Clinical analysis of 155 patients with Duchenne muscular dystrophy

Abstract

Objective To investigate the clinical manifestations and laboratory examinations of Duchenne muscular dystrophy (DMD) patients and evaluate the principle of intermittent intravenous combined with oral glucocorticoid therapy.  Methods The clinical features, laboratory examinations andfollow-up data of 155 DMD patients were collected. These patients were given dexamethasone 5-10 mg/d by intravenous infusion for 10-15 d and oral prednisone acetate 0.50-0.75 mg/(kg·d) for one month. After treatment, the motor ability of lower limbs, the level of serum creatine kinase (CK) and 99mTc-MIBI gated myocardial perfusion imaging (GMPI) findings were compared with those before glucocorticoid therapy by statistical analysis.  Results 1) The motor ability was improved in 70 follow-up cases of DMD patients with long-term oral prednisone (squat and rise: Z = 207.000, P = 0.034; climbing stairs: Z = 237.000, P =0.008). 2) The level of serum CK of 155 first diagnosed patients reached the peak at 3 years old, and declined after the age of 8 (P < 0.05, for all). The serum CK of 70 follow-up cases was significantly decreased after 10-15 d dexamethasone intravenous infusion (P = 0.000), and increased again after one-month oral administration of prednisone acetate (P = 0.000), but was still lower than that before treatment (P = 0.008). 3) The 99mTc-MIBI GMPI of 77 patients showed different degrees of myocardial involvement and significantly uneven left ventricular radionuclide distribution, especially in apex cordis (55 cases), inferior wall (45 cases) and anterior wall (30 cases) of apex. Conclusions There exists increased level of serum CK in the sub⁃clinical stage of DMD. The level of serum CK declined year by year after the age of 8. Intermittent intravenous combined with oral glucocorticoid therapy has an important effect on protecting motor and cardiac functions, extending walking time and reducing serum CK level and muscle cell damage. Early glucocorticoid therapy is recommended.

DOI: 10.3969/j.issn.1672-6731.2015.05.008