Bone Abstracts

Objectives: Asfotase alfa (AA), an enzyme replacement therapy, is the only approved medical treatment for pediatric-onset hypophosphatasia (HPP), which is caused by deficient tissue-nonspecific alkaline phosphatase activity. We detail the long-term efficacy of AA observed from the pediatric clinical trial program.Methods: Efficacy data collected to study completion were pooled from 3 open-label, multicenter investigations of children who manifested HPP s...

Objective: Existing case series examining outcomes following spine surgery for patients with skeletal dysplasias demonstrate high rates of complications. Yet, these earlier studies are from a single institution, contain surgeries performed almost 50 years ago, and only include patients with a diagnosis of achondroplasia. The objective is to determine contemporary outcomes after spinal surgery in patients with skeletal dypslasias, focusing on complications and revisions. We pre...

Objectives: Vertebral fractures are common in boys with DMD taking daily corticosteroids. Treatment is usually initiated when vertebral fractures have been identified. However, prophylactic treatment may be possible if reliable risk factors for vertebral fracture can be identified. The aim of this work was to compare the diagnostic accuracy of three different bone strength assessment techniques in a cohort of DMD boys.Methods: Thirty-three boys with DMD ...

Odanacatib (ODN), a selective oral inhibitor of cathepsin K, is in development for the treatment of osteoporosis. In the primary efficacy analysis of the Phase 3, Long-Term ODN Fracture Trial (LOFT; NCT00529373), ODN significantly reduced fracture risk compared with placebo in postmenopausal women with osteoporosis. Pre-specified subgroup analyses evaluated the efficacy of ODN in patient subgroups.Women aged ≥65 years, without baseline radiographic...

Background: In patients with gout, urate deposition has been observed both adjacent to and within tendons, suggesting that monosodium urate monohydrate (MSU) crystals are likely to be in direct contact with tenocytes, the stromal cells of tendons. The aim of this study was to determine the effects of MSU crystals on tenocyte viability and function.Methods: Cultures of primary rat tenocytes were prepared from Wistar rat tails. Primary human tenocytes were...

The switch from a cartilage template to bone during endochondralossification of the growth plate requires dynamic and close interaction between the cartilage and the developing vascular structures. Vascular invasion of hypertrophic cartilage, with blood vessels coming from the bone collar, serves to bring in osteoblast- andendothelial precursor cells along with chondroclasts and their precursors into future ossification centres of the growth plate.Potent...

Objective: Oosteoporoti fractures are a growing problem especially in aged societies of industrialized countries. Therefore, a clinical demand for synthetic bone graft substitutes is increasing. Despite the general success CPC showed clinically, development of CPC-based material by adding stronsium could improve its suitability to treat osteoporotic fractures.Methods: Sprague–Dawley rates model of induced osteoporosis via multi-deficiencies diet com...

Bone remodeling is a crucial process to maintain a healthy bone structure in order to avoid diseases like osteoporosis or osteopetrosis. Osteoclasts contribute to this process by resorbing old and brittle bone allowing osteoblasts to renew the bone substance. During resorption osteoclasts rearrange their actin cytoskeleton by forming an F-actin ring generating a resorptive cavity on the bone surface. Recently, we reported that the F-actin binding protein SWAP-70 regulates oste...

Introduction: Lymphoblastic leukemia is the predominant form of childhood malignancies with survival rates of >80%. Late effects of cancer and treatment can affect endocrine function and may account for acute and chronic impairment of bone health.Aim and design: To assess bone health in pediatric patients after therapy for lymphoblastic leukemia we initiated a clinical trial investigating clinical and biochemical parameters of growth, puberty, bone t...

Juvenile Paget’s disease (JPD) is an extremely rare, yet painful and debilitating bone disease with onset occurring during early childhood. JPD can be caused by loss of function of osteoprotegerin, resulting in subsequent osteoclast stimulation via the activated receptor activator of nuclear factor-kappa B (RANK) pathway. Increased bone turnover and a lack of bone modelling lead to severe deformities, frequent fractures, short stature and loss of hearing.<p class="abs...