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Congress Report ValEncia spain CONGRESS REPORT a proDucT of This publicaTion is supporTED by Foreword from Contents Anna Teti, ECTS president Progress for rare diseases at ECTS 1 ECTS 2018 supports Camurati- The ECTS 2018 congress was again Engelmann patients’ association 7 exciting and successful. Around ECTS perspectives on clinical hot topics 9 1000 delegates attended the Omics moving forward to practical translation 11 meeting in Valencia, for teaching and learning in a ECTS Steven Boonen Award 14 relaxed and lively atmosphere. Our pre-congress day Screening for fracture risk in osteoporosis 17 was also very successful and included the Mellanby training course, the first ECTS/ICCBH workshop on ECTS and ASBMR debate screening for fracture risk 19 Rare Bone Diseases, the East meets West events, 10 Bone and ageing 21 very successful Working Gro ups and the ECTS Risk factors for bone disease 24 Academy events. Scientists were able to take advantage of the networking opportunities, clinicians could hear Clinical weight loss and bone 28 about the latest developments and best clinical Management of clinical vertebral fractures 30 practice, and young investigators could benefit from Exploring the human microbiome 33 contact with experienced colleagues and opinion New data on efficacy of osteoporosis therapies 36 leaders, including through our Meet the Expert Advances in regenerative medicine 40 sessions. ECTS aims at promoting crosstalk between Fish as a model of skeletal diseases 42 the generations and we believe this goal was fully New insights into bone and cartilage biology 44 achieved in Valencia, particularly as a result of the Cancer and bone 48 tremendous efforts of our ECTS Academy members. Bone and vascular alterations in kidney disease 52 This report will walk you through the congress Bone marrow adipose tissue – highlights. If you have missed anything or want to complex questions 55 review the presentations, this is an excellent tool for Inflammation and bone 57 you. We are very grateful to our delegates, sponsors ECTS supports New Investigators 59 and staff who made ECTS 2018 a great congress, and Insights from outside: the reproducibility crisis 60 to Michael Baldwin and the editorial team for preparing Recipients of ECTS grants and such a useful and interesting report. awards at ECTS 2018 62 The next ECTS congress in 2019 will be in Budapest, Feedback on ECTS 2018 63 Hungary. Our excellent pre-congress will be held on ECTS through the year 64 May 10th, followed by the main programme during May ECTS Academy 64 11th-14th 2019. Meanwhile, take advantage of the many activities organized by ECTS during the year. To know published by the European Calcified Tissues Society more, please visit the ECTS website, download the EcTs Executive Director: Roberta Mugnai ECTS app and register to receive the ECTS Newsletter. Website: www.ectsoc.org Twitter: @EcTs_soc Stay tuned, we care about you all year long! Editor: Michael Baldwin Professor Anna Teti other contributors: Natalie Butterfield, Alfredo Cappariello, Kashmala Carys, Annabel Curle, Melanie EcTs president Haffner-Luntzer, Antonio Maurizi, Hanna Taipaleenmäki Design/production: Sublime Creative photography: Valencia Conference Centre Video production: The Informed Scientists congress secretariat: Interplan SUPPORTED BY: © European calcified Tissues society 2018 1 Progress for rare diseases at ECTS Results of clinical trials for treatment was performed in 13 children aged 1 to 4 years receiving for X-Linked Hypophosphatemia two-weekly treatment. across all age groups, burosumab treatment was found to consistently raise mean serum At ECTS 2018 Robin Lachmann (London), presented the phosphorus levels after 40 weeks of treatment, while the results of a multi-centre phase 3 study to determine the Total rickets severity score decreased over 40 weeks,s a efficacy and safety of burosumab, an anti-FGF23 did the serum alkaline phosphatase levels. none of the antibody, for treatment of adult XLH. This rare inherited subjects discontinued therapy or developed disorder results from mutations in phEX leading to raised hyperphosphatemia. results demonstrate substantial phosphate excretion, osteomalacia, fractures and dental healing of rickets in children treated with burosumab. complications. The results from one of the pediatric trials have a group of 134 adult Xlh patients ceased their usual already been published. burosumab is now licenced for phosphate and vitamin D supplementation and were treatment of children with Xlh in Europe, and in the usa treated with burosumab or placebo every 4 weeks. after for both children and adults, and may well be life- 24 weeks, the placebo group was transferred also to transforming for people with Xlh. burosumab and the study continued a further 24 weeks. oliver Gardiner, founder of Xlh uK who attended The primary endpoint of reaching the normal range of EcTs 2018, told us “burosumab is life-changing for phosphate levels (lln) was achieved for 94.1 % of patients with Xlh as it is the first and only treatment that subjects in the burosumab group in the first 24 weeks, targets the underlying mechanism of their with benefits