic anemias. Hematol Oncol Clin North Am. 2009 Apr; 23(2):283-306. Accessed 6/25/2015.
  2. Renella R. Progress in the congenital dyserythropoietic anemias: juicy but high-hanging fruits?. Am J Hematol. 2010 Dec; 85(12):913-4. Accessed 6/25/2015.
  3. Unal S. Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II. Pediatr Transplant. 2014 Jun; 18(4):E130-3. Accessed 6/25/2015.
  4. Braun M. Successful treatment of an infant with CDA type II by intrauterine transfusions and postnatal stem cell transplantation. Pediatr Blood Cancer. 2014 Apr; 61(4):743-5. Accessed 6/25/2015.


GARD Video Tutorial

  • Finding Treatment Information - A video developed by GARD Information Specialists that explains how you can find information about treatment for a rare disease.

    Finding Treatment Information
Other Names for this Disease
  • Anemia, dyserythropoietic, congenital type 2
  • CDA
  • CDA II
  • CDA type 2
  • CDA type II
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.