Fanconi Anemia Medication
- Author: Jeffrey M Lipton, MD, PhD; Chief Editor: Max J Coppes, MD, PhD, MBA more...
Medication Summary
Fanconi anemia is one of the few forms of aplastic anemia in which the response to androgens is more than 50%.
Other agents used in the management of Fanconi anemia include antifibrinolytic agents, hematopoietic growth factors, and glucocorticoids.
Androgenic agents
Class Summary
These enhance the production and urinary excretion of erythropoietin in anemias caused by bone marrow failure and often stimulate erythropoiesis in anemias caused by deficient red cell production. They appear to make hematopoietic stem cells more responsive to differentiation, but the exact mechanism is not clear. The usual agent in the United States is oral oxymetholone, a 17-beta-hydroxylated androgen. Although oral androgens have a risk of liver toxicity, they are easier to use in children than parenteral androgens. The lowest effective dose should be used.
Oxymetholone (Anadrol-50)
This is an anabolic and androgenic derivative of testosterone in an oral formulation.
17 Alpha-ethynyl testosterone (Danazol, Danocrine)
A parenteral fat-soluble androgen has been studied experimentally and is sometimes selected because it is less virilizing, although the results of a large ongoing trial are not published. The risk of hepatic tumors compared with other androgens has not been determined. As with oxymetholone, the lowest effective dose should be used.
Antifibrinolytic agents
Class Summary
These agents may decrease bleeding, particularly oral mucosal bleeding, in patients with thrombocytopenia by stabilization of thrombi.
Aminocaproic acid (Amicar)
This medication competitively inhibits activation of plasminogen to plasmin.
Hematopoietic growth factors
Class Summary
These factors are glycoproteins that act on hematopoietic cells by binding to specific cell surface receptors and stimulating proliferation, differentiation, commitment, and some end cell functional activation.
Filgrastim (G-CSF, Neupogen)
Filgrastim is a G-CSF that activates and stimulates the production, maturation, migration, and cytotoxicity of neutrophils.
Epoetin alfa (Epogen, Procrit)
Epoetin alfa stimulates the division and differentiation of committed erythroid progenitor cells; it induces the release of reticulocytes from bone marrow into the bloodstream.
Glucocorticoids
Class Summary
Corticosteroids are used on alternate days and may delay the growth acceleration caused by androgens. They may also stabilize endothelial cells, leading to reduced bleeding at a given degree of thrombocytopenia. Most clinicians no longer use corticosteroids.
Prednisone (Deltasone, Liquid Pred)
Prednisone elicits anti-inflammatory properties and causes profound and varied metabolic effects. It modifies the body's immune response to diverse stimuli.
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