Bone Abstracts

Objectives: Endocrine late effects, including osteopathologies, following diagnosis and treatment of childhood malignancies are studied in adult survivors with alarming results. However, in pediatric patients with brain tumors the risk to develop endocrine late effects is high even during childhood and adolescence.Aim and design: To investigate osteopathologies and endocrine function in juvenile survivors of pediatric brain tumors we conducted a cross-se...

Objective: We constructed height growth curves for children with XLH from birth to early adolescence, a majority of whom received conventional therapy consisting of multiple daily doses of oral phosphate and active vitamin D.Methods: Growth data from four clinical studies were pooled to construct the growth curves. UX023-CL002 was an observational, retrospective chart review of 103 children with XLH, 1–14 years of age. Pre-treatment data were collec...

McCune-Albright-Syndrome (MAS) is a rare multisystem disorder presenting with polyostotic fibrous dysplasia accompanied by a list of extraskeletal features including café-aulait spots and various endocrine hyperfunctioning. There is no effective treatment for FD in MAS nowadays. Patients with MAS (n=60, 49 girls(G) and 11 boys (B) have been diagnosed and followed up during 20 years in the Institute of Pediatric Endocrinology. First clinical manifestations were pe...

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...