Date
|
Trial or Study |
Benefits |
Side
Effects |
1970s,
1980s
|
4 trials used prednisone in high doses ranging
from 1.5 mg/kg/day to 5 mg/kg on alternate days |
prolongation of walking by about 2 years in
one study; one study showed no benefit |
Majority in group that walked longer had
Excessive weight gain; hyperactivity, cataracts,
high blood pressure, bone fractures also noted
|
| 1989 |
prednisone or prednisolone at 0.3, 0.75 and
1.5 mg/kg/day for 6 months |
improved ability to rise from floor, time
to walk 9 meters (30 feet) and respiratory function;
0.75 mg group showed more benefit than 0.3 mg
group, but 1.5 mg group did not show additional
benefit |
Excessive weight gain, fat redistribution
and behavioral changes in treated boys |
| 1991 |
prednisone or prednisolone at 0.3, 0.75 and
1.5 mg/kg/day for 6 months |
improved ability to rise from floor, time
to walk 9 meters (30 feet) and respiratory function;
0.75 mg group showed more benefit than 0.3 mg
group, but 1.5 mg group did not show additional
benefit |
weight gain, fat redistribution and behavioral
changes in treated boys |
| 1991 |
daily prednisone compared with alternate-day
prednisone at equivalent weekly doses |
strength improved in alternate-day group but
was not sustained as with daily prednisone |
side effects not significantly different with
daily vs. alternate-day prednisone |
| 1991 |
daily prednisone at starting dose of 0.75
mg/kg/day |
slower progression of disease than in untreated
group; function improved in some treated boys |
side effects required decrease in dose in
some; final dose ranged from 0.15 mg/kg/day
to 0.75 mg/kg/day |
| 1993 |
prednisolone 0.75 mg/kg/day for 10 days alternating
with 20 days off drug |
positive influence on strength at 6 months,
followed by slow decline |
weight gain and other side effects much less
than expected with daily treatment |
| 1993 |
immunosuppressant azathioprine didn’t
show benefit when added to 0.3 mg/kg/day of
prednisone or when used alone |
|
weight gain, growth slowing, other side effects
with prednisone |
| 1994 |
deflazacort at 2 mg/kg every other day for
2 years |
treated boys did better in walking, rising
from chair and from floor; treated boys lost
walking ability at average of 11.8 years, compared
to 10.5 years in untreated group |
moderate weight gain, slight behavioral changes |
| 2000 |
prednisone at 0.75 mg/kg/day or deflazacort
at 0.9 mg/kg/day for a year |
at 1 year, two treated groups had same degree
of better motor function than untreated |
at 9 months, average weight increase in deflazacort
group was 5 percent; in prednisone group, was
18 percent |
| 2001 |
prednisone or prednisolone at 0.3, 0.75 and
1.5 mg/kg/day for 6 months (pooled results of
1989, 1991, 2001 studies) |
improved ability to rise from floor, time
to walk 9 meters (30 feet); 0.75 mg group showed
more benefit than 0.3 mg group, but 1.5 mg group
did not show additional benefit |
weight gain, fat redistribution and behavioral
changes in boys treated with 0.75 mg/kg/day
in two studies; 0.3 mg/kg/day group did not
differ in fat distribution from untreated in
one study |
| 2001 |
prednisone at 0.75 mg/kg/day for average of
10 years; dose decreased over time |
average age of loss of walking was 14.5 years,
compared to expected loss of walking at 8.8
to 10.5 years |
side effects required decrease in dose; average
tolerated dose was 0.35
mg/kg/day |
| 2001 |
deflazacort at 0.9 mg/kg/day for average of
3.8 years |
7 out of 30 treated boys stopped walking
at average of 12.3 years; all 24 untreated
stopped walking at average of 9.8 years
respiratory function in treated group significantly
greater at age 15 than in untreated
none of treated boys required scoliosis surgery,
compared to 13 in untreated group
|
10 out of 30 treated boys developed cataracts
significant growth slowing in treated group
compared to untreated |
| 2002 |
prednisolone at 0.75 mg/kg/day for 10 days
alternating with 10 days off drug for 4 to 5+
years |
improvement in ability to rise from floor,
hop, jump, and run without waddle compared to
untreated |
did not see abnormal weight gain, bone loss
or other signs of toxicity |
| 2002 |
prednisone at 5 mg/kg/day Fridays and Saturdays
only |
strength improved over 6 to 12 months |
irritability requiring dose reduction or stopping
drug in at least 6 of 20 boys |
| 2003 |
deflazacort for average of 5.1 years |
cardiomyopathy in 1 out of 21 treated boys,
compared to 7 out of 12 untreated boys
treated boys had significantly better respiratory
function
10 of 21 treated boys stopped walking, compared
with all 12 untreated boys
|
dose had to be decreased to average of
.59 mg/kg/day at age 18 because of side effects
cataracts,
below-average height in treated group
|
| 2003 |
prednisone, prednisolone or deflazacort at
1 mg/kg/day for average of 4.5 years, with calcium
and sometimes vitamin D |
|
10 out of 25 treated boys developed spinal
(vertebral) fracture |
| 2004 |
European Neuromuscular Centre (ENMC) publishes
standards for use of corticosteroids in DMD |
evidence for use of daily steroids in DMD
established; monitoring, minimizing, treating
side effects necessary; alternatives to daily
steroids should be tested against daily steroids
in large trials; cardiac and bone protection
studies should be planned; quality of life should
be assessed |
|
| 2005 |
American Academy of Neurology publishes guidelines
for corticosteroids in DMD |
prednisone at 0.75 mg/kg/day is optimal, but
dosages as low as 0.3 mg/kg/day still have benefit;
deflazacort at 0.9 mg/kg/day can be substituted
for prednisone;
benefits and side effects need monitoring;
if excessive weight gain occurs, dosage should
be decreased to 0.5 mg/kg/day and further decreased
to 0.3 mg/kg/day, if necessary |
|
| 2006 |
deflazacort starting at 0.9 mg/kg/day with
1,000 units of vitamin D and 750 mg of calcium
for average of 5.5 years |
ability to climb four stairs, rise from
floor and walk 10 meters (33 feet) preserved
significantly in treated boys compared to
untreated
respiratory function significantly greater
at age 18 in treated compared to untreated
boys; by 18, no treated boy required ventilation,
compared to 46 percent untreated
4 of 40 treated boys had cardiomyopathy,
compared to
20 of 34 untreated
by age 18, 4 of 40 treated boys had significant
spinal curvature, compared to 30 of 34 untreated
|
treated boys were significantly shorter
than untreated
some treated boys gained excessive weight
22 of 40 treated boys developed cataracts,
compared to none of the untreated
long bone fractures same (25 percent) in
treated and untreated groups
vertebral fractures in 3 treated boys and
none of untreated
2 deaths out of 40 treated boys, compared
with 12 deaths out of 34 untreated
|
