Endocrine Treatment Helps Patients with Rare Telomere Diseases

Several mutations cause telomeres — the nucleotides and proteins at the ends of chromosomes — to shorten more readily with each cell division, which leads to cellular aging and “telomere diseases.” Telomere diseases include dyskeratosis congenita in children, aplastic anemia, pulmonary fibrosis, and liver cirrhosis. The androgenic drug, danazol, encourages expression of telomerase, a telomere-lengthening enzyme, and improves blood cell counts in patients with marrow-failure syndromes.

An international team conducted a 2-year, uncontrolled study of danazol (800 mg daily) in 12 patients with various telomere diseases. Compared with telomere lengths and blood cell counts at baseline, telomere lengths at 24 months were longer, and blood cell cou…