Abstract • There is no effective treatment at present for Duchenne's muscular dystorphy (DMD). Recently, clinical improvement and increased muscle strength have been reported after 100 mg of allopurinol was administered daily for 41/2 months to 16 boys with DMD. Our objective was to validate the effectiveness of 100 mg of allopurinol given daily to 21 boys with DMD in a double-blind crossover study. Its effectiveness was determined by an extensive battery of muscle function, cardiac, and laboratory tests. The ten-month study was divided into four periods: (1) one month of baseline observation; (2) three months of therapy to allopurinol and placebo control groups; (3) three months of therapy to crossed over groups; and (4) three months of therapy to all patients. No statistically significant changes between the allopurinol and control periods occurred in the muscle function tests. The earlier report of clinical improvement during allopurinol treatment was not confirmed. References 1. Brooke MH: A Clinician's View of Neuromuscular Diseases . Baltimore, Williams & Wilkins Co, 1977, pp 95-107. 2. Vignos PJ: Respiratory function and pulmonary infection in Duchenne muscular dystrophy . Isr J Med Sci 1977;13:207-214. 3. Heymsfield SB, McNish T: Sequence of cardiac changes in Duchenne muscular dystrophy . Am Heart J 1978;95:283-293.Crossref 4. Thomson WHS, Smith J: X-linked recessive (Duchenne) muscular dystrophy and purine metabolism: Effects of oral allopurinol and adenylate . Metabolism 1978;27:151-163.Crossref 5. Thomson WHS, Sweetin JC, Hilditch TE: Studies on the carrier state in X-linked recessive (Duchenne) muscular dystrophy . Clin Chim Acta 1975;63:383-394.Crossref 6. Murray AW, Elliot DC, Atkinson MR: Nucleotide biosynthesis from preformed purines in mammalian cells: Regulatory mechanisms and biological significance . Prog Nucleic Acid Res Mol Biol 1970;10:87-119. 7. Mendell JR, Wiechers DO: Lack of benefit of allopurinol in Duchenne dystrophy . Muscle Nerve 1979;2:53-56.Crossref 8. Stern LM, Fewings JD, Bretag AH, et al: The progression of Duchenne muscular dystrophy: Clinical trial of allopurinol therapy . Neurology 1981;31:422-426.Crossref 9. Bretag AH, Stern LM, Ballard FJ, et al: Allopurinol ineffective in Duchenne muscular dystrophy . Lancet 1981;1:276.Crossref 10. Doriguzzi C, Bertolotto A, Ganzit GP, et al: Ineffectiveness of allopurinol in Duchenne muscular dystrophy . Muscle Nerve 1981;4:176-178.Crossref 11. Byrer AJ: Interpreting the Southern California Sensory Interpretation Tests . Los Angeles, Western Psychological Services, 1976. 12. Moore JE: The Moore Eye-Hand Coordination and Color-Matching Tests: Manual of Directions . Los Angeles, California Testing Bureau, 1949. 13. Winer BJ: Statistical Principles in Experimental Design , ed 2. New York, McGraw-Hill Book Co, 1971, pp 261-308. 14. Snedecor GW, Cochran WG: Statistical Methods , ed 6. Ames, Iowa, Iowa State University Press, 1967, pp 120-134.
Archives of Neurology – American Medical Association
Published: May 1, 1983