MICA: A UK-wide networking partnership to facilitate collaborative research in the idiopathic inflammatory myopathies

Lead Research Organisation: University of Manchester
Department Name: School of Biological Sciences

Abstract

Objective
To improve the way we treat sufferers with unexplained inflammation in muscle (myositis), by bringing together UK experts, helping research activities, increasing awareness of the disease, improving the way we diagnose and monitor, and improving training opportunities for the UK health-care community.

Background
Unexplained inflammation in muscles is known as "myositis", a condition that affects about 15 in 100,000 people. The muscles affected usually include those around the shoulders and hips, but other parts of the body can be affected. Unlike other muscle diseases, such as muscular dystrophy, the inflammation in myositis is potentially treatable, although the drugs used can be toxic and don't always work. As myositis is uncommon, doctors are often not familiar with recognizing the disease. Delays in diagnosis may lead to irreversible muscle damage, so even after treatment has started, sufferers often remain weak and disabled, leading to significant illness burden on families, carers and society.

The way we diagnose myositis varies widely, which can lead to a wrong diagnosis being made, and use of toxic treatments that may cause harm. Current treatments for myositis are mostly "borrowed" from other inflammatory musculoskeletal diseases. There is no national or international guidance about treatments. We need to improve the care given to myositis sufferers by combining expertise across different medical disciplines. A non-funded organisation, UKMYONET, created by the applicants, aims to share expertise and generate ideas for future research studies and drug trials. An existing UK network working on childhood myositis, the Juvenile Dermatomyositis Research Group, also closely aligns with UKMYONET.

Aim 1: To support current and future myositis research activities and optimize their quality, quantity and success in the UK for years to come. Myositis is rare, meaning that many samples are needed for research and cannot be collected in a single hospital. Instead, collection has to be done by many collaborating muscle experts and hospitals, requiring careful and detailed organisation. The success of this grant application would allow a coordinator to support a coordinated collection of clinical information and blood samples from myositis sufferers, establish UKMYONET meetings as an important national event, and help collaborations with patient-run organizations.

Aim 2: To improve the way we work together to manage myositis better. This grant will help support the collaborating applicants to work together to develop further scientific work aimed at improving the tests that we use to diagnose and monitor myositis. We would like to diagnose myositis earlier with a minimum of delay, tease out the different forms of myositis with ease, and standardize myositis treatment across the UK, with a view to improving future outcomes for patients. The grant will help to support ongoing research activities to make these activities happen, by further developing a study resource of myositis patients.

Aim 3: To improve training opportunities in the field of myositis and the way that sufferers are cared for. We wish to bring different medical experts together, as myositis is truly a multisystem disease: currently these specialists tend to work in "silos", do not attend meetings out of their discipline, may read different literature, and thus have different approaches to treatment. Current funding streams may not attract the spectrum of expertise needed, and exchange of knowledge is poor across disciplines. Health care professionals need more understanding about the variation of how myositis may present, and this can be tackled by improving standards of care and treatment for different types of myositis. Training workshops will be established in national centres of expertise.

Technical Summary

The term myositis refers to a group of rare autoimmune diseases affecting about 10,000 UK patients, the idiopathic inflammatory myopathies (IIM), which cause muscle weakness and disability. It is imperative to bring together IIM expertise and knowledge in the UK to improve infrastructure and coordinate research, clinical trial and training activities. To address this, a clinical and scientific network, UKMYONET, was established by the applicants. Funding for laboratory-based research work on samples recruited through UKMYONET has been available via an Arthritis Research UK programme grant, but no funding supports UKMYONET administrative costs. Similarly, there is no current professional funding to support training opportunities or UKMYONET meetings.

The partnership grant would provide funding otherwise not available by other funding streams. The funds requested will facilitate coordinated UK-wide research activities including genetics, serology, and fundamental cellular mechanisms, and align parallel efforts for adults and children with IIM. Research activities will use datasets including ongoing collection of a core clinical dataset, establishment of a UK-wide adult IIM prospective cohort study, a national treatment outcomes registry to assess the safety and efficacy of novel therapies and establishment of a national myositis biobank. The resource will allow implementation of international research activities to be conducted using UK-based resources and expertise, including validation of new classification criteria and definitions of disease improvement/worsening/remission, and consensus on diagnostic and treatment approaches. Training workshops and visits will allow health care professionals to gain disease-specific experience.

Overall, a more coordinated, collaborative approach and sharing of expertise for IIM research and patient care is anticipated to improve translation of existing/emerging research outputs and knowledge into clinical practice.

