Abstract-Text (inkl. Referenzen)

The objective of this analysis is to compare the effect of PDN, DFZ and VAM (all shown to be efficacious in the treatment of DMD) on muscle function after 1 year of treatment in DMD patients aged 4 to <7 years at the time of treatment initiation. Data were analyzed from two randomized, double-blind studies (FOR-DMD and VISION-DMD). Two groups from the FOR-DMD study (daily PDN 0.75 m/kg/day and daily DFZ 0.9 mg/kg/day) were compared to patients treated with VAM 6.0 mg/kg/day in the VISION-DMD study. Based on the propensity scores, 39 PDN and 47 DFZ treated patients were extracted from the FOR-DMD study and 27 VAM treated patients from the VISION-DMD study. All patients were glucocorticoid naïve at baseline with an average (SD) age of 5.4 (0.6), 5.4 (0.8) and 5.5 (0.9) years for PDN, DFZ and VAM groups, respectively. After 48 to 52 weeks of treatment, the least square mean (LSM) TTSTAND velocity increased in all treatment groups by 0.04 rises/sec, with no significant LSM difference between PDN vs VAM (0.002; 95% CI -0.033 to 0.037; p=0.929) or DFZ vs VAM (0.002; 95% CI -0.029 to 0.032; p=0.923). In other efficacy measures comparable increases were seen in all three treatment groups, with no statistically significant differences between PDN vs VAM or DFZ vs VAM. These data show that daily regimens of prednisone, deflazacort and vamorolone have comparable effect in improving motor function in patients with DMD. On behalf of the VISION-DMD, FOR-DMD and CINRG investigators.