Original Article

Trends With Corticosteroid Use

in Males With Duchenne Muscular
Dystrophy Born 1982-2001

Journal of Child Neurology

2015, Vol. 30(1) 21-26
The Author(s) 2014
Reprints and permission:
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DOI: 10.1177/0883073813517263
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Deborah J. Fox, MPH1, Anil Kumar, MS1, Nancy A. West, PhD2,

A. Gregory DiRienzo, PhD3, Katherine A. James, PhD4, Joyce Oleszek, MD5,
and the Muscular Dystrophy Surveillance, Tracking,
and Research Network (MD STARnet)

Abstract
This study examines trends in corticosteroid use for males with Duchenne muscular dystrophy by birth year, race/ethnicity, and
knowledge of Duchenne muscular dystrophy family history. Firstborn males (n 521) selected from a population-based surveillance system of Duchenne muscular dystrophy were analyzed using Kaplan Meier and regression methods. Comparing males born
1982 to 1986 with males born 1997 to 2001, steroid use increased from 54% to 72% and mean age at steroid initiation decreased
from 8.2 to 7.1 years. Hispanics and non-Hispanic Black males used steroids less frequently and delayed initiation compared to
white males. Compared to males without a Duchenne muscular dystrophy family history, males with known family history were
half as likely to use steroids. Duration of steroid use increased over time and age at initiation decreased. Racial/ethnic disparities
exist for steroid use and should be addressed to improve outcome and quality of life for boys with Duchenne muscular dystrophy.
Keywords
Duchenne muscular dystrophy, steroids, health disparities, age of treatment initiation, length of use
Received October 22, 2013. Received revised November 25, 2013. Accepted for publication November 25, 2013.

Duchenne muscular dystrophy is an X-linked recessive disorder resulting from a gene defect causing a deficiency in dystrophin, a critical cytoskeletal protein necessary for muscle
stabilization and repair.1 Duchenne muscular dystrophy is
characterized by a progressive deterioration in skeletal muscle
function whereby boys lose the ability to walk independently
between ages 7 and 12 years.2,3 Without pharmaceutical or
technological intervention, most boys do not survive more than
3 decades. Duchenne muscular dystrophy results from the total
absence of dystrophin protein whereas a milder form of the disorder, Becker muscular dystrophy, is characterized by the presence of less effective protein variations or a smaller quantity of
dystrophin.3 In this milder phenotype, clinical signs of muscular weakness generally develop later than with Duchenne muscular dystrophy around age 12, and there is typically no
wheelchair dependency before the 16th birthday.
The benefit of corticosteroid use on motor power and muscular activities for boys with Duchenne muscular dystrophy
has been shown since the 1970s.4,5 Early on, treatment was
often discontinued when a boy became nonambulatory. Subsequent studies document preservation of cardiac and respiratory function and reductions in orthopedic complications with
continuing use.6-12 Despite possible side effects such as

weight gain and behavioral problems, an annual increase in

percentage of boys with Duchenne or Becker muscular
dystrophy using steroids has been reported, from 20% in
1991 to 44% in 2005.13 Most clinicians agree that corticosteroid treatment should be initiated either before or when a boy
plateaus on certain mobility measures (ability to walk, rise
from floor, and/or climb stairs), generally from ages 6 to
8.14,15 Notwithstanding, there continues to be variations in use
by clinics, particularly in regard to pattern and frequency of
use.13,16 Trends in age at start and length of use have not been

New York State Department of Health, Albany, NY, USA

Department of Epidemiology, University of Colorado School of Public Health,
Aurora, CO, USA
3
Department of Epidemiology and Biostatistics, State University of New York
at Albany, Rensselaer, NY, USA
4
Colorado Department of Public Health and Environment, Denver, CO, USA
5
Department of Physical Medicine and Rehabilitation, University of Colorado
School of Medicine, Childrens Hospital Colorado, Aurora, CO, USA
2

