Development of an Internationally Agreed Minimal Dataset for Juvenile Dermatomyositis (JDM) for Clinical and Research Use

Liza J. McCann¹, Lucy R Wedderburn², Clarissa Pilkington³, Adam M. Huber⁴, Angelo Ravelli⁵, Jamie Kirkham⁶, Paula Williamson⁶ and Michael W. Beresford⁷, ¹Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom, ²Infection, Inflammation and Rheumatology Section, UCL Institute for Child Health, London, United Kingdom, ³Department of Rheumatology, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom, ⁴Pediatric rheumatology, IWK Health Centre, Halifax, NS, Canada, ⁵Pediatria-II, IRCCS G. Gaslini and University of Genova, Genova, Italy, ⁶Department of Biostatistics, University of Liverpool, MRC North West Hub for Trials Methodology Research, Liverpool, United Kingdom, ⁷Alder Hey Children's NHS Foundation Trust Hospital, Institute of Translational Medicine (Child Health), University of Liverpool, Liverpool, United Kingdom

Meeting: 2015 ACR/ARHP Annual Meeting

Date of first publication: September 29, 2015

Keywords: Clinical, data collection, international and juvenile dermatomyositis, Research

Session Information

Date: Sunday, November 8, 2015

Title: Muscle Biology, Myositis and Myopathies I

Session Type: ACR Concurrent Abstract Session

Session Time: 2:30PM-4:00PM

Background/Purpose: Juvenile dermatomyositis (JDM) is a rare disease. International collaboration is essential for scrutiny of sufficient patient numbers. We aim to develop a core set of data for all clinicians/ researchers to collect in a standardized way, with international agreement.

Methods: Comparing variables in existing clinical registries led to a provisional minimal dataset.¹A two-stage e-mail Delphi-process engaged the opinion of a large number of key stakeholders via established international paediatric rheumatology/ myositis organizations. Consensus that each outcome should be included in the dataset was defined by ≥70% of participants scoring ‘critical for decision making’. This, together with a formalized patient / parent participation process (ongoing) informed a consensus meeting of internationally representative myositis experts. Nominal group technique was used, with agreement of ≥80% consensus required for each variable to be included. The resulting dataset will be tested for feasibility within existing databases and sent to all internationally representative collaborators for final comment.

Results: Delphi responses from healthcare professionals (n=181 in round 1, 146 in round 2; 12% attrition), patients (n=14) and parents (n=20) were used to inform a consensus meeting of experts. The expert group (n=18) determined the need to include a measure of improvement /worsening of disease as well as binary documentation (feature ‘present / absent’). A visual analogue scale (VAS) was thus included for each major domain. 74 variables were included in the proposed dataset (Table 1 & 2) with further damage items under consideration. ‘Optimal dataset’ replaced the term ‘minimal dataset’. The dataset will be developed further after testing accuracy of data capture in clinical practice and analyzing additional patient/parent questionnaires.

Conclusion: An internationally agreed optimal dataset has the potential to significantly enhance collaboration, provide a standard of care and enable analysis of the largest possible number of JDM patients to provide a greater understanding of this disease. The final approved core dataset could be rapidly incorporated into national/international collaborative efforts.

Table 1: Variables to be collected at first visit only (diagnosis):

Section headings	Variables to be collected
Personal factors (demo-graphics)	Date of birth
	Sex
	Age / date at first symptom of myositis
	Age / date at diagnosis of JDM
Diagnostic factors	MRI scan at diagnosis (if done) consistent with myositis
	Muscle biopsy (if done) consistent with myositis
	Myositis specific antibodies (MSA) positive at diagnosis – if taken
	Myositis associated antibodies (MAA) positive at diagnosis – if taken
Treatments received prior to diagnosis	Has this patient received systemic glucocorticoids prior diagnosis of JDM – yes / no
Treatments received prior to diagnosis	Has this patient received any synthetic or biologic disease modifying anti-rheumatic drug prior to the diagnosis?

Table 2: Variables to be collected at every visit:

Section headings	Variables to be collected (details not shown)
Growth & development	Height, weight, puberty self assessment
Muscular	Symmetrical muscle weakness, Childhood Myositis Assessment Scale (CMAS), Manual Muscle Testing (MMT8)
Cutaneous	Features included in abbreviated Cutaneous Assessment Tool (CAT) score
Skeletal	Arthritis / contractures & Visual Analogue Scale (VAS) for skeletal activity
Gastrointestinal	Dysphagia, abdominal pain, gastrointestinal ulceration & VAS for gastrointestinal
Pulmonary	Pulmonary involvement / interstitial lung disease, dysphonia & VAS for pulmonary activity
Cardiovascular	Cardiovascular involvement, blood pressure & VAS for cardiovascular activity
Constitutional features	Fever, weight loss, fatigue & VAS for constitutional activity
Physician Global	Physician global VAS & extra-muscular activity VAS
Patient / parent outcome	Patient / Parent measure of function & participation
Investigations	Muscle enzymes & other specimens available
Treatment	Drug treatment & exercise routine: range of movement & muscle strength
Outcome / disease impact	Malignancy / death
Damage items (annual)	To include damage items across domains

References: 1. McCann LJ et al. Pediatric Rheumatology 2014. 12: 31

Acknowledgements: We wish to thank all collaborative groups including Euromyositis, JDRG, IMACS, CARRA, PRINTO, PReS, COMET, OMERACT, NIHR Clinical Studies Group consumer representatives, BSPAR Parent Group, NIHR Young Person’s Advisory Group, UK JDM Young Person’s Group, Cure JM, Myositis UK. Funding body: Arthritis Research UK.

Disclosure: L. J. McCann, None; L. R. Wedderburn, None; C. Pilkington, None; A. M. Huber, None; A. Ravelli, None; J. Kirkham, None; P. Williamson, None; M. W. Beresford, None.

To cite this abstract in AMA style:

McCann LJ, Wedderburn LR, Pilkington C, Huber AM, Ravelli A, Kirkham J, Williamson P, Beresford MW. Development of an Internationally Agreed Minimal Dataset for Juvenile Dermatomyositis (JDM) for Clinical and Research Use [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/development-of-an-internationally-agreed-minimal-dataset-for-juvenile-dermatomyositis-jdm-for-clinical-and-research-use/. Accessed .

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