Fanconi Anemia Medication

Updated: Jul 08, 2022
  • Author: Jeffrey M Lipton, MD, PhD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication Summary

Fanconi anemia is one of the few forms of aplastic anemia in which the response to androgens is more than 50%. Hematopoietic growth factors are occasionally helpful in Fanconi anemia.


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 blood 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. Recent studies suggest that the less potent androgen, danazol, may be effective in delaying the onset of clinically significant cytopenias in patients with Fanconi anemia.

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.