Bone Abstracts

Several environmental and genetic factors may interfere on bone mass in children. However, the pediatric studies that approach the socioeconomic status and quantitative ultrasound (QUS) parameters are scarce. The aim of this study was to compare the bone mass in girls from 7 to 15 years old of different socioeconomic status. The sample consisted of 860 Brazilian girls (8.95±1.32) of different socioeconomic status. The bone mass parameter, amplitude dependent speed sound (...

The childhood is an important period for bone mass acquisition, and this tissue may be influenced by the nutritional status. However, the relation between the nutritional status and the bone parameters of quantitative ultrasound (QUS) remains unclear. The aim of this study was to verify the influence of nutritional status on bone mass assessed by QUS in male children from 7 to 10 years old. The sample consisted of 461 Brazilians pre-pubertal schoolchildren (8.30±1.13 year...

The chronic use of glucocorticoids in patients with congenital adrenal hyperplasia (CAH) may result in decreased bone mass. Therefore, using simple and accurate methods for assessing bone status in these patients could facilitate the treatment of disease. The purpose of this study was to verify the association between parameters of bone mass measured by dual energy X-ray absorptiometry (DXA) and quantitative ultrasound (QUS) of proximal phalanges in children adolescents with c...

The quantitative ultrasound (QUS) of the proximal phalanges has been used for the indirect evaluation of bone status. Furthermore, the relative simplicity and non-exposure to radiation, presents advantages for the use of QUS compared to other methods in children and adolescents. The aim of this study was to determine the influence of age and pubertal development on Amplitude Dependent Speed Sound (AD-SoS) assessed by QUS of proximal phalanges in girls and boys aged 10–15 ...

Engulfment adaptor phosphotyrosine-binding (PTB) domain containing 1 (GULP1) is an adaptor protein involved in the engulfment of apoptotic cells via phagocytosis. Although GULP1 is widely expressed in various tissues, including the brain, muscle, testis, and bone, the function of GULP1 has not been well studied. Here, we investigated whether GULP1 plays a role in the regulation of bone remodeling and examined its expression in bone cells. Conditional Gulp1 floxed mice...

Paget disease of bone (PDB) is a common metabolic bone disease characterized by increased bone resorption and disorganized bone formation which can affect single or multiple sites of bone. Although the exact cause of PDB is still controversial, genetic factor is considered to play an important role in PDB. The causative gene of classical PDB was identified as Q8STM1 gene. Familial expansile osteolysis caused by the mutation of TNFRSF11A(RANKL) gene a...

Objectives: In XLH, FGF23-mediated hypophosphatemia leads to defective bone mineralization and rickets. Investigational product KRN23 binds FGF23 and inhibits its activity. The objective of this Phase 2 study was to evaluate the safety and efficacy of KRN23 in 52 children with XLH (ages 5–12 years, ≤Tanner 2).Methods: Patients were randomized to receive KRN23 biweekly (Q2W) or monthly (Q4W) by SC injection. KRN23 dose was titrated (maximum 2 m...

Objectives: In XLH, musculoskeletal outcomes of current treatment with oral phosphate (Pi)/active vitamin D are suboptimal for many patients. In a Phase 2, open-label study, we tested the hypothesis that KRN23 improves rickets and functional outcomes in XLH children.Methods: Fifty-two children with XLH (ages 5–12 years at baseline) received KRN23 subcutaneously biweekly (Q2W) or monthly (Q4W). At study entry, most participants had received oral Pi/a...

Objective: We evaluated the long-term efficacy of burosumab, a monoclonal antibody against FGF23, in a Phase 2 Study (NCT02163577) in children with XLH.Methods: Fifty-two children with XLH (5-12 years-old, Tanner ≤ 2) were randomized 1:1 to receive subcutaneous burosumab Q2W or Q4W for 64 weeks. Doses were titrated up to 2 mg/kg/dose targeting serum phosphorus levels within 1.1–1.6 mmol/l. All subjects entered the long-term extension at Week 6...

Osteoporosis (OP) is characterised by low bone mass and microarchitectural deterioration of the bone tissue, leading to enhanced bone fragility and increased fracture risk. OP causes an imbalance in the cellular remodelling process thus inducing changes in the bone’s mineral and organic phases that are responsible for its strength and stiffness. The purpose of this study is to investigate the early effects of OP progression on the arrangement of bone tissue mineral phase ...