Androgen steroids are male hormones that can stimulate the production of red blood cells (the cells which carry oxygen in the blood) and platelets (cells that help blood clot) and oxandrin is a more recenty approved androgen. Approximately half of FA patients respond well to androgens (male hormones), which stimulate the production of red blood cells, and often, platelets. Sometimes white cell production is stimulated as well. This treatment may be effective for many years, but most patients eventually fail to respond. It is essential that the use of androgens is considered in the context of an eventual bone marrow transplant, as their use may affect adversely the ultimate success of a transplant.
This drug is in a current study: Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia, NCT00243399. The primary purpose of this study is to evaluate the safety of the drug oxandrolone in patients with Fanconi anemia (FA), and secondarily to determine if this drug can help in the treatment of bone marrow failure in these patients. It is hoped that oxandrolone will have less side effects than oxymetholone, the androgen used most frequently in the short-term treatment of bone marrow failure in FA patients. The study s to prove its superiority but it is not expected to be inferior to any other androgen.
Alter BP. Inherited bone marrow failure syndromes. In: Nathan DG, Orkin SH, Ginsburg D, Look T, eds. Hematology of Infancy and Childhood. 6th ed. Philadelphia, Pa: Harcourt Health Sciences; 2003:280-365.
Bagby GC, Alter BP. Fanconi anemia. Semin Hematol. Jul 2006;43(3):147-56.