continuing during the rest of the study, also hypophosphatemia. The trial results suggest in the crossover group. healing of fractures and improvements in the areas that matter to patients, pseudofractures was more likely with burosumab including reductions in daily pain and stiffness as well as treatment and scoring of pain, stiffness and physical improvements in healing of fractures and rickets severity, functioning all improved. injections were well tolerated which may limit the need for invasive, corrective surgeries. with no drug-related adverse events. We at Xlh uK are currently working closely with the patient community and nicE, as the cost-effectiveness of Also, Wolfgang Högler (Birmingham, UK) presented the burosomab is currently being evaluated with guidance results of two phase 2 pediatric clinical studies of expected for England and Wales in late october 2018.” burosumab. one study was conducted in 52 children with Xlh between the ages of 5 and 12 years with treatment either every two or four weeks, and the other What is new in musculoskeletal research around the world over 17,000 publications on bone-related research appeared in the last year, generating a flood of new data. hans Van leeuwen (Erasmus Medical centre) used his What is New in Basic Science? talk to ask how we can get the most out of this data. firstly, we need to be sure that the data itself is high quality, published with sufficient methodological details to allow the results to be replicated, and meeting the FAIR data principles. hans van leeuwen then highlighted the growing trend for using artificial intelligence (ai) in basic and clinical research, which could help us make better use of the data we generate, and to also fully “digest” the data generated in previous years. one of the most established uses of ai is analysis of massive datasets describing biochemical and genomic profiles. also, ai is being explored for in silico simulation of biological processes, a recently developed example being the D-cell simulation that is using machine learning to compute how gene mutations and other changes could affect cell growth. Despite the growing availability of ai tools, and the appearance already of some clinical applications, none of the abstracts submitted to EcTs 2018 involves such approaches, but we can probably expect to see ai feature much more in the coming years. In other news… look out through the rest of the report for other interesting papers highlighted in our What is New? session by hans van leeuwen (for basic science) and franz Jakob (for clinical science). 2 PROGRESS fOR RaRE diSEaSES aT ECTS ECTS meets with rare disease Moving forward in understanding patient groups and treating fibrodysplasia Ossificans Progressiva ECTS is organising a new initiative to support rare bone disease patient groups in networking and mutual The EcTs 2018 educational symposium on fibrodysplasia support. ossificans progressiva (fop) featured as speakers Eileen To kick off this network, a meeting was organised in Shore (philadelphia), a key contributor to the genetic Valencia, alongside the EcTs 2018 congress, for patient discovery of the disease, and Genevieve Baujat, from the group leaders to discuss their priorities. These included reference centre for skeletal dysplasias in paris, who is representatives of oifE (osteogenesis imperfecta), the involved in the development of the FOP Connection spanish association for camurati-Engelmann patients, Registry. hypophosphat asie Deutschland and the uK and spanish fop is a very rare genetic condition featuring skeletal patient groups for X-linked hypophosphatasia. also dysplasia and heterotopic ossifications. Estimates of participating in the meeting were representatives of prevalence range between 1 in 2 million and 1 in 700,000. EcTs, luca sangiorgi from the European reference fop manifests at birth with a malformation of the big toe. network Ern-bonE, and companies involved in The disease progresses in “flare ups” with bone forming developing new therapies for rare bone diseases. in soft connective tissue, skeletal muscle, tendons and in a breakout discussion session the patient group ligaments in particular. loss of independence typically leaders identified current challenges for their work, occurs by the third decade of life with a high risk of early where they see a new network as being helpful. several death during the fourth decade due to thoracic points were agreed. as many of the groups are for insufficiency syndrome. patients with very rare diseases, small numbers of people are involved in each group, spread out geographically, and this makes organising and membership engagement quite difficult. it would be interesting to share experience of how to handle having a rare disease from a psychological point of view. communication was also raised as a major issue. social media provides many opportunities for misinformation to circulate, and it is also not easy for patients to always understand the latest developments in research – patient groups can play a key role here in providing information.
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  • RECENT MAJOR CHANGES------­ Age
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