Searchable abstracts of presentations at key conferences in endocrinology

ea0035s19.2 | Osteoporosis - An update | ECE2014

Denosumab: current perspectives

Briot Karine

The interaction of RANKL with RANK is critical for the formation and function of bone-resorbing osteoclasts. Denosumab, a fully human MAB against RANKL, is an anti-resorptive drug that acts by preventing RANKL from interacting with RANK on the osteoclast precursor cells. Twice-yearly denosumab treatment is associated with markedly improved bone mineral density (BMD) and cortical and trabecular bone strength, and significantly reduced osteoporotic fracture. In clinical studies,...

ea0081p34 | Calcium and Bone | ECE2022

Skeletal consequences of long-term replacement therapy with human recombinant PTH(1-34) in chronic hypoparathyroidism

Fischler Rebecca , Lecoq Anne-Lise , Briot Karine , Trabado Severine , Besson Florent , Bouziotis Jason , Goujard Cecile , Salenave Sylvie , Leboulleux Sophie , Carreira Emma , Chabrolle Christine , Bernard Corvilain , Chanson Philippe , Linglart Agnes , Grimon Gilles , Kamenicky Peter

Context: In patients with hypoparathyroidism refractory to conventional treatment, recombinant human (rh)PTH(1-84) or rhPTH(1-34) can be used as second-line therapy and effectively control hypocalcaemia. However, whether rhPTH replacement therapy is safe in the long term is unclear. Our objective was to assess bone effects of long-term therapy of chronic hypoparathyroidism with rhPTH(1-34). Methods: We conducted a monocenter retrospective cross-sectional...

ea0063oc7.3 | Endocrine Connections 1 | ECE2019

Higher dose of burosumab is needed for treatment of children with sever forms of X-linked hypophosphatemia

Zhukouskaya Volha V , Audrain Christelle , Lambert Anne-Sophie , Colao Annamaria , Kamenicky Peter , Adamsbaum Catherine , Nevoux Jerome , Chaussain Catherine , Wicart Philippe , Briot Karine , Rocco Federico Di , Trabado Severine , Prie Dominique , Rothenbuhler Anya , Linglart Agnes

Background/aim: Burosumab is a monoclonal antibody against anti-FGF23, which has been recently approved for the treatment of X-linked hypophosphatemia (XLH). Beyond clinical trials, little is known about its efficacy/safety in clinical practice which is the aim of the present study.Patients/methods: Thirty-nine children with XLH were switched from conventional therapy to burosumab (starting dose 0.4 mg/kg), on the basis of following indications: non-resp...

ea0070aep122 | Bone and Calcium | ECE2020

Phase 3b open-label study of burosumab in adults with X-linked hypophosphatemia (XLH): Baseline and Week 12 results

Kamenický Peter , Javaid Kassim , Keen Richard , Lachmann Robin , Ralston Stuart , Cohen-Solal Martine , Brandi Maria , Briot Karine , Crowley Rachel , Walsh Jennifer , Kolta Sami , Rylands Angela , Sun Wei , Nixon Annabel

Introduction: Burosumab, a fully human monoclonal antibody to fibroblast growth factor 23 (FGF23), is the only approved treatment for XLH, a rare genetic disorder characterized by renal phosphate wasting and substantial cumulative musculoskeletal morbidity. BUR02 (NCT03920072) is a European phase 3b open-label study monitoring the long-term safety and efficacy of burosumab in adults with XLH from sites who participated in the CL303/CL304 studies (NCT02526160/02537431).<p c...

ea0070oc2.1 | Bone and Calcium | ECE2020

Real-life clinical study: 1-year of treatment with burosumab of children and adolescents affected with X-linked hypophosphatemia

Zhukouskaya Volha , Mannes Ines , Chaussain Catherine , Audrain Christelle , Lambert Anne-Sophie , Adamsbaum Catherine , Kamenicky Peter , Nevoux Jerome , Wicart Philippe , Briot Karine , Di Rocco Federico , Trabado Séverine , Prié Dominique , Di Somma Carolina , Colao Annamaria , Rothenbuhler Anya , Linglart Agnès

Background/Aim: X-linked hypophosphatemia (XLH) is a rare disease caused by PHEX mutation, leading to elevated FGF23, renal phosphate wasting, hypophosphatemia, insufficient 1,25(OH)2D synthesis. Clinically, it manifests with rickets including leg deformities, poor growth, dental abscesses, craniosynostosis, and hearing loss. Beyond conventional treatment (phosphate supplements + active vitamin D), burosumab is pathogenetic anti-FGF23 therapeutic approa...

ea0056oc3.1 | New insights in bone disorders | ECE2018

A Phase 3 randomized, double-blind, placebo-controlled study investigating the efficacy and safety of Burosumab, an anti-FGF23 antibody, in adult X-Linked Hypophosphatemia (XLH)

Kamenicky Peter , Lachmann Robin , Carpenter Thomas O. , Cohen-Solal Martine , Eastell Richard , Brandi Maria Luisa , Crowley Rachel K. , Ralston Stuart H. , Javaid Muhammad K. , Keen Richard , Briot Karine , Il Cheong Hae , Imanishi Yasuo , Ito Nobuaki , Tanaka Hiroyuki , Zhang Lin , Theodore-Oklota Christina , Mealiffe Matt , Martin Javier San , Insogna Karl L.

UX023-CL303 is an ongoing, Phase 3, double-blind, multicenter study examining the efficacy and safety of burosumab, a fully human monoclonal antibody against FGF23, in adults with XLH. Eligible subjects had serum phosphorus levels <0.81 mmol/l and skeletal pain (BPI – Worst Pain ≥4). Subjects (N=134) were randomized 1:1 to receive burosumab 1 mg/kg or placebo subcutaneously every 4 weeks. After 24 weeks, subjects in the placebo group crossed-over to rec...