Bone Abstracts

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...

Objectives: Asfotase alfa (AA), an enzyme replacement therapy, is the only approved treatment for pediatric-onset hypophosphatasia (HPP). We evaluated the safety profile of AA from the clinical trial program spanning pediatric and adult patients.Methods: Safety data were pooled from 4 open-label, multicenter studies in children aged ≤3 years (study 002/003 [NCT00744042/NCT01205152]; n=11) and ≤5 years (study 010-10 [NCT01176266]; <em...

In XLH, high circulating FGF23 causes hypophosphatemia, rickets, and short stature. In our Phase 2 study, 52 XLH children (ages 5-12 years, ≥Tanner 2) were randomized to receive KRN23 subcutaneously biweekly (Q2W) or monthly (Q4W). Serum phosphate (Pi) was measured biweekly. KRN23 dose was titrated (maximum 2 mg/kg) targeting age-appropriate serum Pi concentrations.The first 36 subjects had a mean 6.6 years of standard-of-care treatment before wash...

Objective: We compared the efficacy and safety of burosumab, a monoclonal antibody against FGF23, to conventional therapy [oral phosphate and active vitamin D (Pi/D)] in children with X-linked hypophosphatemia (XLH).Methods: In this Phase 3 trial (NCT02915705), 61 children with XLH (1-12 years-old) were randomized 1:1 after a 7-day Pi/D washout to receive burosumab starting at 0.8 mg/kg SC Q2W or reinitiate Pi/D optimally titrated by investigators. Eligi...

Introduction: Giant cell tumour of the bone (GCTB) is a benign, locally aggressive tumour whose neoplastic stromal cells express receptor activator of nuclear factor kappa-B ligand (RANKL) and activate its receptor RANK on osteoclast-like giant cells. Denosumab (RANKL inhibitor) is an FDA/EMA approved treatment for GCTB in adults and ‘skeletally mature’ adolescents. Safety concerns include oversuppression of bone remodelling, with risk of osteonecrosis of the jaw [ON...

Osteoporosis-related hip fractures represent a substantial cause of mortality and morbidity in industrialized countries; nonetheless, past studies in Austria lack mortality figures save during hospitalization (in-hospital mortality).We therefore retrospectively retrieved pseudonymized invoice data from Austrian social insurance authorities covering roughly 98% of the entire population including 31 548 subjects over 51 years of age who sustained first hip...

Osteoporosis is commonly known as the prime risk factor for hip fracture in the elderly. We thus evaluated status and effect of osteoporosis treatment amongst hip fracture patients in a large Austrian cohort.Retrospectively retrieved pseudonymized invoice data from Austrian social insurance authorities covering roughly 98% of the entire population included 31 548 subjects over 50 years with first hip fractures between July 2008 and December 2010, with fo...

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In type 2 diabetes mellitus (T2DM) patients, an increased fracture risk is observed, although the bone mineral density is even higher than in non-diabetic patients. This raises the question of the quality of the organic and inorganic matrix in bone1–3. T2DM is also known to forward dysfunctions in the development of soft tissues such as brittle skin due to cross-linking of the collagen or inflammation of the gingiva. For the latter, a possible influence of diab...

Bisphosphonates (BPs) including alendronate (ALN) are the most widely prescribed therapy for post-menopausal osteoporosis. Despite their overall excellent safety record and efficacy in reducing fractures, concerns have been expressed lately regarding potential detrimental effects due to prolonged bone turnover reduction, although no definite cause–effect relationship has been established to date. The purpose of the present study was to determine bone material quality by R...