Abstract-Text (inkl. Referenzen)

We determined VF burden in VAM-treated boys vs. daily deflazacort (DEFL), daily prednisone (PREDDAILY) and intermittent prednisone (PREDINT); 39 boys 4 to <7 years-old from VBP15-LTE study were compared to 68 boys 4 to <8 from FOR-DMD. In VBP15-LTE, boys initiated VAM 0.25, 0.75, 2.0, or 6.0 mg/kg/d for six months, followed by permitted dose escalations/de-escalations for 2 years. In FOR-DMD, boys were randomized to DEFL (0.9mg/kg/d), PREDDAILY (0.75mg/kg/d) or PREDINT (0.75mg/kg/d 10 days on/off). VF were read centrally on x-rays (Genant method) after 30 (VAM) or 36 (classic GC) months. In the VAM group, VF parameters were adjusted (adj) for shorter duration by a factor of 1.2 (36/30 months), as appropriate. Mean age on classic GC was 8.8±1.1 and on VAM 8.0±1.0 years. VF burdens (high to low): 1. VF prevalence: PREDDAILY 7/24 boys (29.2%), DEFL 4/21 (19.0%), VAM 4/39 (10.3%; 12.3% adj), PREDINT 0/23; 2. Highest fracture severity: DEFL moderate (n=1, 9.5%), PREDDAILY mild (N=7, 100%), VAM mild (n=4; 100%), and PREDINT N/A; 3. Avg number of VF/child: PREDDAILY 1.3, DEFL 1.3, VAM 1.0; 1.2 adj and PREDINT 0; 4. Maximum Spine Deformity Index: DEFL 6, PREDDAILY 4, VAM 1; 1.2 adj, PREDINT 0; and 5. Average SDI/child: DEFL 3, PREDDAILY 2.7, VAM 1.0; 1.2 adj, and PREDINT 0. VF burden was higher on PREDDAILY and DEFL than VAM but lowest on PREDINT. VAM may be a bone-sparing strategy to improve muscle strength.