Patients who are not good candidates for bone marrow transplantation (e.g. because they don’t have an HLA-matched donor) or gene therapy (because they have a rare complementation group) may benefit from hormone therapy. Treatment with synthetic male hormones (androgens) improve blood counts in about half of patients. Treatment plans are developed individually for each patient and modifications are made as needed based on tests and results. Changes in red blood cell counts can usually be seen in the first two months. Improvements in white blood cell and platelet counts may take longer, between six and 12 months.
Side effects of androgen therapy include elevated liver enzymes, cholestasis (decreased bile), and other problems. Other side effects include acne, oily skin, enlarged penis/clitoris, hoarse or deep voice, hair growth or loss, decreased growth, behavior changes, hot flashes, breast enlargement, fluid retention, and secondary hypertension. Receiving androgen therapy puts patients at increased risk for liver tumors in the future.