Bone Abstracts

Objective: To evaluate safety and efficacy after 5 years of treatment with asfotase alfa in adolescents and adults with hypophosphatasia (HPP) in a Phase 2, open-label, randomized, dose-ranging study (NCT01163149).Methods: Treatment with subcutaneous asfotase alfa 0.3 or 0.5 mg/kg per d was compared with no treatment (control) for 6 months in patients aged 13–66 years. After 6 months, all patients (treatment and control groups) received active treat...

Poor bone mineralization remains a major health problem in patients with Thal and has been linked to functional zinc deficiency despite adequate dietary intake. The global etiology of poor bone mineralization includes inadequate dietary intake of calcium and vitamin D, endocrinopathies leading to disturbed calcium homeostasis, dysregulation of the GH–IGF1 axis, and delayed puberty, all resulting in limited growth, decreased bone formation and increased bone resorption. Th...

Clinical trials involving drug therapies for postmenopausal osteoporosis typically compare effects of the active drug combined with vitamin D (vit D) and calcium (Ca) vs vit D and Ca supplementation on its own. Bone strength is estimated based on the amount of bone, frequently expressed as bone mineral density determined by dual X-ray absorptiometry, and quality of bone, hardly measured in clinical practice.The purpose of the present study was to compare...

Osteoclasts are multinucleated giant cells derived from the monocyte/macrophage lineage in the presence of receptor activator of nuclear factor kappa-B ligand (RANKL) and colony-stimulating factor 1 (CSF1). The bone remodeling process in zebrafish is incompletely understood. Here we describe several methods to quantify bone formation and resorption using a zebrafish mutant that lacks functional colony stimulating factor 1a receptor (csf1ramh5/mh5). Mice deficient in...

Many disorders cause osteosclerosis, and many exclusively affect adults. Pediatricians are likely to encounter those that are Mendelian diseases, with most still classified as ‘dysplasias’ although now understood at the gene level. Thus, there is promise for defining their molecular and biochemical pathogeneses, and for developing targeted medical treatments. Sclerosing bone dysplasias too have become the ‘turf’ of the metabolic bone disease specialist. How...

Hypoparathyroidism results in low bone turnover and increased bone mineral density (BMD). Replacing deficient PTH with rhPTH(1–84) has the potential to correct these skeletal abnormalities. To investigate the effect of rhPTH(1–84) on BMD and bone turnover markers (BTMs), data from two studies were assessed.REPLACE, a double-blind, multicenter, placebo-controlled study, randomized 134 patients with hypoparathyroidism to receive once-daily rhPTH(...

: In osteoporosis, poor adherence to bisphosphonate (BP) therapy is common, and is associated with poor outcomes and increased treatment costs (Siris 2006; Recker 2005). Although compliance is improved with monthly vs weekly dosing (Reginster 2008), no evidence suggests cycling through BP agents offers therapeutic benefit, assessed by bone mineral density (BMD). In two randomized, open-label studies in postmenopausal women aged ≥55 years previously treated with, but subo...

Objective: To evaluate musculoskeletal development using pQCT in children with coeliac disease (CD) on gluten free diet (GFD) compared with age and gender matched healthy controlsMethod: 38 children (18 males) with CD on GFD for a duration of 3.6 years (0.6, 12.5) underwent pQCT at 4%, 38 and 66% tibial sites. Bloods were collected in CD children only. Results reported as median (range).Result: Median TTG was 1.8 IU/l (0.1, 114) wi...

Objectives: To elucidate the incidence and risk factors of skeletal toxicity in children with ALL treated with the dexamethasone-based DCOG-ALL9 protocol.Methods: Prospectively, the cumulative incidence of fractures was assessed in 672 patients and compared between different subgroups using the log-rank test. Serial measurements of bone mineral density of the lumbar spine (BMDLS) were performed in 399 ALL patients using dual energy X-ray absor...

Background: Low vitamin D concentrations among children and adolescents at northern latitudes are frequently observed. Also, inverse associations between 25-hydroxyvitamin D (25(OH)D) and PTH concentrations have been found in children of different ages. More studies on the link between vitamin D status and childhood bone health are needed.Objective: To evaluate the status of serum 25(OH)D in autumn and the association between 25(OH)D concentrations and b...