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 |