An open trial of long‐term testosterone suppression in spinal and bulbar muscular atrophy

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