Planned Impact

Pharma: Stakeholders in existing and in-development biologic therapies, Servier, BMS and Novartis, have recently collaborated with the applicants to test existing and novel agents in IIM as part of pharma-sponsored clinical trials. Industrial collaborations are already in place to explore regenerative technologies in chronic muscle disease. Several of the applicants are involved in a pivotal phase IIb study to increase muscle mass and endurance in sIBM. Genzyme produce enzyme replacement therapy in lysosomal storage disorders including Pompe disease, a myositis mimicker. Funding for this expensive treatment is commissioned via NHS Specialised Services. It is vital to make an accurate diagnosis for the right patients can benefit, and ensuring that patients with other neuromuscular conditions do not receive unnecessary therapy. Genzyme have sponsored previous UKMYONET meetings and support educational activities organized by the applicants.

The establishment of a novel drug registry with linked biobank is vital in a rare disease where agents are borrowed from other diseases, and not specifically licensed. Longitudinal data will enable safety signals to be monitored over time for adverse or unexpected events. Establishment of high quality data and samples combined with clinical expertise will also facilitate testing of discovery biomarker platforms, to accelerate development and commercialization of future diagnostics.

NHS England: The Department of Health has undergone a consultation exercise on Rare Diseases, and this application is of direct relevance in addressing unmet needs in orphan diseases such as IIM, where improved training opportunities, accurate diagnostics, registers and coordinated care are required. The consultation document acknowledges the need for networks as crucial for rare diseases. Helping to facilitate a biologics registry to monitor safety issues is of paramount importance, and is an obligation in Specialist NHS Commissioning.

Local stakeholders: The proposed grant will run parallel to a national drive to raise awareness of neuromuscular orphan conditions. Salford Royal NHS Foundation Trust is part of the Manchester Academic Health Science Centre, which has committed resource into a quality improvement programme around rare neuromuscular diseases, aiming to accelerate research implementation using improvement science for wider national and international dissemination.

Clinicians: Workshops will bring experts together, e.g. rheumatology, neurology, dermatology, respiratory, pathology, neurophysiology; myositis is a multisystem disease and these specialists tend to not to attend meetings out of their discipline, read different literature, and have different treatment approaches. Current funding streams may not attract the spectrum of expertise needed. An appreciation of myositis heterogeneity is required and how to recognize disease early. The network will foster UKMYONET and establish a platform for future UK myositis trial work. Cohort studies will help develop better diagnostics and antibody/other biomarker monitoring, and create standardized treatment protocols to improve standards of care for different types of myositis.

Patients: Patients and relatives will, as "end-users", benefit from anticipated improvement of quality of clinical care, and future development of new and more effective treatments for IIM, resulting in improved prognosis and quality of life. Patients will engage as part of a research user group and have input into future research priorities and academic activities.

Charities: UKMYONET already has close links with the Myositis UK patient self help support group and The Myositis Association (USA). Results and scientific output will be disseminated through the websites of these organizations. The grant will result in useful output for patients, relatives and other end-users, for example web content and information leaflets.

Timescales: Refer to pathways to impact.

Publications

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publication icon
Bellutti Enders F (2017) Consensus-based recommendations for the management of juvenile dermatomyositis. in Annals of the rheumatic diseases

 
Description Member of Scientific Programme committee GCOM19
Geographic Reach Multiple continents/international 
Policy Influence Type Influenced training of practitioners or researchers
Impact Global Conference in Myositis is now thew leading meeting in the field taking place every 2 years
URL https://www.gcom2019.org
 
Description Specialist Rheumatology Commissioning for NHS England
Geographic Reach National 
Policy Influence Type Participation in a advisory committee
Impact Better access to biologic drugs for patients with myositis. Physicians wishing to put patients on such drugs no longer have to undergo the laborious process of completing individual funding requests. There is now an NHS England policy, which will partly be audited with the aid of the MYOPROSP infrastructure. Dr Chinoy is now the RCP representative for Specialised Commissioning in Rheumatology, and will help to report back findings to NHS England, and shape future policy.
URL https://www.england.nhs.uk/commissioning/wp-content/uploads/sites/12/2013/04/16036_FINAL.pdf
 
Description Arthritis Research UK Centre for Adolescent Rheumatology
Amount £2,113,790 (GBP)
Organisation Versus Arthritis 
Start 04/2018 
End 03/2023
 
Description CURE JM Foundation research grant
Amount $50,000 (USD)
Organisation Cure JM Foundation 
Sector Charity/Non Profit
Country United States
Start 05/2016 
End 05/2017
 
Description FOREUM Registry Funding
Amount € 300,000 (EUR)
Organisation The European League Against Rheumatism (EULAR) 
Sector Charity/Non Profit
Country Switzerland
Start 04/2017 
End 03/2020
 
Description Great Ormond Street Biomedical Research Centre
Amount £37,005,790 (GBP)
Organisation University of Leicester 
Department NIHR Biomedical Research Centre
Sector Hospitals
Country United Kingdom
Start 04/2017 
End 03/2022
 
