IMPROVEMENT – Improving the outcome in myositis spectrum diseases: core set variables harmonization and use from children to adulthood

Concept

Myositis spectrum disorders (MSDs) inclu- de a wide range of conditions deeply affecting patients’ prognosis and quality of life. Health problems related to MSDs include not only muscle (myositis), but also joints (arthritis/arthralgias), skin (typical cuta- neous lesions) and lungs (Interstitial lung disease).

The timing of onset of different MSDs’findings is generally variable and the risk of a not proper patients’ classification is very high. The myositis expert community recognizes that other steps are necessary for the clarification of different MSD patterns (in both adulthood and childhood), instrumental and laboratory tests to apply and best treatment options.

These steps are mandatory to improve patients’ survival and quality of life, paying special attention to a very vulnerable period for pediatric patients carrying a chronic illness: the transition to an adult age.

Facts and figures

Project lead
L Cavagna
Policlinico S.Matteo Foundation

H Chinoy
University of Manchester
lorenzo.cavagna@unipv.it, hector.chinoy@manchester.ac.uk
FOREUM research grant: € 300.000
2017–2020

Meet the team

H Chinoy
University of Manchester
L Cavagna
Policlinico S.Matteo Foundation
L Wedderburn
University College London
M Gonzalez-Gay
Hospital Universitario Marqués de Valdecilla
U Viora
Associazione Nazionale Malati Reumatici ANMAR

Objectives

To harmonize the international MSDs registries EUMYONET and AENAS with national registries and hospital records; to create a longitudinal database to improve patients’ follow-up, treatment and prognosis.

1. Define and reach consensus among clinicians and patients on a minimum core set of myositis spectrum disease organ-specific measures to address unmet needs, further characterise disease subsets, evolution and treatment, building on existing work in AENEAS and Euromyositis
2. To facilitate collection of longitudinal outcome data by harmonization of national registers
3. To collect existing patient-recorded outcome data more frequently using smartphone applications for aiding patient engagement and clinical practice/research. To use this technology for streamlining the transition between adolescent and adult care in MSDs through harmonized data collection from adult and paediatric clinicians
4. To facilitate standardized data capture from electronic hospital records by technical integration and initiate a common technological platform for the Euromyositis and AENEAS registers

 

Patient voice

Patients are involved in every phase of the project. Participants are invited through myositis centres and through already existing registries for myositis. Associazione Nazionale Malati Reumatici (ANMAR), Italy is involved as a patient organisation.

Interim results

1. A list of applicable measures has been obtained from the systematic literature review and from the analysis of existing datasets addressed to different myositis conditions.  A survey will be send to selected centers/people, in order to understand which variables can be easily collected and for identify potentially lacking aspects.
2. The process of harmonization will start after the definition of clinical variables that should be collected.
3. A bespoke smartphone-based app has been designed that allows collection of myositis-specific patient reported outcome measurements at high frequency (up to daily).

Publications

EULAR Abstracts

2019

  • FR0352: Differences in Antisynthetase Syndrome definition and related diagnostic performance. A systematic literature review informing the new ACR/EULAR classification criteria
  • FR0335: Prognostic impact and clinical characteristics of interstitial pneumonia with autoimmune features in a multidisciplinary setting
  • SAT0271: Relationship between Anti-mda5 antibodies and cancer: retrospective analysis of an international and multidisciplinary cohort
  • SAT0286: Evaluation of swallowing in patients with Idiopathic Inflammatory Myopathies

Go to EULAR Abstract Archive

ACR Abstract

2020

  • Number 1061: Daily Myositis Symptom Changes Collected via a Smartphone-Based App Are Associated with Flare Occurrence – Providing Evidence of Potential Digital Biomarkers
    Read more

Goals/Milestones

Y1: Teleconferences and ethical approvals, data collection harmonization and core set measure to share with other centers/groups, development of technologies, pilot testing of technologies in 12 sites, definition/agreement of core set, submission of LOINC definitions
Y2: Clinical data collection from registers &analysis of shared data.
Y3: Rolling out of technologies in other Research Partner sites, collection of longitudinal smartphone app data, collection of longitudinal clinic based PROM data, research partner meeting of group data shared analysis. Successful implementation of core data into an existing platform, Proof of concept that remote data system can be completed longitudinally