Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: A randomised placebo-controlled trial

The Lancet Neurology

Volumn 10, Issue 2, 2011, Pages 140-147

  Fernández Rhodes, Lindsay E ^a   Kokkinis, Angela D ^a   White, Michelle J ^a   Watts, Charlotte A ^a   Auh, Sungyoung ^a   Jeffries, Neal O ^b   Shrader, Joseph A ^c   Lehky, Tanya J ^a   Li, Li ^c   Ryder, Jennifer E ^c   Levy, Ellen W ^c   Solomon, Beth I ^c   Harris Love, Michael O ^c   La Pean, Alison ^a   Schindler, Alice B ^a   Chen, CheunJu ^a   Di Prospero, Nicholas A ^a   Fischbeck, Kenneth H ^a  

^a NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^b NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CREATINE KINASE; DUTASTERIDE; PLACEBO;

ADULT; AGED; ARTICLE; CLINICAL ARTICLE; DAILY LIFE ACTIVITY; DIARRHEA; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; DYSPEPSIA; DYSPNEA; FALLING; FATIGUE; FRACTURE; GASTROINTESTINAL SYMPTOM; HEADACHE; HUMAN; INFECTION; KENNEDY DISEASE; MALE; MOTOR NERVE CONDUCTION; MUSCLE CRAMP; MUSCLE STRENGTH; MUSCLE WEAKNESS; MYALGIA; PAIN; PARESTHESIA; PENIS ERECTION; PRIORITY JOURNAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; SIDE EFFECT; UPPER RESPIRATORY TRACT INFECTION;

ACCIDENTAL FALLS; ADULT; AGED; AZASTEROIDS; BULBO-SPINAL ATROPHY, X-LINKED; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; FOLLOW-UP STUDIES; FRACTURES, BONE; HUMANS; MALE; MIDDLE AGED; TREATMENT OUTCOME;

EID: 78751590932     PISSN: 14744422     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1474-4422(10)70321-5     Document Type: Article

References (30)