Description High Cellular Phenotyping of Myositis Patients Starting Rituximab Therapy
Amount $100,000 (USD)
Organisation The Myositis Association (TMA) 
Sector Charity/Non Profit
Country Unknown
Start 01/2018 
End 08/2019
 
Description Identification of Signatures of Microbial Infection in Idiopathic Inflammatory Myopathies
Amount $90,000 (USD)
Organisation The Myositis Association (TMA) 
Sector Charity/Non Profit
Country Unknown
Start 04/2017 
End 03/2018
 
Description Identification of type 1 interferon related transcripts and known pathways in adult idiopathic inflammatory myopathy
Amount $350,000 (USD)
Organisation MedImmune 
Sector Private
Country United States
Start 08/2016 
End 01/2020
 
Description Immune Cell Profiling of Myositis Patients Starting Rituximab Therapy
Amount £19,800 (GBP)
Organisation Myositis UK 
Sector Charity/Non Profit
Country United Kingdom
Start 01/2018 
End 12/2019
 
Description NIHR Senior Investigator Award
Amount £60,000 (GBP)
Organisation National Institute for Health Research 
Sector Public
Country United Kingdom
Start 04/2017 
End 03/2021
 
Description The Myositis Association (TMA)
Amount £57,122 (GBP)
Organisation The Myositis Association (TMA) 
Sector Charity/Non Profit
Country Unknown
Start 01/2016 
End 12/2017
 
Title The JDM muscle biopsy score tool 
Description Report 2016 2016 We have now assessed 127 JDM muscle biopsies and continue to grow the data analysis from 2015. REPORT 2015 2015 Since developing the JDM Biopsy score tool, and through support from this award we have now assessed a large set of biopsies form different cases of JDM (n=101) We have carried out two large analyses using the resulting data on this large set, by integrating the histology data with myositis specific autoantibody (MSA) and detailed longitudinal clinical data: and has led to several new notable impacts see below. REPORT 2014 we generated a quantitative assessment tool for JDM muscle biopsy, now in routine clinical use at GOSH (Wedderburn et al ), and shown that this tool correlates with disease activity, in particular the inflammatory and muscle fibre domains [Varsani, H., Charman S. C. Li, C.K., Marie, S.K., Amato, A.A. Banwell, B., Bove, K.E., Corse, A.M., Emslie-Smith, A.M., Jacques, T.S., Lundberg, I.E., Minetti, C., Nennesmo, I., Rushing, E.J., Sallum, A.M.E., Sewry, C., Pilkington, C.A., Holton, J.L., Wedderburn L.R and the UK JDRG. (2015). Validation of a score tool for measurement of histological severity in juvenile dermatomyositis and association with clinical severity of disease. Annals Rheum Dis 74(1):204-10]. 
Type Of Material Model of mechanisms or symptoms - human 
Year Produced 2013 
Provided To Others? Yes  
Impact Report 2016 This work resulted in the paper from Deakin CT, et al Muscle biopsy in combination with myositis-specific autoantibodies aids prediction of outcomes in juvenile dermatomyositis, Arthritis Rheumatol. 2016 May 23. doi: 10.1002/art.39753 which had significant international and local reactions in being featured as highlighted research in Nature Reviews Rheumatology, ICH bulletins and GOSH BRC research newsletters. The biposy score tool has also been used by other researchers across Europe for published research, showing the successful adoption of this tool. There are future publications in preparation from our lab on this tool. REPORT 2015 1. By correlating the histology data with autoantibody status (ie the particular myositis specific autoantibody found in each case) we have seen that the histology seen in muscle biopsy varies according to MSA type for some, but not all MSA types. 2. We have carried out a complex model using a statistical method called generalising estimating equations (GEE) which takes into account longitudinal clinical data over time. Using this to analysis the prognostic value of biopsy and MSA data, we have shown that the histology data is predictive of clinical treatment status over time. This very exciting discovery means that information in the biopsy may help doctors to advise families about the chance of their child going into drug free remission, or not 3. this work has led to significant new funding including further funding from the NIHR Translational Research Collaboration (TRC) for rare diseases, a prestigious fellowship from The Myositis Association USA , to Dr C Deakin and award from Myositis UK. REPORT 2014 1. The tool is now widely in use for assessment of JDM biopsy in clinical practice. 2. the tool has led to considerable extra funding to take the work forward 
URL https://www.juveniledermatomyositis.org.uk/Research.html
 
