Bone Abstracts

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...

Objectives: Osteopenia is a complication of childhood cancer treatment but it is unknown to which extend this occurs in adult survivors, and which subgroups are at risk. We examined bone mineral density (BMD) and assed the relative importance of potential determinants of low BMD in very long-term adult survivors of childhood cancer.Methods: The single-center cohort study included 410 adult CCSs (median age at diagnosis: 6.6 years (range: 0–17 years)...

Objectives: Osteonecrosis (ON) and decline of bone mineral density (BMD) are serious side effects during and after treatment of childhood acute lymphoblastic leukemia (ALL). It is unknown whether ON and low BMD co-occur in the same patients, and whether these two osteogenic side-effects can mutually influence each other’s development.Methods: BMD and the incidence of symptomatic ON were prospectively assessed in a national cohort of 466 patients wit...

Objective: Increase the percentage of elderly fracture patients undergoing a dual energy X-ray absorptiometry (DXA) measurement, and investigate why some patients did not respond to invitation to our fracture liaison service (FLS).Materials and methods: In four Dutch hospitals, fracture patients ≥50 years were invited for a DXA measurement and visit to our FLS. Patients who did not respond, were contacted by telephone. In patients diagnosed with os...

Introduction: Osteopenia is a well-known complication of anorexia nervosa (AN) in older adolescents and adults, especially in those with a long duration of the disease and a severe underweight.Aim: We investigated whether young premenarchal girls with AN have similar risk factors for a disturbed bone growth and mineralization.Methods: Twenty-four female premenarchal AN patients as well as 24 age and height matched female controls u...

Objectives: This study aims to evaluate and compare the preferences of patients and health care professionals for osteoporotic drug treatment.Materials and methods: A discrete choice experiment was conducted among patients and health care professionals (general physicians, rheumatologists, and geriatricians) in Belgium. Participants were asked to choose between two hypothetical unlabelled drug treatments (and an opt-out option) that vary in several attri...

Introduction: Despite similarities in upfront treatment, impairment of bone mineral density(BMD) varies in long-term adult survivors of childhood cancer (CCS). We studied for the first time whether genetic variation is involved in impairment of BMD in adult long-term CCS.Method: This cross-sectional single-center cohort study included 334 adult CCS (median follow-up time: 15.2 years (range 5.1–39.8); median age at follow-up: 26.1 years (range 18.1&#...

Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure. Data from adult ADPKD population show increased fibroblast growth factor 23 (FGF23) levels while circulating Klotho levels decrease, with a low TmP/GFR even in patients with normal renal function. Moreover, in ADPKD animal models, cyst lining renal cells were demonstrated to produce FGF23, although the animals displayed FGF23 resistance. No data are available in a paediatr...

Signaling pathways crucial in bone development, including Wnt, are also upregulated in breast cancer cells to promote tumor growth in the skeleton, a process known as osteomimicry. Thus, we hypothesized that bone metastatic tumor cells also aberrantly express osteogenic miRNAs to support osteomimetic properties. We have previously shown that miR-218 is highly expressed in osteoblasts and promotes osteogenic differentiation. Interestingly, expression analysis revealed a signifi...