Bone Abstracts

Objectives: Respiratory and Ear, Nose and Throat (ENT) complications are commonly reported in children with achondroplasia but little data is available regarding prevalence and outcome. In an unselected cohort of children with achondroplasia, we studied the prevalence of:•Abnormal cardiorespiratory sleep studies•Speech and hearing impairment</ce:p...

Short stature and osteoporosis are common in DMD. Disease progression can be slowed by glucocorticoids but these are associated with further growth retardation and skeletal fragility. The defect in growth and skeletal development in children with DMD is probably multifactorial and not solely dependent on glucocorticoid exposure. The muscular dystrophy x-linked (mdx) mouse is the most commonly used animal model of DMD. However, its growth phenotype has not been studied...

The cathepsin K inhibitor odanacatib (ODN) is a bone formation-sparing inhibitor of osteoclastic resorption activity. This drug is currently under development for the treatment of postmenopausal osteoporosis. To support the bone safety profile of ODN, we evaluated the effects of ODN on trabecular bone hard tissue properties in the estrogen-deficient model of the ovariectomized (OVX) rhesus monkeys. Animals (n=16/group, age 11–22 years) were treated immediately af...

In children with spinal muscular atrophy (SMA): i) describe bone mineral density (BMD) measured by dual-energy x-ray absorptiometry (DXA); ii) examine bone health factors; iii) examine change in BMD over time and pamidronate effect.DXA results, medications, ambulation, 25-OH-Vit D levels, and fracture history were retrospectively reviewed in 14 children (five girls) with SMA. Sites scanned included whole body (WB), lumbar spine (LS), and lateral distal f...

Objective: To examine the bone mineral density (BMD), fracture occurrence and history in a cross-section of subjects with Pelizaeus Merzbacher Disease (PMD).To describe the effect of pamidronate therapy on BMD over time in treated PMD patients.Methods: We examined the medical history, medications, level of ambulation, fracture history and DXA results in 15 boys with PMD. Body sites scanned included whole body (WB) when possible, lu...

Objectives: Dual Energy X-ray Absorptiometry (DXA) is commonly used to monitor changes in bone mineral density (BMD). Small changes in BMD can be clinically meaningful; therefore precision-error calculations are needed to estimate true changes in BMD. The few published studies of precision errors in children with cerebral palsy (CP) are diverse in terms of ethnicities and medical comorbidities. Application of these estimates is inappropriate to our particular Midwest American ...

Objectives: There are conflicting data on the skeletal health of patients with perinatal HIV. We aimed to evaluate the bone profile of a paediatric population followed in a reference centre for perinatal HIV.Methods: The following data were recorded: dietary calcium intake, extra-curricular exercise, fracture history, medications and comorbidities. All patients were assessed for growth and skeletal deformities. They underwent laboratory tests:CD4 count, ...

Objectives: Examine the effects of a ramped dosage schedule of pamidronate on BMD, fracture rate and location compared to a uniform 5-course regimen. The ramping regimen is intended to alter the tendency for post-treatment fractures to occur at the juncture of pamidronate bands where stress-riser related fractures have been described.Methods: Ten non-ambulatory children (seven females) with neuromuscular disabilities who received IV pamidronate with a ta...

Objectives: Burosumab, a monoclonal antibody that therapeutically targets the underlying elevated levels of fibroblast growth factor 23 (FGF23) in X-linked hypophosphatemia (XLH), is now available to children out of trial conditions. Our objective was to describe the effect of burosumab on quality of life, functionality and pain in a clinical setting.Methods: Questionnaire tools were completed at baseline, 6 and 9 months for 9 children with XLH starting ...

Burosumab, a monoclonal antibody targeting fibroblast growth factor 23, is now available for clinical use in children with X-linked hypophosphatemia (XLH). We aimed to explore the effects of burosumab in children with XLH in a clinical setting, considering: a) Biochemistry b) Growth c) Lower limb deformity (LLD) d) Radiology e) Motor function<p class="abstext"...