Title Euromyositis Registry 
Description This database has been created in order to obtain uniform, longitudinal data over adult and juvenile myositis cases in order to achieve increased knowledge on disease course and prognosis of myositis. In addition, this registry could be used as a tool in the clinic to assess your patients. The Euromyositis Registry was developed within the integrated European FP6 Autocure project and led to a creation of the basic database. Starting from summer 2010, further development and support for 5 years was through the European Science Foundation Research Networking Programme EuMyoNet (European Myositis Network). 
Type Of Material Database/Collection of data 
Year Produced 2017 
Provided To Others? Yes  
Impact Recent publication on the registry, https://www.ncbi.nlm.nih.gov/pubmed/28855174 Recent conference in Prague devoted to the Registry 
URL https://euromyositis.eu/
 
Title Juvenile Dermatomyositis Cohort and Biomarker Study Database 
Description The Juvenile Dermatomyositis Cohort and Biomarker Study database stores longitudinal prospective clinical data from patients with Juvenile Dermatomyositis and other idiopathic inflammatory myopathies (As at May 2018 n=580) linked to a research sample biobank. Data is collected and stored from 17 centres across the UK through standardised proformas in accordance with the study GANTT chart. The database holds data including but not limited to basic demographics, diagnosis, skin and muscle assessments, treatments, laboratory results and sub-phenotypes of JDM. There are three new centres across the UK about to start recruitment to this study. In October 2016 a new website and database was launched that was developed with the input from researchers, patients and their families to best reflect their priorities and requests for information. 
Type Of Material Database/Collection of data 
Provided To Others? Yes  
Impact 1. Improved care for patients with JDM in the UK and beyond 2. Developed new biomarkers (Ab) 3. Developed tool to assess and score JDM biopsies 4. Improved patient and parent involvement in research. 5. Generated evidence for new treatments and care pathways. 6. Established JDM as a key priority in the Clinical Study Group ( MCRN/ARUK funded) for Paediatric Rheumatology across the UK 
URL https://www.juveniledermatomyositis.org.uk/Index.html
 
Description Biopsy prediction of long term treatment status 
Organisation Great Ormond Street Hospital (GOSH)
Country United Kingdom 
Sector Hospitals 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Dr T. Jacques). Dr S Yasin performed staining and scoring of biopsy samples. Ms Simou contributed to data collection and data cleaning. Dr C Deakin performed data analysis and statistical modelling. Drs Pilkington and Nistala contributed to data collection. Dr Jacques processed the biopsies.
Collaborator Contribution Dr S Tansley and Dr Z Betteridge performed serology analysis (N McHugh laboratory, Bath).
Impact Paper published Deakin CT et al, 2016, "Muscle biopsy in combination with myositis-specific autoantibodies aids prediction of outcomes in juvenile dermatomyositis, Arthritis Rheumatol. 2016 May 23. doi: 10.1002/art.39753. This paper was featured in Research Highlights in Nature Reviews Rheumatology, (2016) 12 374-5, in the ICH Director's Bulletin as "paper of the month" in June 2016, and the GOSH BRC newsletter in September 2016. It was also featured in the opening presentation for the Paediatric Rhuematology European Society Congress in Genoa, Italy in September 2016. Oral abstracts presentations at meetings: 1. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015 2. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Wedderburn LR, "An integrative analytical approach to sub-phenotyping of juvenile dermatomyositis", British Rheumatology Society meeting, Manchester, 28-30 April 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Sub-phenotyping of juvenile dermatomyositis", British Neuropathology Society meeting, London, 4-6 March 2015 Poster presentations at meetings: 1. Shireena A Yasin, Peter W Schutz, Claire T Deakin, Erdal Sag, HemlataVarsani, Stephanie Simou, Sarah Tansley, Neil McHugh, Janice L Holton, Lucy R Wedderburn, Thomas S Jacques, on behalf of the UK Juvenile Dermatomyositis Research Group, Biopsy pathology in a large cohort of juvenile dermatomyositis is heterogeneous and, for the most part, independent of autoantibody phenotype, annual meeting of the Canadian Association of Neuropathologists 2016. 2. Deakin CT, Yasin SA, Simou S, Tansley SL, Betteridge ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington CA, Wedderburn LR, "Associations between biomarkers of juvenile dermatomyositis and long-term markers", Research Symposium for the National Institute for Health Research Rare Diseases Translational Research Collaboration, 15 September 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015
Start Year 2014
 
