Bone Abstracts

Background: Camurati-Engelmann disease (CED) is a rare bone dysplasia characterised by hyperostosis and sclerosis of the diaphyses of the long bones and skull. It is caused by autosomal dominant gain-of function mutations within TGFB1, which result in increased activity of transforming growth factor β1 (TGF-β1). It typically presents in mid-childhood with bone pain, myopathy and progressive immobility. Evidence for treatment is based on a number of case repo...

Objectives: To determine the national prevalence, management and long term outcomes of patients who develop osteonecrosis after initiation of treatment for acute lymphoblastic leukaemia (ALL).Methods: The central trials unit for the leukaemia trial UKALL2003 identified patients with reported bone toxicity out of the 3126 patients recruited into the study. Questionnaires were sent to each relevant treatment centre requesting information about each patient...

Objectives: To assess the safety, tolerability and pharmacokinetic (PK) profile of single and multiple doses of TA-46 administered subcutaneously to healthy volunteers.Methods: This was a double-blind, randomized, placebo-controlled trial in a total of 72 subjects. Cohorts of 8 subjects were randomised to receive either TA-46 or placebo in a 3:1 ratio in single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. SAD doses were 0.3, 1, 3, 10 a...

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...

Introduction: Achondroplasia, the commonest skeletal dysplasia, is caused by specific variant(s) in the fibroblast growth factor receptor 3 (FGFR3) gene that cause abnormal spine, skull and limb bone growth. Surgical indications for foramen magnum stenosis in this population vary widely in the literature.Methods: We performed a retrospective analysis of patients with achondroplasia aged >20 years (n=33) in our regional skeletal dysplasia cli...

Hip development is influenced by mechanical loading during fetal movement by joint and reaction forces equivalent to several times bodyweight. These forces increase with fetal size and gestation length, and are lower in oligohydramnios and breech presentation which are both risk factors for developmental hip disorders. However, associations between indicators of prenatal mechanical loading and hip shape in later life remain unexplored. We therefore examined associations betwee...

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 ...

STRUCTURE was a phase 3, open-label study evaluating the effect of romosozumab or TPTD for 12 months in women with postmenopausal osteoporosis transitioning from bisphosphonate therapy (NCT01796301). This study enrolled women with postmenopausal osteoporosis who had taken an oral bisphosphonate for ≥3 years prior to screening and alendronate in the year prior to screening; had a BMD T-score ≤−2.5 at the total hip (TH), lumbar spine (LS), or femoral neck (FN);...

We have previously demonstrated the presence of circulating mesenchymal stem cells (MSCs) in fracture patients. However, the exact time for their appearance in circulation remains unknown. Nine patients (five females, four males, age 70±12 years, range 55–89 years) with a fresh femoral neck fracture were recruited in the study, which was approved by the local ethical committee. Bone marrow (BM) sample drawn from the iliac crest of all patients served as the individua...

Histomorphometric analysis is an essential technique to measure bone formation and resorption parameters. Here we present three novel open source image analysis packages that allow the rapid semi-automated analysis of histomorphometric bone resorption, osteoid, and calcein double labelling parameters.Mice were injected with calcein 5 and 2 days before killing. Tibia and vertebrae were fixed in formalin, embedded in methylmethacrylate and the blocks secti...