1. Chahin N, Klein C, Mandrekar J, Sorenson E Natural history of spinal-bulbar muscular atrophy. Neurology 2008, 70:1967-1971.
2. Dejager S, Bry-Gauillard H, Bruckert E, et al. A comprehensive endocrine description of Kennedy's disease revealing androgen insensitivity linked to CAG repeat length. J Clin Endocrinol Metab 2002, 87:3893-3901.
3. Adachi H, Waza M, Katsuno M, et al. Pathogenesis and molecular targeted therapy of spinal and bulbar muscular atrophy. Neuropathol Appl Neurobiol 2007, 33:135-151.
4. Katsuno M, Adachi H, Kume A, et al. Testosterone reduction prevents phenotypic expression in a transgenic mouse model of spinal and bulbar muscular atrophy. Neuron 2002, 35:843-854.
5. Banno H, Katsuno M, Suzuki K, et al. Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy. Ann Neurol 2009, 65:140-150.
6. Katsuno M, Banno H, Suzuki K, et al. Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol 2010, 9:875-884. for the Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) study group.
7. Tindall DJ, Rittmaster RS The rationale for inhibiting 5α-reductase isoenzymes in the prevention and treatment of prostate cancer. J Urol 2008, 179:1235-1242.
8. Rhodes LE, Freeman BK, Auh S, et al. Clinical features of spinal and bulbar muscular atrophy. Brain 2009, 132:3242-3251.
9. Hislop HJ, Montgomery J, Connelly B, Daniels L Daniels and Worthingham's muscle testing: techniques of manual examination 1995, W B Saunders, Philadelphia, USA. 6th edn.
10. Harris-Love M, Joe G, Abbett K, Koziol D Performance-based assessment of functional limitation and muscle endurance: reliability of the adult myositis assessment tool. J Neurol Phys Ther 2004, 28:179-180.
11. Takeuchi Y, Katsuno M, Banno H, et al. Walking capacity evaluated by the 6-minute walk test in spinal and bulbar muscular atrophy. Muscle Nerve 2008, 38:964-971.
12. Light KE, Behrman AL, Thigpen M, Triggs WJ The 2-minute walk test: a tool for evaluating walking endurance in clients with Parkinson's disease. Neurol Rep 1997, 21:136-139.
13. Lynch DR, Farmer JM, Tsou AY, et al. Measuring Friedreich ataxia: complementary features of examination and performance measures. Neurology 2006, 66:1711-1716.
14. Rosen RC, Cappelleri JC, Gendrano N The International Index of Erectile Function (IIEF): a state-of-the-science review. Int J Impot Res 2002, 14:226-244.
15. Liveson JA, Ma DM Laboratory reference for clinical neurophysiology 1992, Oxford University Press, New York, USA.
16. Shefner JM, Cudkowicz ME, Zhang H, Schoenfeld D, Jillapalli D The use of statistical MUNE in a multicenter clinical trial. Muscle Nerve 2004, 30:463-469.
17. Lehky TJ, Chen CJ, di Prospero NA, et al. Standard and modified statistical MUNE evaluations in spinal-bulbar muscular atrophy. Muscle Nerve 2009, 40:809-814.
18. Morris JF, Koski A, Johnson LC Spirometric standards for healthy nonsmoking adults. Am Rev Respir Dis 1971, 103:57-67.
19. Fleiss J The design and analysis of clinical experiments 1999, John Wiley and Sons, Chichester, UK.
20. National Center for Health Statistics. Hyattsville, MD, USA; 2005-2006 National Health and Nutrition Examination Survey (NHANES), (accessed Dec 20, 2010). http://www.cdc.gov/nchs/nhanes/nhanes2005-2006/nhanes05_06.htm.
21. Takeyama K-I, Ito S, Yamamoto A, et al. Androgen-dependent neurodegeneration by polyglutamine-expanded human androgen receptor in Drosophila. Neuron 2002, 35:855-864.
22. Chevalier-Larsen ES, O'Brien CJ, Wang H, et al. Castration restores function and neurofilament alterations of aged symptomatic males in a transgenic mouse model of spinal and bulbar muscular atrophy. J Neurosci 2004, 24:4778-4786.
23. Schmidt BJ, Greenberg CR, Allingham-Hawkins DJ, Spriggs EL Expression of X-linked bulbospinal muscular atrophy (Kennedy disease) in two homozygous women. Neurology 2002, 59:770-772.
24. Sar M, Stumpf WE Androgen concentration in motor neurons of cranial nerves and spinal cord. Science 1977, 197:77-79.
25. Thigpen AE, Silver RI, Guileyardo JM, et al. Tissue distribution and ontogeny of steroid 5 alpha-reductase isozyme expression. J Clin Invest 1993, 92:903-910.
26. Mayhew JE, Florence JM, Mayhew TP, et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve 2007, 35:36-42.
27. Barkin J, Roehrborn CG, Siami P, et al. Effect of dutasteride, tamsulosin and the combination on patient-reported quality of life and treatment satisfaction in men with moderate-to-severe benign prostatic hyperplasia: 2-year data from the CombAT trial. BJU Int 2009, 103:919-926.
28. Tokui K, Adachi H, Waza M, et al. 17-DMAG ameliorates polyglutamine-mediated motor neuron degeneration through well-preserved proteasome function in an SBMA model mouse. Hum Mol Genet 2009, 18:898-910.
29. Yang Z, Chang Y-J, Yu IC, et al. ASC-J9 ameliorates spinal and bulbar muscular atrophy phenotype via degradation of androgen receptor. Nat Med 2007, 13:348-353.
30. Palazzolo I, Stack C, Kong L, et al. Overexpression of IGF-1 in muscle attenuates disease in a mouse model of spinal and bulbar muscular atrophy. Neuron 2009, 63:316-328.