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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]
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172