Corresponding Author:
Deborah J. Fox, MPH, New York State Department of Health, Empire State
Plaza, Corning Tower Room 1203, Albany, NY, 12237, USA.
Email: djf03@health.state.ny.us

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Journal of Child Neurology 30(1)

Figure 1. Inclusion criteria for data set.

documented, nor have the effects of other factors on steroid

use beyond clinic recommendations.
For this investigation, we document trends with corticosteroid
therapy for young males with Duchenne muscular dystrophy
born from 1982 to 2001. Our aims were (1) to determine the
strength of the association between steroid use and 3 factors
(birth year, race/ethnicity, and knowledge of family history),
(2) to assess the age at steroid initiation and evaluate the associations between these 3 factors and on the age of initiation, and (3)
to determine if the length of steroid use has changed over time.

Methods
Study Population
The Muscular Dystrophy Surveillance, Tracking, and Research Network
(MD STARnet) is a population-based surveillance system funded by the
Centers for Disease Control and Prevention to identify retrospectively all
individuals diagnosed with Duchenne or Becker muscular dystrophy
born since January 1, 1982, who resided in one of the participating sites
(Arizona, Colorado, Georgia, Hawaii, Iowa, and western New York).
Multiple source case finding methods were used to identify potential
cases. Trained abstractors reviewed medical records annually and
detailed clinical and demographic data were collected for each individual. Key clinical and diagnostic data were used to assign a case status
(definite, probable, possible, female, asymptomatic, or not Duchenne
or Becker muscular dystrophy), which was then reviewed by a committee
of neuromuscular clinicians from all sites to validate the final case status.
A detailed description of the Muscular Dystrophy Surveillance,
Tracking, and Research Network surveillance methodology has been
published previously.17 Institutional review board approval was maintained at each study site for the project duration.
Of the 885 males diagnosed with Duchenne or Becker muscular
dystrophy who met case status criteria of definite or probable,
521 met the inclusion criteria. Firstborn males born prior to 2002 were
included to ensure accurate assessment and statistical independence. A
number of exclusions were made to create the final data set of 521
males (Figure 1). A 2-tiered strategy was used to include males most

likely to be the Duchenne muscular dystrophy phenotype. Males were

included if they ceased ambulating before age 16 or, if younger than age
16 at the most recent clinic visit and still walking, they had signs of
muscle weakness before age 7. Cases were also excluded when there
were gaps of 5 years or more between annual clinic visits, when there
was less than 7 years of follow up from date of birth and when there was
uncertainty with accuracy of the abstracted steroid data.
Abstracted data on steroid use included the date or age first started,
dates when the specific steroid or dosage was changed, and date or age
when use was discontinued, along with the reason for discontinuation.
Dates and ages when steroids were offered and whether the patient
began steroid therapy were also abstracted. If a patient was switched
from deflazacort to prednisone, or vice versa, but did not discontinue
use, it was considered to be continual use. Changes in medication
dosage were also treated as continuous use. A gap in steroid use
of less than 1 year was defined as continuous use. Patients with
no documentation about steroid use were considered nonusers.
Race/ethnicity was categorized as Hispanic, non-Hispanic white, nonHispanic black, or other (Asian, Hawaiian or Pacific Islander, Native
American, other or multiple) using information from medical records
as well as birth certificates, when available. A known family history was
assigned when the mother was aware of other affected family members
diagnosed with Duchenne or Becker muscular dystrophy at any time
before diagnosis of her firstborn affected male child.

