Bone Abstracts

Objectives: Deteriorating bone mineral density and fragility fractures are common complications of Duchenne Muscular Dystrophy (DMD). This study aims to evaluate the effects of bisphosphonate therapy and testosterone supplementation (for pubertal induction) on the bone health of children with DMD.Methods: This retrospective cohort study examined the clinical records of children with DMD managed at the Neurogenetics and Endocrine clinics at The Children&#...

Objectives: To evaluate the diagnostic outcomes of massively parallel sequencing (MPS) and identify genotype-phenotype correlations in children and adolescents with recurrent fractures.Methods: A retrospective chart audit was conducted of children and adolescents referred to the endocrine clinic at a tertiary children’s referral hospital with a history of recurrent fractures. Phenotypic data including fracture history, bone mineral density (BMD) and...

Objectives: To evaluate the clinical outcomes of intravenous bisphosphonate treatment in children with mild-moderate osteogenesis imperfecta (OI) who had progressed from active bisphosphonate treatment to maintenance therapy for >2 years.Methods: A retrospective review was conducted on 17 patients with mild-moderate OI. Clinical data, fracture history, biochemistry, dual energy X-ray absorptiometry (DXA) parameters, vertebral measurements, bone age a...

Objectives: Bisphosphonate infusions are associated with numerous adverse effects including acute systemic inflammatory reactions and electrolyte abnormalities. The aims of this study were to evaluate the safety and efficacy of a 3-day course of prednisone on children receiving their first dose of pamidronate or zoledronic acid.Methods: A retrospective cohort of 166 patients (85 males) were commenced on pamidronate (16%) or zoledronic acid (84%) for trea...

Objectives: Childhood osteoporosis can be treated with i.v. bisphosphonates in order to improve bone mass and density. The aims of this study were to evaluate the safety and efficacy of 6-monthly zoledronic acid (ZA) in children with osteoporosis.Methods: A retrospective cohort study of 27 patients (16 males and 11 females) were treated with 6-monthly ZA (0.05 mg/kg per dose) for a minimum of 1 year. Seventeen patients were immobile, 4 had steroid-induce...

Objectives: To compare and contrast the natural history of osteoporosis and response to zoledronate in children and adolescents with Duchene muscular dystrophy (DMD), spinal muscular atrophy (SMA) or other congenital muscular dystrophies (CMD).Methods: A retrospective medical record review of fracture history, treatment and bone mineral densitometry of children managed at a tertiary centre in Sydney over the last 6 years.Results: A...

Objective: The potential for clinical trials to impact patient care may be limited if the outcomes reported vary by trial and lack direct relevance to patients. We aimed to systematically assess the scope and consistency of outcomes reported in randomised trials of interventions for children with osteogenesis imperfecta.Methods: We systematically searched for all published and unpublished randomised trials of interventions for children with osteogenesis ...

Objectives: To evaluate the effect of 24 weeks of weight bearing vibration therapy (WBVT) on muscle strength, motor function and bone health in children with congenital myopathies.Methods: A prospective pilot study incorporating a six month observational period followed by 6 months home-based WBVT (Galileo® Pro) was undertaken. Ambulant children with congenital myopathies aged 4–16 years were eligible for inclusion. Participants were assessed a...

Background: Denosumab (DMAB) is used in adults for the treatment of osteoporosis, giant cell tumour of bone, and cancer metastases. There are little data on paediatric use with clinical decision making reliant on adult data and clinical experience.Presenting problem: We have treated 33 children with DMAB: Perthes disease (n=9), avascular necrosis (n=17), osteoporosis (n=1), aneurysmal bone cyst (n=4) and giant cell tum...