Résumé :
|
Study participants and setting: Patients without medication were evaluated at 3 months for 13 patients (mean age 11y 7 mo; SD 1y 10mo) and at one year for 41 patients (mean age 14y 1 mo; SD 5y 5mo) in a referral center. Twelve patients treated with steroids (mean age 10 y 2 mo; SD 2y 2 mo) were evaluated after one year of treatment and compared with 12 age-matched DMD controls. Results: In a 3-month period, subscore D1 showed a significant average decrease of 4.7% (p<0,01). In a 1-year period, all scores showed significant decreases: D1 4.9% (p<0,01), D2 7.7% (p<0,01), D3 4.3% (p<0,03) and Total Score 5.8% (p<0,01). For the 11 walking patients at the beginning of the study, the average value of D1 annual decrease is 26.1%. For the non-ambulant patients, the annual average decrease was 11.8% for D2 and 6.3% for Total Score. A sensitive threshold value for the loss of the ability to walk and a predictive value a year before loss of ambulation could be estimated. In the group of patients treated with steroids, compared to controls, it was evidenced a stabilization of the total score (-0.59 vs. -5.87, p<0,02 ) and dimension D2 (0.98 vs. -8.50, p<0,01). Conclusions: Our preliminary results are promising for the use of the MFM in clinical trials to evidence either deterioration of disease or lack of deterioration induced by therapeutics.
|