Statistical Analysis
Descriptive analyses calculated means and proportions of key variables.
Chi-square tests and Fisher exact tests were used to investigate independence between categorical variables. Logistic regression was used to estimate the impact of birth year, knowledge of family history, and race/
ethnicity on whether steroids were used (yes/no), with adjustment for
state of residence. Crude and adjusted odds ratios were calculated, along
with 95% confidence intervals and P values. Multiple linear regression
methods were used to assess associations between predictors and the
mean age at start of steroids, among those who started steroids. To investigate trends in duration of steroid use, we classified cases into 4 birth
cohorts (1982-1986, 1987-1991, 1992-1996, and 1997-2001). Among
steroid users, Kaplan-Meier curves were used to assess length of steroid
use by birth cohort. If individuals had not discontinued steroid use, cases
were right-censored at the age of last neuromuscular clinic visit. The logrank test was used to assess statistical significance of birth cohort on the
length of steroid use. All statistical analyses were conducted with SAS
software version 9.2.

Results
Study Population
Mean age at last clinic visit was 16.1 years, with a range of 7.1
to 28.1 years (data not shown). As shown in Table 1, the largest
number of cases were from Georgia (n 144; 28%) and 63%
were non-Hispanic white (n 327). Steroid use by birth cohort
is nonlinear for the first 2 birth cohorts, affected by steroid clinical trials in the late 1980s and early 1990s. Twenty-three percent of cases had knowledge of Duchenne muscular dystrophy
in their family prior to diagnosis. When family history was
known, mean age of diagnosis was 3.0 years as opposed to
5.5 years for those with no family history (not shown in table).
Knowledge of family history was not associated with average

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Table 1. Demographic and Clinical Characteristics of Oldest Affected Male Child by Steroid Use (n 521).
Distribution by any steroid use
Characteristic

Distribution of categories

Birth cohort
1982-1986
1987-1991
1992-1996
1997-2001
Race/ethnicity
White, non-Hispanic
Hispanic
Black, non-Hispanic
Other
Unknown
MD STARnet Site
Arizona
Colorado
Georgia
Hawaii
Iowa
Western New York
Knowledge of family history
Yes
No
Undetermined
Age at initiation of corticosteroids (y), mean
With knowledge of family history
With no knowledge of family history

Yes (n 310)

No (n 211)

n
89
154
137
141

(%)
(17)
(30)
(26)
(27)

n (%)
48 (53.9)
78 (50.6)
82 (59.9)
102 (72.3)

n
41
76
55
39

(%)
(46.1)
(49.4)
(40.1)
(27.7)

327
112
42
30
10

(63)
(21)
(8)
(6)
(2)

221 (67.6)
52 (46.4)
17 (40.5)
14 (46.7)
6 (60.0)

106
60
25
16
4

(32.4)
(53.6)
(59.5)
(53.3)
(40.0)

124
108
144
13
69
63

(24)
(21)
(28)
(2)
(13)
(12)

64 (51.6)
74 (68.5)
77 (53.5)
1 (7.7)
50 (72.5)
44 (69.8)

60
34
67
12
19
19

(48.4)
(31.5)
(46.5)
(92.3)
(27.5)
(30.2)

120 (23)
382 (73)
19 (4)

55 (45.8)
246 (64.4)
9 (47.4)

65 (54.2)
136 (35.6)
10 (52.6)

7.3
7.5

P-valuea
.0009

<.0001

<.0001

.0003

.49

Abbreviation: MD STARnet, Muscular Dystrophy Surveillance, Tracking, and Research Network.

a
P values are from w2 tests for categorical variables and from t tests for continuous variables.

age of steroid initiation (known family history: 7.3 y at initiation; unknown family history: 7.5 y at initiation; P .49).
There were no statistically significant differences between the
521 males included and the 364 excluded cases on race/ethnicity (P .84) nor state of residence (P .40).

Attributes Associated With Corticosteroid Use

Of the 521 males in the analytic data set, 310 (60%) had a history
of steroid use. With each successive birth year, the odds of a boy
using steroids increased by 8% (P < .0002; Table 2). Males with
a family history of Duchenne muscular dystrophy are less than
half as likely to use steroids compared to those without a family
history (P .0006). Compared to non-Hispanic whites, Hispanics are 43% (P .0010) as likely to use steroids and nonHispanic blacks are 40% as likely (P .0094).