Description Biopsy prediction of long term treatment status 
Organisation University College London
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Dr T. Jacques). Dr S Yasin performed staining and scoring of biopsy samples. Ms Simou contributed to data collection and data cleaning. Dr C Deakin performed data analysis and statistical modelling. Drs Pilkington and Nistala contributed to data collection. Dr Jacques processed the biopsies.
Collaborator Contribution Dr S Tansley and Dr Z Betteridge performed serology analysis (N McHugh laboratory, Bath).
Impact Paper published Deakin CT et al, 2016, "Muscle biopsy in combination with myositis-specific autoantibodies aids prediction of outcomes in juvenile dermatomyositis, Arthritis Rheumatol. 2016 May 23. doi: 10.1002/art.39753. This paper was featured in Research Highlights in Nature Reviews Rheumatology, (2016) 12 374-5, in the ICH Director's Bulletin as "paper of the month" in June 2016, and the GOSH BRC newsletter in September 2016. It was also featured in the opening presentation for the Paediatric Rhuematology European Society Congress in Genoa, Italy in September 2016. Oral abstracts presentations at meetings: 1. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015 2. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Wedderburn LR, "An integrative analytical approach to sub-phenotyping of juvenile dermatomyositis", British Rheumatology Society meeting, Manchester, 28-30 April 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Sub-phenotyping of juvenile dermatomyositis", British Neuropathology Society meeting, London, 4-6 March 2015 Poster presentations at meetings: 1. Shireena A Yasin, Peter W Schutz, Claire T Deakin, Erdal Sag, HemlataVarsani, Stephanie Simou, Sarah Tansley, Neil McHugh, Janice L Holton, Lucy R Wedderburn, Thomas S Jacques, on behalf of the UK Juvenile Dermatomyositis Research Group, Biopsy pathology in a large cohort of juvenile dermatomyositis is heterogeneous and, for the most part, independent of autoantibody phenotype, annual meeting of the Canadian Association of Neuropathologists 2016. 2. Deakin CT, Yasin SA, Simou S, Tansley SL, Betteridge ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington CA, Wedderburn LR, "Associations between biomarkers of juvenile dermatomyositis and long-term markers", Research Symposium for the National Institute for Health Research Rare Diseases Translational Research Collaboration, 15 September 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015
Start Year 2014
 
Description Biopsy prediction of long term treatment status 
Organisation University of Bath
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Dr T. Jacques). Dr S Yasin performed staining and scoring of biopsy samples. Ms Simou contributed to data collection and data cleaning. Dr C Deakin performed data analysis and statistical modelling. Drs Pilkington and Nistala contributed to data collection. Dr Jacques processed the biopsies.
Collaborator Contribution Dr S Tansley and Dr Z Betteridge performed serology analysis (N McHugh laboratory, Bath).
Impact Paper published Deakin CT et al, 2016, "Muscle biopsy in combination with myositis-specific autoantibodies aids prediction of outcomes in juvenile dermatomyositis, Arthritis Rheumatol. 2016 May 23. doi: 10.1002/art.39753. This paper was featured in Research Highlights in Nature Reviews Rheumatology, (2016) 12 374-5, in the ICH Director's Bulletin as "paper of the month" in June 2016, and the GOSH BRC newsletter in September 2016. It was also featured in the opening presentation for the Paediatric Rhuematology European Society Congress in Genoa, Italy in September 2016. Oral abstracts presentations at meetings: 1. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015 2. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Wedderburn LR, "An integrative analytical approach to sub-phenotyping of juvenile dermatomyositis", British Rheumatology Society meeting, Manchester, 28-30 April 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Sub-phenotyping of juvenile dermatomyositis", British Neuropathology Society meeting, London, 4-6 March 2015 Poster presentations at meetings: 1. Shireena A Yasin, Peter W Schutz, Claire T Deakin, Erdal Sag, HemlataVarsani, Stephanie Simou, Sarah Tansley, Neil McHugh, Janice L Holton, Lucy R Wedderburn, Thomas S Jacques, on behalf of the UK Juvenile Dermatomyositis Research Group, Biopsy pathology in a large cohort of juvenile dermatomyositis is heterogeneous and, for the most part, independent of autoantibody phenotype, annual meeting of the Canadian Association of Neuropathologists 2016. 2. Deakin CT, Yasin SA, Simou S, Tansley SL, Betteridge ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington CA, Wedderburn LR, "Associations between biomarkers of juvenile dermatomyositis and long-term markers", Research Symposium for the National Institute for Health Research Rare Diseases Translational Research Collaboration, 15 September 2015 3. Deakin CT, Yasin SA, Arnold KA, Tansley SL, Betteridge, ZE, McHugh NJ, Holton JL, Jacques TS, Pilkington C, De Iorio M, Wedderburn LR, "Biomarkers for sub-phenotyping of juvenile dermatomyositis", 1st International Myositis Meeting, Stockholm, 8-11 May 2015
Start Year 2014
 
