A COMPARISON OF DAILY AND ALTERNATE-DAY PREDNISONE THERAPY IN THE TREATMENT OF DUCHENNE MUSCULAR-DYSTROPHY

被引:96
作者
FENICHEL, GM
MENDELL, JR
MOXLEY, RT
GRIGGS, RC
BROOKE, MH
MILLER, JP
PESTRONK, A
ROBISON, J
KING, W
SIGNORE, L
PANDYA, S
FLORENCE, J
SCHIERBECKER, J
WILSON, B
机构
[1] WALTER MCKENZIE CTR HLTH SCI, DEPT NEUROL, EDMONTON, ALBERTA, CANADA
[2] OHIO STATE UNIV, COLL MED, DEPT NEUROL, COLUMBUS, OH 43210 USA
[3] WASHINGTON UNIV, SCH MED, DIV BIOSTAT, ST LOUIS, MO 63110 USA
[4] UNIV ROCHESTER, DEPT NEUROL, ROCHESTER, NY 14627 USA
[5] WASHINGTON UNIV, SCH MED, DEPT NEUROL, ST LOUIS, MO 63110 USA
关键词
D O I
10.1001/archneur.1991.00530180027012
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
We previously reported the results of a randomized, double-blind 6-month trial of prednisone therapy in which 102 boys aged 5 to 15 years with Duchenne muscular dystrophy received daily doses of 1.5 and 0.75 mg/kg per day and were compared with those receiving placebo. The strength and function in both prednisone-treated groups improved equally and were significantly better than in the placebo group. To compare alternate-day and daily dosing of prednisone with respect to benefits and adverse side effects, the placebo group was started on alternate-day prednisone therapy, and the treatment group regimens were changed to equivalent doses of alternate-day prednisone without breaking the double-blind nature. At the end of 6 months, the group that was changed from daily to alternate-day therapy had declined in strength back to levels observed 12 months previously, at the start of daily therapy. The group in which alternate-day therapy was started showed a significant improvement in strength at 3 months, similar in magnitude to the response of boys treated with daily therapy. However, their strength declined significantly in the subsequent 3 months compared with boys who received daily therapy. The frequency of side effects was not significantly different for alternate-day therapy compared with daily therapy. We conclude that alternate-day prednisone therapy effectively increases strength but does not sustain the improvement to the same extent as daily therapy or mitigate side effects.
引用
收藏
页码:575 / 579
页数:5
相关论文
共 17 条
[11]   DYSTROPHIN - THE PROTEIN PRODUCT OF THE DUCHENNE MUSCULAR-DYSTROPHY LOCUS [J].
HOFFMAN, EP ;
BROWN, RH ;
KUNKEL, LM .
CELL, 1987, 51 (06) :919-928
[12]  
KISSEL J T, 1990, Neurology, V40, P412
[13]   COMPLETE CLONING OF THE DUCHENNE MUSCULAR-DYSTROPHY (DMD) CDNA AND PRELIMINARY GENOMIC ORGANIZATION OF THE DMD GENE IN NORMAL AND AFFECTED INDIVIDUALS [J].
KOENIG, M ;
HOFFMAN, EP ;
BERTELSON, CJ ;
MONACO, AP ;
FEENER, C ;
KUNKEL, LM .
CELL, 1987, 50 (03) :509-517
[14]  
Marascuilo L. A., 1977, NONPARAMETRIC DISTRI, P167
[15]   RANDOMIZED, DOUBLE-BLIND 6-MONTH TRIAL OF PREDNISONE IN DUCHENNES MUSCULAR-DYSTROPHY [J].
MENDELL, JR ;
MOXLEY, RT ;
GRIGGS, RC ;
BROOKE, MH ;
FENICHEL, GM ;
MILLER, JP ;
KING, W ;
SIGNORE, L ;
PANDYA, S ;
FLORENCE, J ;
SCHIERBECKER, J ;
ROBISON, J ;
KAISER, K ;
MANDEL, S ;
ARFKEN, C ;
GILDER, B .
NEW ENGLAND JOURNAL OF MEDICINE, 1989, 320 (24) :1592-1597
[16]   CLINICAL INVESTIGATION OF DUCHENNE MUSCULAR-DYSTROPHY - A METHODOLOGY FOR THERAPEUTIC TRIALS BASED ON NATURAL-HISTORY CONTROLS [J].
MENDELL, JR ;
PROVINCE, MA ;
MOXLEY, RT ;
GRIGGS, RC ;
BROOKE, MH ;
FENICHEL, GM ;
MILLER, JP ;
KAISER, KK ;
KING, W ;
ROBISON, J ;
SIGNORE, L ;
PANDYA, S ;
FLORENCE, JM ;
SEYFRIED, W ;
MANDEL, S .
ARCHIVES OF NEUROLOGY, 1987, 44 (08) :808-811
[17]   CLINICAL CONCEPTS OF DUCHENNE MUSCULAR-DYSTROPHY - THE IMPACT OF MOLECULAR-GENETICS [J].
ROWLAND, LP .
BRAIN, 1988, 111 :479-495