Table 2. Multivariate Logistic Analysis Showing Odds Ratios for Using

Steroids.

Birth year (continuous)b

Knowledge of Duchenne
family history
Race/ethnicity
White, non-Hispanic
Hispanic
Black, non-Hispanic
Other

Adjusted odds
ratioa

95% CI

P value

1.08
0.45

1.04-1.12
0.29-0.71

.0002
.0006

Reference
0.43
0.40
0.56

0.26-0.71
0.20-0.80
0.21-1.47

.0010
.0094
.2371

Abbreviation: MD STARnet, Muscular Dystrophy Surveillance, Tracking, and

Research Network.
a
Model also adjusted for Muscular Dystrophy Surveillance, Tracking, and
Research Network site.
b
Crude odds ratio 1.07 (1.03-1.11), P < .0001.

Characteristics Associated With Age at Steroid Initiation

The overall mean age at steroid initiation was 7.5 years, with a
downward trend from 8.2 years for those born in 1982-1986 to
7.1 years for those born in 1997-2001. Table 3 shows that age at
steroid initiation became significantly lower over 2 decades
(P .0003) such that there was a 1.1-month decrease in age
of initiation for each year increase in birth year, adjusting for
race/ethnicity, Muscular Dystrophy Surveillance, Tracking,

and Research Network site, and knowledge of family history.

There was a significant delay of 14.2 months in age of steroid
initiation for non-Hispanic black males compared to nonHispanic white males (P .035). The delays in mean age at
steroid initiation for Hispanics of 7.2 months (P .11) and for
the Other racial group of 8.4 months (P .24) were not
statistically significant.

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Journal of Child Neurology 30(1)

Table 3. Multiple Linear Regression for Mean Steroid Start Age.

Characteristic

ba

SE

0.09
0.0252
Birth year (continuous)a
Knowledge of Duchenne family history
0.39
0.3307
Race/ethnicity
White, non-Hispanic
Reference

Hispanic
0.60
0.3695
Black, non-Hispanic
1.18
0.5545
Other
0.73
0.6214

P value
.0003
.2452

.1054
.0349
.2428

Abbreviation: SE, standard error.

a
Model also adjusted for Muscular Dystrophy Surveillance, Tracking, and
Research Network site.

Figure 3. Proportion of males using steroids that were offered

steroids (P .0084).*

Figure 2. Kaplan-Meier for length of steroid use for boys using

steroids, for birth cohorts 1982-1986, 1987-1991, 1992-1996, and
1997-2001.

Trends in Length of Steroid Use

Among steroid users, there was a significant increased duration
of steroid use for recent birth cohorts compared to earlier
cohorts (log-rank P .004; Figure 2). The median length of
steroid use for the earliest birth cohort (1982-1986) was
5.3 years and increased to 9.6 years for the 1992-1997 cohort.
The median length of steroid use for males born 1997-2001
could not be determined because more than 50% of the users
have continued to use steroids.

Attributes Associated With Being Offered Steroids

Stratification by race/ethnicity showed differences in the proportion of males who were offered steroids and eventually initiated them. Compared to non-Hispanic white males, Hispanic
and non-Hispanic Black males were significantly less likely to
choose steroid treatment even though it had been recommended
(P .0084; Figure 3).

Discussion
For males with Duchenne muscular dystrophy, corticosteroid
treatment is the standard of care. Our results document progress
to adopt these clinical recommendations, and that implementation

