Abstract
A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P <0.01), in rising from a chair, Gower's maneuver, and walking (P <0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P <0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P <0.02), and grade and time of Gower's maneuver (P <0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 20.5 ± 11 months; for the placebo group it was 33.2 ± 9 months (placebo vs. deflazacort group, P <0.05). Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes.
| Original language | English |
|---|---|
| Pages (from-to) | 386-391 |
| Number of pages | 6 |
| Journal | Muscle and Nerve |
| Volume | 17 |
| Issue number | 4 |
| Publication status | Published - 1994 |
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Keywords
- Duchenne dystrophy
- steroid
- trial
ASJC Scopus subject areas
- Clinical Neurology
- Neuroscience(all)
Cite this
Deflazacort in Duchenne dystrophy : Study of long-term effect. / Angelini, C.; Pegoraro, E.; Turella, E.; Intino, M. T.; Pini, A.; Costa, C.
In: Muscle and Nerve, Vol. 17, No. 4, 1994, p. 386-391.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Deflazacort in Duchenne dystrophy
T2 - Study of long-term effect
AU - Angelini, C.
AU - Pegoraro, E.
AU - Turella, E.
AU - Intino, M. T.
AU - Pini, A.
AU - Costa, C.
PY - 1994
Y1 - 1994
N2 - A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P <0.01), in rising from a chair, Gower's maneuver, and walking (P <0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P <0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P <0.02), and grade and time of Gower's maneuver (P <0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 20.5 ± 11 months; for the placebo group it was 33.2 ± 9 months (placebo vs. deflazacort group, P <0.05). Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes.
AB - A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P <0.01), in rising from a chair, Gower's maneuver, and walking (P <0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P <0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P <0.02), and grade and time of Gower's maneuver (P <0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 20.5 ± 11 months; for the placebo group it was 33.2 ± 9 months (placebo vs. deflazacort group, P <0.05). Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes.
KW - Duchenne dystrophy
KW - steroid
KW - trial
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UR - http://www.scopus.com/inward/citedby.url?scp=0028280495&partnerID=8YFLogxK
M3 - Article
VL - 17
SP - 386
EP - 391
JO - Muscle and Nerve
JF - Muscle and Nerve
SN - 0148-639X
IS - 4
ER -