Cittadini 2003.
Methods | Randomised, double‐blind, parallel‐group (pilot study) | |
Participants | 6 participants with DMD (8 to 19 years) and 10 participants with BMD (24 to 55 years) with documented cardiac involvement Mean age: DMD 13 years (SD 2); BMD 39 years (SD 3) Background therapy unchanged in all participants DMD or BMD diagnosis biopsy‐confirmed; no other inclusion/exclusion criteria specified |
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Interventions | Weekly growth hormone 0.23 mg/kg/week (DMD) and 0.07 mg/kg/week (BMD) Placebo Self‐injected, subcutaneously at bedtime for 3 months |
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Outcomes | Hormonal measures, ECG (cardiomyopathic index), ECG cardiomyopathic index (QT‐PQ ratio), echocardiography (M‐mode, 2D and echo‐Doppler), measures of left ventricular size and function, timed function tests ((timed Gowers' manoeuvre, time to climb 4 standard stairs, timed 10 metre walk, and 'dynamic index'). Pulmonary function measures comprised forced vital capacity (FVC), maximal voluntary ventilation, and maximal expiratory pressure | |
Funding sources | Grant from Telethon | |
Declarations of interest | None given | |
Notes | Dates of enrollment and follow‐up not reported Location: Italy |
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Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Random sequence generation (selection bias) | Unclear risk | Stated to be randomised but method of randomisation not described |
Allocation concealment (selection bias) | Unclear risk | Not described |
Blinding of participants and personnel (performance bias) All outcomes | Low risk | Placebo‐controlled |
Blinding of outcome assessment (detection bias) All outcomes | Low risk | Investigators performing the echocardiographs were blind to treatment allocations |
Incomplete outcome data (attrition bias) All outcomes | Unclear risk | The manuscript did not mention if there were any dropouts or withdrawals |
Selective reporting (reporting bias) | High risk | The report provided no numerical data for left ventricular mass index, end‐systolic stress and ejection fraction outcomes, which were presented graphically |
Other bias | High risk | The age range of participants was wide and ranged from 8‐19 years for DMD and 24‐55 years for BMD and they were all taking multiple other treatments for cardiomyopathy BMD: 'background' therapy including fosinopril 20 mg/day to 30 mg/day (ACE inhibitor), warfarin, magnesium supplements, pidolatum, antioxidants (vitamins E, C, glutathione, ubiquinone), furosemide, deflazacort. One participant in each group was also receiving digoxin and amiodarone. All DMD participants were receiving deflazacort, fosinopril and antioxidants (vitamin E, glutathione and ubiquinone) |