of treatment is influenced by race/ethnicity and knowledge of

family history. Some findings are not surprising, such as boys
with Duchenne muscular dystrophy are started on steroids at
increasingly younger ages and are staying on steroids for a longer
duration. These trends would be expected given increases in
knowledge about the benefits of steroid therapy and better monitoring of the drugs (fewer complications from adverse events).
Several findings, however, were not expected.
Disparities in health care delivery based on race/ethnicity
have frequently been documented.18,19 Although our findings
suggest a significantly lower rate in the use of steroids by Hispanic and non-Hispanic black males compared to non-Hispanic
white males, we also show evidence that a greater proportion of
Hispanic and black males chose not to initiate steroid treatment. This may reflect cultural differences in acceptance of the
disability and a reluctance to change the natural course of the
disease.20,21 Alternatively, it may indicate that interventions
are not being presented in a culturally responsive manner.22
One might expect that when families have older affected
males with Duchenne muscular dystrophy in their extended family, they may be more aware of potential treatments and be more
proactive seeking these treatments. We found the opposite;
families with a prior family history were less than half as
likely to choose steroid therapy. A recent study indicated that,
increasingly, families with known family histories are making
use of prenatal testing to prevent the birth of an affected
male.23 When a family with known family history does have
an affected firstborn male, it may reflect acceptance of Duchenne muscular dystrophy as a characteristic of their family.
What we cannot glean from the data is whether this acceptance may be culturally or ethnically based, and underlies
or contributes to the documented racial/ethnic differences in
steroid use. Alternatively, these families may have a negative
opinion about steroids because of side effects from steroid use
in their family members.
One of the most significant strengths of this study is the use
of the largest population-based cohort of Duchenne muscular
dystrophy patients in the United States. Most studies of Duchenne muscular dystrophy are derived from clinic-based populations,24-26 which introduces the possibility of selection bias and

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reducing the ability to generalize findings. Furthermore, the

Muscular Dystrophy Surveillance, Tracking, and Research
Network surveillance methodology collects data longitudinally
and permits evaluation of trends over time, as was done here.
A study limitation is the reliance on medical records abstraction to obtain key clinical and treatment information. In particular, we had to use clinical findings and observations about males
with Duchenne or Becker muscular dystrophy to define our
study population of boys with the Duchenne muscular dystrophy
phenotype. While our patient selection criteria are fairly robust,
we may have excluded some males with Duchenne muscular
dystrophy because they responded successfully to steroid therapy and were still ambulating at age 16; or likewise included
Becker muscular dystrophy patients with noted early muscle
weakness and less than 16 years of clinical history. The phenotype severity would certainly influence treatment choices, particularly with a treatment that can have significant adverse events
in some individuals. A second but unavoidable limitation is our
inability to evaluate trends in steroid use for boys born after
2001.
These results provide baseline information on the likelihood
of using steroids, age at steroid start, and length of steroid use.
Additional data collection will allow continued evaluation of
current and future adherence to the practice parameters published in 2004-2005 as well as the 2010 clinical care guidelines
for individuals with Duchenne muscular dystrophy. It will be
important to repeat these analyses in several years to monitor
trends in steroid use for boys born after 2001, and adherence
to these care considerations. Clearly, additional work is
required to reduce racial/ethnic disparities in access to and
acceptance of steroid therapy. It may be valuable to survey parents of Duchenne muscular dystrophy boys who have older
affected males in their extended family to understand reasons
for differences in use of steroids compared to families without
a family history of Duchenne muscular dystrophy.
Author Contributions
DJF conceived and developed the study, with statistical advice from
AGD. DJF reviewed the literature, provided citations, and wrote the
first draft. DJF/AK acquired the data and DJF/AK/KF performed case
review. DJF/AK/NAW/AGD/JO discussed analytic results. AK produced graphs and tables. NAW validated statistical analyses by AK.
AGD/NAW were statistical consultants. All authors reviewed and critiqued the drafts.

Declaration of Conflicting Interests

The authors declared no potential conflicts of interest with respect to
the research, authorship, and/or publication of this article.

Funding
The authors disclosed receipt of the following financial support for the
research, authorship and/or publication of this article: This research
was supported by Cooperative Agreement 5 U01 DD000191 from the
Centers for Disease Control and Prevention (CDC). Its contents are
solely the responsibility of the authors and do not necessarily reflect
the official views of CDC.

Ethical Approval
Institutional Review Board approval was maintained at each study site
for the project duration.

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