Description EUMYONET 
Organisation European Commission
Department EC FP6 Collaborative Projects
Country European Union (EU) 
Sector Academic/University 
PI Contribution A main aim of EUMYONET is to contribute capacity to our EU/UK efforts to correlate myositis genotype with myositis serotype and phenosubtype. The University of Manchester was designated as the genotyping centre for the UK/EUMYONET collaboration, and as a result we have collectively recruited 1800 EU/UK myositis cases to date. These samples are being genotyped in CIGMR in the University of Manchester. The immunoprecipitaion laboratory in the University of Bath is the designated serotyping centre
Collaborator Contribution All of the senior Manchster based "myositis team" were co-founders of EUMYONET, and have encouraged recruitment of cases both in the UK and the EU. The genotyping laboratory staff in CIGMR are coordinating all aspects of DNA testing and analysis of the EU/UK samples, and their correlation with the serotyping results from the University of Bath, which is serotyping all 1800 EU/UK adult samples, mas well as the 350 UK juvenile samples.
Impact New antibodies have been discovered, and a possible interaction between smoking with certain HLA class II genes. The latter was only possible because of the capacity derived from this EU/UK collaboration, which is multidisciplinary, as per the UK MYONET. A new classification criteria has been developed in IIM as a result of this consortium.
Start Year 2008
 
Description JDM International Core Data Set Project 
Organisation Alder Hey Children's NHS Foundation Trust
Country United Kingdom 
Sector Hospitals 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation Childhood Arthritis and Rheumatology Research Alliance (CARRA)
Country United States 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation European Commission
Department EC FP6 Collaborative Projects
Country European Union (EU) 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation Great Ormond Street Hospital (GOSH)
Country United Kingdom 
Sector Hospitals 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation Imaxio SA
Country France 
Sector Private 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation MyoNet
Country Global 
Sector Charity/Non Profit 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation Pediatric Rheumatology INternational Trials Organisation (PRINTO)
Country Italy 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation University College London
Department Institute of Child Health
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description JDM International Core Data Set Project 
Organisation University of Liverpool
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team consists of Dr S Yasin , Dr Deakin, Ms S Simou (Study coordinator), Prof L Wedderburn (PI) as well as the clinical team at GOSH Rheumatology (Dr Pilkington, Dr Nistala, Mrs Y. Glackin). The ICH team has contributed in the design of the project, participants' recruitment and data collection, participation in the consensus meeting, development of Delphi questionnaire
Collaborator Contribution The partners have contributed in the design of the project, participation in the consensus meeting, development of Delphi questionnaire.
Impact 2016: Since previous reporting the International minimal core data set for JDM in clinical use and research/trials, has been agreed on. This includes input from patient and parent groups. The resulting form is ready to be piloted and this will go ahead very soon in two large clinical centres in the UK. Previous work: A proposed International minimal core data set for JDM in clinical use and research/trials, has been generated and will now be tested. Publications: - McCann, L.J., Kirkham, J.J., Wedderburn, L.R., Pilkington, C.A., Huber, A.M., Ravelli, A., Appelbe, D., Williamson, P.R., Beresford, M.W. (2015). Development of an internationally agreed minimal dataset for Juvenile Dermatomyositis (JDM) for clinical and research use. Trials Jun 12; 16(1):268. - McCann, L.J., Arnold, K., Pilkington, C.A., Huber, A.M., Ravelli, A., Beard, L., Beresford, M.W., Wedderburn, L.R. (2014). Developing a provisional, international Minimal Dataset for Juvenile Dermatomyositis: for use in clinical practice to inform research. Pediatric Rheumatology. 12:31 -42. Poster presentations at meetings: - McCann, L.J., Wedderburn, L.R., Pilkington, C.A., M Huber, A.M., Ravelli, A., Williamson, P., Kirkham, J., Beresford, M.W., Development of an internationally agreed minimal dataset for juvenile dermatomyositis (jdm) for clinical and research use, 1st International Myositis Meeting, Stockholm, May 2015. - McCann, L.J., Pilkington, C.A., Beard, L., Ravelli, A., Huber, A.M., Wedderburn, L.R., Proposal for the development of an international minimal data set collection for Juvenile Dermatomyositis (JDM), PReS 2011.
Start Year 2014
 
Description MYOPROSP 
Organisation Great Ormond Street Hospital (GOSH)
Country United Kingdom 
Sector Hospitals 
PI Contribution MYOPROSP is a prospective observational study aimed at collecting incident cases of myositis. Our team has been responsible for setting up the study, getting ethics approved and getting approval in over 20 centres within England
Collaborator Contribution The partners in the collaboration have contributed towards helping to refine the study protocol andrecruiting participants into the study
Impact Over 80 participants have been recruited into MYOPROSP. Investigators include those from both Rheumatology and Neurology, as well as adult and paediatric medicine
Start Year 2016
 
