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Table 2 Key study and patient characteristics, included studies

From: The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

Author, year

Sample characteristics

N

Geographic location

Mean age at baseline, y

Study design/data source

Study focus

Follow-up, y

Bach, 2011 + [40]

Non-ambulatory; Progressed to ventilation

134

US

19.0

Single center chart review

Survival among ventilated patients

Mean, 11.5

Bach, 2015 + [39]

Progressed to ventilation

133

US

18.6

Single center chart review

Costs and RU among ventilated patients

Mean, 8.7 Max (29)

Barber, 2013a [20]

Ambulatory DMD

462

US

7.4

MD STARnet

Age at cardiomyopathy

Mean, 4

Barnard, 2018a [36]

Ambulatory DMD

136

US

8.3

Multicenter chart review

qMR biomarkers in DMD

Up to 4

Bello, 2015 [28]

Ambulatory DMD

252

Internationalb

6.8

CINRG-DNHS

Age at LOA and AEs of CS

Mean, 3.8

Bello, 2015 (2)a [29]

Ambulatory DMD

225

Internationalb

NR

CINRG-DNHS

LTBP4 and SPP1 polymorphisms on age at LOA

Mean, 4

Bello, 2016 [27]

Ambulatory DMD

157

Internationalb

NR

CINRG-DNHS

Genotype x age at LOA

Mean, 4

Connolly, 2016a [48]

Non-ambulatory DMD

81

US

16.8

MDA-DMD research network

Responsiveness of measures for non-ambulatory DMD

Up to 2

Deshpande, 2018a [32]

Ambulatory and non-ambulatory DMD

437

US and Canada

Unclear; study entry in 2005

Administrative

Characterize clinical course; incl. in those with heart failure

Unclear; 10 per patient

Gambetta, 2018a [33]

Ambulatory and non-ambulatory DMD

324

US and Canada

6.0

Multicenter chart review

Impact of genotype on outcomes

Unclear; 10 per patient

Henricson, 2017a [41]

Unclear

233

Internationalb

12.6

CINRG-DNHS

Impact of CS use on pulmonary function decline

Up to 9

Kim, 2015 [21]

Ambulatory DMD

220

US

Unclear; CS initiation at age 7

MD STARnet

Impact of CS on LOA

Unclear; 29

Kim, 2017 [31]

Ambulatory DMD

307

US

2.6

MD STARnet

Impact of CS on LOA

Median, 11–15

King, 2007 [22]

Ambulatory and non-ambulatory DMD

75

US

15.7

Single center chart review

Impact of CS on orthopedic outcomes

Up to 3

Labove, 2018 [23]

Cannot climb stairs

70

Canada

Unclear; age-initiated steroids 7, dx 4.2

Single center chart review

Height and age at LOA

Unclear; ≥ 7.7 per patient

Lopez-Hernandez, 2014 [24]

Unclear

432

Mexico

6.0

Multicenter chart review

Diagnosis and management of DMD in Mexico

Unclear; 20 per patient

Mayer, 2015a [42]

Ambulatory and non-ambulatory

60

US

10.3

Single center chart review

Pulmonary function in DMD

Up to 5

Mcdonald, 2018 [30]

Ambulatory and non-ambulatory DMD

330

Internationalb

10.7

CINRG-DNHS

Long-term effects of CS

Unclear; at > 10 per patient

Mcdonald, 2018 (2) [43]

Ambulatory and non-ambulatory

330

Internationalb

11.2

CINRG-DNHS

CS use and pulmonary function in DMD

Mean, 6.1

McKane, 2017a [34]

Ambulatory and non-ambulatory DMD

85

US

14.9

Single center chart review

Assoc. of body habitus with age at cardiomyopathy

Unclear; 6 per patient

Pandya, 2018a [35]

Adults (non-ambulatory) with DMD

208

US

Unstated; 'adults'

MD STARnet

Clinical course among adult DMD patients

Unclear; likely > 10 per patient

Posner, 2016a [25]

Ambulatory and non-ambulatory DMD

77

US

14.1

Single center chart review

Skeletal muscle and cardiac dysfunction

Unclear; 18 per patient

Schram, 2013 [45]

Boys with DMD treated with RAAS antagonists to prevent cardiomyopathy

63

Canada

9.1

Single center chart review

Characterize natural history

Mean, 11.3 (Overall)

Thomas, 2012a [47]

Patients undergoing cardiac evaluation

55

US

10.6

Single center chart review

To assess elevated heart rate and cardiomyopathy onset

Mean, 4.6

Van Dorn, 2018a [44]

DMD with baseline DMD with normal LV function

101

US

12.0

Multicenter chart review

Assoc. between genotype and age at LV dysfunction

Mean, 5.4

Velasco, 2007 + [38]

Non-ambulatory DMD; underwent spinal stabilization

56

US

14.0

Single center chart review

Compare rate of respiratory decline

Unclear; 12 per patient

Wang, 2018 (2) [26]

Genotyped DMD

765

US

NR

The Duchenne Registry

Age at LOA x genotype

NR

Wang, 2018a [46]

DMD on cardiopulmonary therapies

57

US

18.1

Single center chart review

Progression among cardiac patients with DMD

Mean, 7.1

Wong, 2017 [37]

Early DMD; likely ambulatory and not ventilated

95

US

5.1

Single center chart review

Clinical outcomes and AEs of CS

Mean, 8.5

  1. y = year; RU = resource use; MAX = maximum; DMD = Duchene muscular dystrophy; MD STARnet = Muscular Dystrophy Surveillance, Tracking, and Research Network, qMR = quantitative magnetic resonance; CINRG-DNHS = The Cooperative International Neuromuscular Research Group Duchene Natural History Study; LOA = loss of ambulation, AEs = adverse events; CS = corticosteroid; MDA = Muscular dystrophy association; dx = diagnosis; RAAS = Renin–angiotensin–aldosterone system; LV = left ventricular
  2. aIncludes samples of mixed corticosteroid treatment status, + Includes samples of unknown (but likely treated) corticosteroid treatment status
  3. bThe CINRG-DNHS included 63% of participants with DMD from North America (20% from Canada and 43% from the US) [15]