A pilot randomized trial of oxandrolone in inclusion body myositis.
BACKGROUND
Inclusion body myositis (IBM) remains without effective therapy. As anabolic steroids have myotrophic properties, the authors studied whether a synthetic androgen, oxandrolone, would have efficacy in IBM.


METHODS
A double-blind, placebo-controlled, crossover design was used. Patients received oxandrolone or placebo for 12 weeks followed by a minimum 2-month washout period, followed by 12 weeks of the alternative treatment. Maximal voluntary isometric contraction testing (MVICT), manual muscle testing (MMT), and functional performance testing were obtained before and after each treatment period, with the whole-body MVICT score as the primary outcome measure.


RESULTS
Of 19 patients enrolled, 16 (14 men, 2 women; median age 68.5 years) had complete data for at least the first treatment period, with 13 completing the entire study. Whole-body MVICT improved by a median of 15.5 kg with drug and 4.1 kg with placebo (p = 0.06), whereas MMT demonstrated a median increase of 2.0 Medical Research Council points with drug and 0.9 point with placebo (p = 0.33). Upper-extremity MVICT demonstrated a significant treatment effect, with strength increasing a median 6.3 kg with drug vs 2.5 kg with placebo (p = 0.006). Stair climbing also increased a median of 1 step on average with drug versus no change with placebo (p < 0.001). Minimal adverse effects occurred.


CONCLUSIONS
Oxandrolone had a borderline significant effect in improving whole-body strength and a significant effect in improving upper-extremity strength as measured by MVICT. Given these findings, further study of this drug, possibly in combination with an immunomodulating agent, is warranted.
Rutkove
S B
SB
Department of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA 02215, USA. srutkove@caregroup.harvard.edu
Parker
R A
RA
Nardin
R A
RA
Connolly
C E
CE
Felice
K J
KJ
Raynor
E M
EM
D000368:Aged; D045930:Anabolic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D018592:Cross-Over Studies; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007537:Isometric Contraction / Q000187:drug effects; D008297:Male; D018979:Myositis, Inclusion Body / Q000188:drug therapy*; D010074:Oxandrolone / Q000009:adverse effects / Q000627:therapeutic use*; D010865:Pilot Projects; D018709:Statistics, Nonparametric
D045930:Anabolic Agents; D010074:Oxandrolone