Description MYOPROSP 
Organisation Kings Health Partners
PI Contribution MYOPROSP is a prospective observational study aimed at collecting incident cases of myositis. Our team has been responsible for setting up the study, getting ethics approved and getting approval in over 20 centres within England
Collaborator Contribution The partners in the collaboration have contributed towards helping to refine the study protocol andrecruiting participants into the study
Impact Over 80 participants have been recruited into MYOPROSP. Investigators include those from both Rheumatology and Neurology, as well as adult and paediatric medicine
Start Year 2016
 
Description MYOPROSP 
Organisation MedImmune
Country United States 
Sector Private 
PI Contribution MYOPROSP is a prospective observational study aimed at collecting incident cases of myositis. Our team has been responsible for setting up the study, getting ethics approved and getting approval in over 20 centres within England
Collaborator Contribution The partners in the collaboration have contributed towards helping to refine the study protocol andrecruiting participants into the study
Impact Over 80 participants have been recruited into MYOPROSP. Investigators include those from both Rheumatology and Neurology, as well as adult and paediatric medicine
Start Year 2016
 
Description MYOPROSP 
Organisation UCL Partners Ltd
Department UCL Partners AHSC and AHSN
Country United Kingdom 
Sector Academic/University 
PI Contribution MYOPROSP is a prospective observational study aimed at collecting incident cases of myositis. Our team has been responsible for setting up the study, getting ethics approved and getting approval in over 20 centres within England
Collaborator Contribution The partners in the collaboration have contributed towards helping to refine the study protocol andrecruiting participants into the study
Impact Over 80 participants have been recruited into MYOPROSP. Investigators include those from both Rheumatology and Neurology, as well as adult and paediatric medicine
Start Year 2016
 
Description MYOPROSP 
Organisation University of Bath
Country United Kingdom 
Sector Academic/University 
PI Contribution MYOPROSP is a prospective observational study aimed at collecting incident cases of myositis. Our team has been responsible for setting up the study, getting ethics approved and getting approval in over 20 centres within England
Collaborator Contribution The partners in the collaboration have contributed towards helping to refine the study protocol andrecruiting participants into the study
Impact Over 80 participants have been recruited into MYOPROSP. Investigators include those from both Rheumatology and Neurology, as well as adult and paediatric medicine
Start Year 2016
 
Description Subphenotyping of JDM 
Organisation University of Bath
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team on this project consists of Professor L Wedderburn (PI), Ms K Arnold (Study Coordinator), Dr C Pilkington, Dr K Nistala, Dr R Marques, Dr T Jacques and recently joined Dr C Deakin. We have defined clear clinical types of JDM that are distinct and have separate associations with the new biomarkers, autoantibodies.
Collaborator Contribution The histology analysis is also supported by advice from Prof J Holton Institute of Neurology UCL The team in Bath led by Professor N McHugh leads all of the autoantibody analysis. The team in Manchester is led by Dr H Chinoy for the genetic analysis.
Impact This collaboration has led to the clear demonstration that different types of JDM have distinct associations with different autoantibody biomarkers that can be readily measured in a small 1 ml sample of blood from a child. Publications: (see full details of each under publications) Deakin, C, et al 2016 Arthritis Rheumatology, May 23 doi: 10.1002/art.39753 Tansley, S, et al, 2014 Arthritis Research Therapy. 16(4) Tansley, S, et al, 2014 Rheumatology Jul 1. doi: 10.1093/rheumatology/keu259 Chinoy, H, et al, 2014 Rheumatology. 51(5):794-9 Jani, M, et al, 2014 Annals Rheum Dis. doi: 10.1136/annrheumdis-2014-205440. [Epub ahead of print] Miller, F.W, et al, 2013 Arthritis Rheum. 2013 Dec;65(12):3239-47. Gunawardena, H, et al, 2009 Arthritis and Rheumatism 60(6), 1807-1814 Chinoy, H, et al, 2009 Rheumatology (Oxford) 48(10), 1213-1217. Chinoy, H, et al, 2008 Arthritis & Rheumatism 58, 3247-325. Gunawardena H, et al, 2008 Rheumatology 47, 324 - 328. Wedderburn, L.R, et al, 2007 Rheumatology 46, 1786-1791.
Start Year 2006
 
