An open trial of long‐term testosterone suppression in spinal and bulbar muscular atrophy. Issue 6 (20th March 2013) - Yamamoto, Tomotaka
ABSTRACT Introduction We investigated the long‐term effects of leuprorelin on leg‐muscle strength in spinal and bulbar muscular atrophy (SBMA). We hypothesized that testosterone suppression by leuprorelin would prevent the progression of muscle weakness. Methods In a prospective, long duration, open trial, 16 SBMA patients underwent medical castration with leuprorelin for 3.5 years. Chlormadinone was coadministered initially to prevent a testosterone surge. The strength of knee extension and flexion were quantitated using a torque machine. Results Our hypothesis was rejected. The leg strength measures decreased significantly with the mean reduction of 22.3–27.8%. In a post hoc analysis, the leg strength of 4 patients with higher pretreatment baseline total testosterone levels and short disease duration of 1–6 years were stronger at baseline and decreased by only 12.3–15.7% after treatment. Conclusions Leuprorelin was not effective in this small long‐term treatment trial in SBMA. The possibility that earlier treatment might be beneficial may deserve further study. Muscle Nerve 47: 816–822, 2013