National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Diamond-Blackfan anemia

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Other than bone marrow transplantation is there any other remedy for Diamond-Blackfan anemia?
Some people have such mild signs and symptoms that they do not require treatment. In people who require treatment it may include:[1]
  • Corticosteroids: Corticosteroid treatment is recommended in children over 1 year of age; this treatment can initially improve the red blood count in approximately 80% of people with Diamond-Blackfan anemia. Prednisone initial dose is 2 mg / kg / day given orally once a day, at morning time.  After a month, if there is no improvement after a month the corticosteroids are tapered-of and suspended
  • Blood transfusions, which are given along with the corticosteroids or in people who do not get better with corticosteroids
  • Bone marrow/stem cell transplantation: It is the only curative treatment for the anemia; however, patients should continue to be followed because they are at increased risk for leukemia and cancer. Results are better for children younger than ten years of age if transplanted using an Human Leukocyte Antigen (HLA)-matched sib
Last updated: 12/1/2017

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  1. Clinton, C & Gazda, HT. Diamond Blackfan Anemia. GeneReviews. 2016; http://www.ncbi.nlm.nih.gov/books/NBK7047/.