Clinical and glucocorticosteroid therapeutic analyses of 96 patients with Duchenne muscular dystrophy

Abstract

Objective To analyze the clinical and laboratory phenotypes of Duchenne muscular dystrophy(DMD)in a retrospective study of 96 cases and to evaluate therapeutic efficacy of glucocorticosteroid.Methods The clinical phenotype,laboratory examinations resuhs and the records in the follow-up in 96 patients with DMD were collected.The level of serum creatine kinase(CK)and motor ability before and after glucocorticosteroid therapy were analyzed by statistical analysis.Their myocardium impairments and intelligence conditions were also assessed.Results(1)The level of serum CK (mmol/L)had three peaks at the age of ≤ 3 years old(16 547.9 ±770.9),5 years old(14 371.9 ± 696.7)and 8 years old(13 089.8 ± 877.6).The CK level significantly decreased after dexamethasone (5-10 mg,iv)treatment for 10-15 days,but increased again after prednisone acetate(0.50-0.75 mg · kg-1 · d-1,oral)administration for one month(F =6.758,P =0.003).(2)The motor ability improved in 51 DMD cases with long-term oral admission of prednisone,including 24 cases receiving repeated dexamethasone,iv.(3)The myocardial perfusion imaging in 37 DMD cases showed remarkable uneven “spotted like” radionuclide distribution in ventricle.(4)The intelligence quotients in 24 DMD were lower than normal population.Conclusions There are high CK hyperlipidemia and myocardial damage in the sub-clinical stage of DMD and myocardium impairment is positively correlated with age.Glucocorticosteroid therapy has an important effect on the protection of motor and cardiac functions,with recommendation of using in early stage of disease. Key words: Muscular dystrophy, ducherme; Glucocorticosteroids; Creatine kinase