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

Muscle Nerve. 2013 Jun;47(6):816-22. doi: 10.1002/mus.23759. Epub 2013 Mar 20.

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.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Androgen Antagonists / therapeutic use*
  • Chlormadinone Acetate / therapeutic use
  • Delayed-Action Preparations / therapeutic use
  • Disease Progression
  • Humans
  • Knee
  • Leuprolide / therapeutic use*
  • Longitudinal Studies
  • Male
  • Middle Aged
  • Muscle Strength / drug effects*
  • Muscle Weakness / drug therapy
  • Muscular Disorders, Atrophic / drug therapy*
  • Prospective Studies
  • Treatment Outcome

Substances

  • Androgen Antagonists
  • Delayed-Action Preparations
  • Chlormadinone Acetate
  • Leuprolide