Bone Abstracts

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"...

Objective: Childhood onset Crohn’s disease (CO-CD) is associated with musculoskeletal deficits. However, there are limited data regarding muscle-bone outcomes in adults with CO-CD. The current study aimed to comprehensively assess the muscle-fat-bone unit in young adults with CO-CD, using novel methods of MRI, in comparison with healthy controls.Methods: Trabecular microarchitecture and cortical geometry of the distal femur were assessed using 3T mi...

Objectives: Progressive heterotopic ossification in fibrodysplasia ossificans progressiva (FOP; OMIM #135100) begins in childhood and leads to irreversible restriction of movement, functional impairment, and shortened life-span. Baseline data from an on-going, global, 3-year, natural history study (NHS) describe FOP disease characteristics, and retrospective flare-up history, causes/symptoms, and outcomes.Methods: Data from 101 subjects (recruited from 2...

Background: There is still limited information on changes in growth especially segmental growth and bone mass of glucocorticoid(GC) treated boys with Duchenne Muscular Dystrophy (DMD).Objectives: To evaluate changes in growth and bone mass in GC treated boys with DMD.Methods: Retrospective study of 15 boys with DMD treated with GC, median age 7.6 years (4.1, 15.5) who had repeated DXA scan for clinical monitoring of bone health, me...

Objectives: To evaluate musculoskeletal trajectories in children with newly diagnosed Crohn’s disease (CD), and to determine whether children treated with anti-tumour necrosis factor-alpha antibody (anti-TNF, TREATED vs NAÏVE) had persistent deficits at two years.Methods: This was a single-centre prospective, observational inception cohort study. Children with CD underwent assessments within 6.5±9.5 days from diagnosis and annually for two...

Background: The role of untrained observers in evaluating vertebrae height and therefore detection of vertebral fracture (VF) from spinal radiographs (SXR) and dual energy-absorptiometry (DXA) images in children with concerns about osteoporosis is currently unknown.Objective: To assess intra-observer agreement of morphometric measurements of vertebra height by an untrained observer using SXR and DXA in boys with Duchenne Muscular Dystrophy (DMD).<p c...

Background: Previous studies with small number of boys with Duchenne Muscular Dystrophy (DMD) suggest that growth failure occurs in glucocorticoid naïve (GC) boys.Objective: To evaluate height and longitudinal growth in boys with DMD prior to GC.Method: Retrospective evaluation in boys with DMD with height measurements obtained for clinical purposes. Out of the 91 boys currently managed in Scotland, 51 had at least one height ...

Fibroblast growth factor-23 (FGF23) controls serum phosphorus by acting on the kidneys to excrete phosphorus and reduce calcitriol. These actions are well established in adults and children, but whether FGF23 regulates fetal phosphorus metabolism is unknown. We used X-linked Hyp or Phex null male fetuses to study the effect of excess FGF23 on fetal phosphorus metabolism. Phex+/− females and WT males were mated to generate WT, Phe...

Background: Height is required for interpretation of bone mineral density in children and is often challenging in non-weight bearing children. Monitoring of linear growth in non-weight bearing children as part of assessment of bone health is also important.Objective: To investigate the feasibility of a novel method of using DXA images to measure height (Ht), sitting height (SH) and leg length (LL).Methods: Ht and SH were measured o...