Searchable abstracts of presentations at key conferences on calcified tissues

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...

ba0001pp405 | Osteoporosis: treatment | ECTS2013

Adherence to therapy: outcomes after seven years of treatment with bisphosphonates

Pastore Renato , Mentuccia Daniela

Introduction: Bisphosphonates are the first-choice treatment for osteoporosis. However, the efficacy observed in clinical trials may not be realized in a real-life setting, partly due to poor adherence to therapy, with a significant worsening of clinical outcomes. The aim of this study conducted on an outpatient cohort is to quantify the adherence to the osteoporosis treatment in real practice setting and to identify the factors that may affect it.Materi...

ba0004p54 | (1) | ICCBH2015

Vitamin D overdose: Poor dosage guidelines for babies?

Peeva Daniela , Jacobs Benjamin

Background: A 9-month-old boy was diagnosed with rickets at the age of 6 months. He was born at term in India, following a normal pregnancy. Mother had taken folic acid and calcium, but not vitamin D, as this is the policy for pregnancy vitamins in Kerala.Presenting problems: At 6 months of age he was seen for routine vaccination. The paediatrician noted a prominent forehead and swollen wrists. Wrist x-ray was said to show early rickets. Blood tests show...

ba0001pp14 | Arthritis and other joint diseases: translational and clinical | ECTS2013

Prophylaxis of gout flare with colchicine and vitamin C

Monov Simeon , Monova Daniela , Rashkov Rasho

Background: The incidence and prevalence of gout have markedly increased over the last few decades in keeping with the rise in prevalence of obesity and metabolic syndrome. The management of gout in patients with associated metabolic syndrome and comorbid illnesses such as renal impairment was difficult because of limited treatment options. Recent efficacy and safety data favour lower over higher doses of colchicine, and oral corticosteroids over non-steroidal anti-inflammator...

ba0006p010 | (1) | ICCBH2017

Transient hyperphosphatasemia in a child with nephrolithiasis And history of severe prematurity

Kutilek Stepan , Formanova Daniela , Senkerik Marian , Skalova Sylva , Markova Daniela , Langer Jan

Background: Transient hyperphosphatasemia of infancy and early childhood (THI) is a benign, usually accidentally detected condition characterized by transiently increased activity of serum alkaline phosphatase (S-ALP), its bone or liver isoform, in children under five years of age, without signs of metabolic bone disease or hepatopathy. When encountered in a child with either chronic bone, liver or kidney disease, THI might concern the physician. We present a patient with urol...

ba0001pp404 | Osteoporosis: treatment | ECTS2013

Sequential therapy after PTH 1–84 treatment: comparison among bisphosphonates and strontium ranelate

Pastore Renato , Mentuccia Daniela , Pasqualetti Patrizio , Frajese Gaetano

Introduction: Evidence in literature shows how is useful to use antiresorptive drugs such as bisphosphonates, in severe osteoporosi severe after PTH (1–34 or 1–84) treatment.Methods: This study was divided into two parts: the first one analyzed BMD changes by DXA at the lumbar and femoral and serum osteocalcin and β-CTX, monitoring their performance after 6, 12 and 18 months in 71 women with severe postmenopausal osteoporosis, treated for ...

ba0003pp334 | Osteoporosis: treatment | ECTS2014

Comparison of efficacy teriparatide between denosumab and on hip BMD in women with severe post-menopausal osteoporosis

Pastore Renato , Mentuccia Daniela , Pasqualetti Patrizio , Frontoni Simona

Introduction: Teriparatide is a potent anabolic drug that has demonstrated efficacy on fracture risk reduction in women with severe postmenopausal osteoporosis. Denosumab, designed to inhibit RANKL (RANK ligand), is a fully human MAB for the treatment of osteoporosis. BMD changes, measured by DXA, is an established tool for monitoring the effects of anti-osteoporotic therapy. Our purpose is to compare teriparatide vs denosumab efficacy on hip BMD variations, in postmenopausal ...

ba0006p150 | (1) | ICCBH2017

DXA based evaluation of the bone mass and body composition in a group of Romanian cystic fibrosis children

Barbu Carmen Gabriela , Lungu Diana , Comanici Valentina Daniela , Stan Iustina

Background: The significant increase in the life expectancy of the patients with cystic fibrosis (CF) came with some costs, as new complications have emerged. Among endocrine disorders, CF-related bone disease (CFBD) is a leading complications reported in the adult patients.Objectives: Our study present the first results of the bone mass and body composition evaluation by DXA in a small group of Romanian children with CF treated in the author’s depa...