P.11.8 The impact of deflazacort on upper extremity function in young adults with Duchenne muscular dystrophy

Abstract

Corticosteroid treatment has significantly delayed the progression of Duchenne muscular dystrophy (DMD). Once wheelchair dependent, the adolescent relies on upper extremity (UE) function for activities of daily living including feeding, wheelchair mobility, transfers and recreation. The loss of independent feeding is a major milestone and an adjustment for both the boys and their parents. The Brooke Scale is a quick and effective tool for clinical practice that provides a classification of UE function. Grade 1 or 2 indicates shoulder strength to elevate their arms above their head. Grade 3 or 4 indicates they can flex at their elbow and feed independently. Grade 5 or 6 indicates they can not feed independently. We present a prospective cohort of 39 males, older than 15 yrs, with DMD, followed at Holland Bloorview Kids Rehabilitation Hospital in Toronto. All boys were treated with daily deflazacort since 7.7 ± 1.6 yrs. The mean dose of deflazacort was 35 ± 3 mg (range 24–42 mg). The mean age at last follow-up was 19.2 ± 3.2 yrs (range 15– 26 yrs). At 16 yrs, 65% were non-ambulatory, by 18 yrs 94% were non-ambulatory and by 22 yrs all patients were non-ambulatory. None had spinal surgery. The Brooke Scale was 1 ± 0 at 8 yrs, 1.0 ± 0.2 at 10 yrs, 1.2 ± 0.4 at 12 yrs, 1.6 ± 0.8 at 14 yrs, 1.9 ± 0.9 at 16 yrs, 2.9 ± 0.9 at 18 yrs, 2.8 ± 0.8 at 20 yrs and 3.0 ± 1.0 at 22 yrs of age. At 18 yrs, 96% (27/28) were able to eat independently and at 22 yrs 100% (9/9) were able to eat independently. In corticosteroid naive boys, Steffensen et al. (2002) describe that boys at 15 yrs had a median Brooke Scale of 5 (range 2–5) and by 20 yrs had a median Brook scale of 5 (range 5–6). Rahbek et al. (2005) describes that boys at 18 yrs could not feed themselves but could operate an electric wheelchair. Deflazacort treatment has significantly maintained UE strength and preserved independence for eating well into their third decade resulting in a significant impact on quality of life for boys and parents.