Ward 2010.
Methods | Multicentre, parallel, double‐blinded placebo‐controlled RCT. | |
Participants | 139 randomised 4 ‐ 19 years (ALN 11.0 (3.6) years; placebo 11.1 (4.0) years 78 males 61 females Children and adolescents with a diagnosis of OI types III or IV or a diagnosis of OR type I associated with 1 or more of the following: chronic pain; more than 3 fractures (including vertebrae) per year with minimal trauma for the previous 2 years; or limb deformity requiring surgery. |
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Interventions | ALN or placebo for 24 months (< 40 kg) 5 mg daily (> 40 kg) 10 mg daily Placebo = 30 ALN = 109 Analysed: ALN = 83; placebo = 26 |
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Outcomes | Fracture reduction Change in BMD as assessed by DEXA Change in biochemical markers of bone and mineral metabolism Growth Bone pain PEDI score Grip strength |
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Notes | ||
Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Random sequence generation (selection bias) | Unclear risk | Randomized in a 3:1 ratio and stratified according to their weight at baseline. |
Allocation concealment (selection bias) | Unclear risk | Not stated. |
Blinding (performance bias and detection bias) All outcomes | Low risk | double blind. outcome assessors blind. |
Incomplete outcome data (attrition bias) All outcomes | Low risk | Total = 30, ALN = 26, Placebo = 4. Reasons given per group in figure 1 – consort diagram. Modified ITT. |
Selective reporting (reporting bias) | Low risk | Outcomes reported. |
Other bias | Low risk | None. |