Description Subphenotyping of JDM 
Organisation University of Manchester
Country United Kingdom 
Sector Academic/University 
PI Contribution The ICH/GOSH team on this project consists of Professor L Wedderburn (PI), Ms K Arnold (Study Coordinator), Dr C Pilkington, Dr K Nistala, Dr R Marques, Dr T Jacques and recently joined Dr C Deakin. We have defined clear clinical types of JDM that are distinct and have separate associations with the new biomarkers, autoantibodies.
Collaborator Contribution The histology analysis is also supported by advice from Prof J Holton Institute of Neurology UCL The team in Bath led by Professor N McHugh leads all of the autoantibody analysis. The team in Manchester is led by Dr H Chinoy for the genetic analysis.
Impact This collaboration has led to the clear demonstration that different types of JDM have distinct associations with different autoantibody biomarkers that can be readily measured in a small 1 ml sample of blood from a child. Publications: (see full details of each under publications) Deakin, C, et al 2016 Arthritis Rheumatology, May 23 doi: 10.1002/art.39753 Tansley, S, et al, 2014 Arthritis Research Therapy. 16(4) Tansley, S, et al, 2014 Rheumatology Jul 1. doi: 10.1093/rheumatology/keu259 Chinoy, H, et al, 2014 Rheumatology. 51(5):794-9 Jani, M, et al, 2014 Annals Rheum Dis. doi: 10.1136/annrheumdis-2014-205440. [Epub ahead of print] Miller, F.W, et al, 2013 Arthritis Rheum. 2013 Dec;65(12):3239-47. Gunawardena, H, et al, 2009 Arthritis and Rheumatism 60(6), 1807-1814 Chinoy, H, et al, 2009 Rheumatology (Oxford) 48(10), 1213-1217. Chinoy, H, et al, 2008 Arthritis & Rheumatism 58, 3247-325. Gunawardena H, et al, 2008 Rheumatology 47, 324 - 328. Wedderburn, L.R, et al, 2007 Rheumatology 46, 1786-1791.
Start Year 2006
 
Description UK Adult Onset Myositis Immunogenetic Collaboration (AOMIC), later called UK MYONET 
Organisation UK MyoNet
Country United Kingdom 
Sector Academic/University 
PI Contribution Collection of 1,600 samples and data from patients with myositis UK-wide UKCRN supported
Collaborator Contribution Coordinating the collaboration and providing samples
Impact Led to consultancy, co-Publications
 
Description Barbra Ansell Address Key points Youtube video 
Form Of Engagement Activity Engagement focused website, blog or social media channel
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Media (as a channel to the public)
Results and Impact As part of the Barbra Ansell inaugural address at BSAPR 2017 Lucy was asked to summarise the key points which was put as video on Youtube for continual dissemination. This means those who could not attend the meeting were able to hear what was said.
Year(s) Of Engagement Activity 2017
URL https://www.youtube.com/watch?v=lccuKBoAmAY
 
Description British Myology Society UK - Invited Speaker 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact British Myology Society UK invited speaker: "Juvenile dermatomyositis : one entity or many"
Year(s) Of Engagement Activity 2016
 
Description Co led a JDM Young peoples focus group held at GOS ICH/GOSH 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Patients, carers and/or patient groups
Results and Impact This was a focus group that regularly meets of patients with JDM. We held general discussion on their involvement in research projects, asked the opinions of research proposals and this also sparked questions from them which were answered. This group also acts as a support network.
Year(s) Of Engagement Activity 2016
 
Description EUROMYOSITIS international meeting, Manchester UK Integration of IIM subsets: juvenile dermatomyositis 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact A talk was given on Integration subsets for JDM.
Year(s) Of Engagement Activity 2016
 
Description Invited talk - Turkish Rheumatology Association 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact About 300 delegates from throughout Turkey attended this conference. I made plans with the organiser to extend our collaboration into Turkey as a result - they will make an application to join the Euromyositis Registry
Year(s) Of Engagement Activity 2018
URL http://www.hedef2018.org
 
Description Key Note speaker American College of Rheumatology 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Lucy was the invited speaker to give the Key note lecture for the year in review, updating all attendees on the newest developments in translational research as it applies to pediatric rehumatology.
Year(s) Of Engagement Activity 2017
 
Description NIHR TRC Rare Disease meeting, invited speaker 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Industry/Business
Results and Impact A talk was given on Childhood myositis: what do patients and families want or need and how can we achieve it? A discussion was had after and questions were answered.
Year(s) Of Engagement Activity 2016
 
Description Oral Presentation First Barbara Ansell Address 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Invited speaker to give the first Barbara Ansell Address at British society of Rheumatology annual general meeting at The Jewels in the crown session. Attended by clinicians and researchers.
Year(s) Of Engagement Activity 2017
 
Description Plenary talk: What is new in the Treatment of Myositis? 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact European League Against Rheumatism 2018, "WIN with EULAR" session, Amsterdam (2,500 in audience)
Year(s) Of Engagement Activity 2018
 
Description Talk to GOSCC charity funders and major donors 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Supporters
Results and Impact Talked to GOSH Children's Charity and their Major donors on Subtypes of juvenile dermatomyositis and how to speed up improvements in care for this disease.
Year(s) Of Engagement Activity 2016