Seikaly 2005.
| Methods | Single‐centre double‐blinded, placebo controlled cross‐over trial | |
| Participants | 20 CYP randomised. Age range: 3 to 15 years, 11 males. Osteogenesis imperfecta types I, III and IV. ICD‐10: Q78.0 | |
| Interventions | Route: Oral alendronate versus placebo Dose: 5 mg/day (participants who weighed < 30 kg); or 10 mg/day (participants who weighed > 30 kg) administered orally with at least 8 ounces of water, 30 minutes before food intake; patients were also advised to maintain the upright position for at least 30 minutes after ingestion of the medication to reduce the chance of oesophageal irritation. All subjects were maintained on a diet with adequate daily calcium (1000 to 1300 mg/d), phosphorus (800 to 1200 mg/d), and vitamin D (400 IU/d) intake, at least 100% daily referenced intake (DRI). Study period: Participants were evaluated at baseline, then every 3 months (except when otherwise indicated), and at the conclusion of the study (2 years) |
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| Outcomes | Primary: bone mineral density (BMD) (measured by BMD DEXA Z scores) and QoL‐functional abilities, mobility (using the modified Pediatric Evaluation of Disability Inventor (PEDI)), self‐care score (using the WeeFIM system), well‐being scores (1 to 10) and pain (likely to be nociceptive rather than neurogenic but possibly of mixed origin) including number of pain free days per month and number of days that analgesia was administered for skeletal pains (parents were asked these two questions too) Secondary: Physical evaluation, food records, blood and urine analysis, stool guaiac, renal ultrasound, skeletal survey |
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| Notes | 17 participants completed the study 2 CYP with type I OI were excluded from the QoL analysis |
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| Risk of bias | ||
| Bias | Authors' judgement | Support for judgement |
| Random sequence generation (selection bias) | Low risk | Randomisation was computer generated (SAS 6.12). Included two period cross‐over |
| Allocation concealment (selection bias) | Low risk | Pharmacist at institution assigned groups so allocation was concealed from those involved |
| Blinding of participants and personnel (performance bias) All outcomes | Low risk | Double‐blind: other members of the research team were blinded to treatment until data analysis, and participants blinded too |
| Blinding of outcome assessment (detection bias) All outcomes | Low risk | Unlikely to be detected as research team assessing outcomes were blinded and radiologists detecting the fractures were also blinded |
| Incomplete outcome data (attrition bias) All outcomes | Unclear risk | Repeated measures analysis of variance. 17/20 participants completed the 2 year study (unclear why dropped out) |
| Selective reporting (reporting bias) | Unclear risk | No information provided on the protocol so unclear |
| Other bias | Unclear risk | Some outcomes excluded CYP with type I OI and so only reflect types III and IV, unclear why |