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 Volume 71, Number 6, August 5, 2008
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NEUROLOGY 2008;71:407-412
© 2008 American Academy of Neurology

Dehydroepiandrosterone for myotonic dystrophy type 1

I. Pénisson-Besnier, MD, M. Devillers, MD, R. Porcher, MD, D. Orlikowski, MD, V. Doppler, MD, C. Desnuelle, MD, PhD, X. Ferrer, MD, M. -C.A. Bes, MD, F. Bouhour, MD, C. Tranchant, MD, E. Lagrange, MD, A. Vershueren, MD, D. Uzenot, MD, P. Cintas, MD, G. Solé, MD, J. -Y. Hogrel, PhD, P. Laforêt, MD, C. Vial, MD, A. L. Vila, MD, S. Sacconi, MD, J. Pouget, MD, PhD, B. Eymard, MD, PhD, S. Chevret, MD, PhD and D. Annane, MD, PhD

Authors’ affiliations are listed at the end of the article.

Address correspondence and reprint requests to Dr. Djillali Annane, Service de Réanimation Médicale, Hôpital Raymond Poincaré (AP-HP), Université de Versailles Saint Quentin en Yvelines, PRES UniverSud Paris, Centre d’Investigation Clinique–Innovation Technologique (INSERM), 104 boulevard Raymond Poincaré, 92380 Garches, France djillali.annane{at}rpc.aphp.fr

Background: Myotonic dystrophy type 1 may be associated with low circulating dehydroepiandrosterone (DHEA) levels. This study was aimed at investigating the efficacy and safety of DHEA in myotonic dystrophy type 1 patients.

Methods: This was a prospective, multicenter, randomized, double-blind, placebo-controlled trial conducted from February 2005 to January 2006 at 10 university-affiliated neuromuscular disease centers in France. Seventy-five ambulatory adults with myotonic dystrophy type 1 received an oral replacement dose (100 mg/d) or a pharmacologic dose (400 mg/d) of DHEA, or placebo. The primary endpoint was the relative change in the manual muscle testing (MMT) score from baseline to week 12. Secondary outcome measures included changes from baseline to week 12 in quantitative muscle testing and timed functional testing, respiratory and cardiac function, and quality of life. This study was registered with ClinicalTrials.gov identifier NCT00167609.

Results: The median (1st, 3rd quartile) relative changes in MMT score from baseline to week 12 after randomization were 3.1 (–0.9, 6.7), 1.9 (–2.7, 3.5), and 2.2 (0, 7.9), in the DHEA 100 mg, DHEA 400 mg, and placebo groups, respectively. There were no differences between placebo and combined DHEA groups (p = 0.34), placebo and DHEA 100 mg (p = 0.86), or placebo and DHEA 400 mg (p = 0.15). There were also no evidence for a difference between groups for the changes from baseline to week 12 in any secondary outcome.

Conclusions: There is no evidence that a 12-week treatment with replacement or pharmacologic doses of dehydroepiandrosterone improves muscle strength in ambulatory myotonic dystrophy type 1 patients.

Abbreviations: BMI = body mass index; DHEA = dehydroepiandrosterone; DHEA-S = dehydroepiandrosterone sulfate ester; IQR = interquartile range; MIRS = muscular impairment rating scale; MMT = manual muscle testing; PSA = prostate-specific antigen; SF36 = Short Form 36.

Supplemental data at www.neurology.org

Supported by a grant from the Association Française de lutte contre les Myopathies. Sponsored by Assistance Publique Hôpitaux de Paris.

Disclosure: The authors report no disclosures.

Received December 10, 2007. Accepted in final form April 29, 2008.






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