Searchable abstracts of presentations at key conferences on calcified tissues

ba0007p144 | (1) | ICCBH2019

A multi-criteria decision analysis of the value of burosumab for the treatment of paediatric patients with X-linked hypophosphatemia in Portugal

Alves Ines , Francisco Telma , Pinto Helena , Sousa Sergio , Connor Paul , Amorim Miguel , Ramos Diogo , Azevedo Sofia , Vandewalle Bjorn

Objectives: Burosumab is an anti-FGF23 fully human monoclonal antibody, recently approved for the treatment of X-linked hypophosphatemia (XLH), presenting a novel treatment approach compared to conventional therapy (CT), composed of oral phosphate and active vitamin D. The objective of this study is to perform a Multi-Criteria Decision Analysis (MCDA) to assess the value of burosumab for the treatment of paediatric patients with XLH in Portugal, in comparison to CT.<p clas...

ba0007p72 | (1) | ICCBH2019

Burosumab experience in UK X-linked hypophosphataemia children under five years old

Dharmaraj Poonam , Burren Christine , Cheung Moira S , Padidela Raja , Mughal Zulf , Shaw Nick , Saraff Vrinda , Nadar Ruchi , Randell Tabitha , Mushtaq Talat , Ramakrishnan Renuka , Sennipathan Senthil , Sakka Sophia , Bath Louise , Elleri Daniela , Davies Justin H , Barton John , Tucker Ian , Rayner Lauren , Arundel Paul , Gilbey-Cross Robyn , Tothill Alexander M , Philip James , Sawoky Nadine , Connor Paul , Mathieson Leigh

Objectives: X-linked hypophosphataemia (XLH) is a rare inherited form of osteomalacia characterised by low blood phosphate levels which lead to inadequate mineralisation of bone and rickets. Burosumab is an anti-FGF23 fully human monoclonal-antibody, and the first treatment to target the underlying pathophysiology of XLH. Real-world evidence is important in validating the findings of clinical studies. We report relevant real-world biochemical data on children under five years ...

ba0007p73 | (1) | ICCBH2019

Burosumab initiation in a UK X-linked hypophosphataemia cohort: real-world use resonates with research evidence

Dharmaraj Poonam , Burren Christine , Cheung Moira S , Padidela Raja , Mughal Zulf , Shaw Nick , Saraff Vrinda , Nadar Ruchi , Randell Tabitha , Mushtaq Talat , Ramakrishnan Renuka , Sennipathan Senthil , Sakka Sophia , Bath Louise , Elleri Daniela , Davies Justin H , Barton John , Tucker Ian , Rayner Lauren , Arundel Paul , Gilbey-Cross Robyn , Tothill Alexander M , Philip James , Sawoky Nadine , Connor Paul , Mathieson Leigh

Objectives: X-linked hypophosphataemia (XLH) is a rare inherited form of osteomalacia characterised by low blood phosphate levels which lead to inadequate mineralisation of bone resulting in rickets, skeletal abnormalities, physical impairment, weakness, and pain. Burosumab is an anti-FGF23 fully human monoclonal-antibody, and the first treatment to target the underlying pathophysiology of XLH. Real-world evidence is important in validating the findings of clinical studies. We...