Abstract
ObjectivesUncertainty around clinical heterogeneity and outcomes for patients with JDM represents a major burden of disease and a challenge for clinical management. We sought to identify novel classes of patients having similar temporal patterns in disease activity and relate them to baseline clinical features.MethodsData were obtained for n = 519 patients, including baseline demographic and clinical features, baseline and follow-up records of physician’s global assessment of disease (PGA), and skin disease activity (modified DAS). Growth mixture models (GMMs) were fitted to identify classes of patients with similar trajectories of these variables. Baseline predictors of class membership were identified using Lasso regression.ResultsGMM analysis of PGA identified two classes of patients. Patients in class 1 (89%) tended to improve, while patients in class 2 (11%) had more persistent disease. Lasso regression identified abnormal respiration, lipodystrophy and time since diagnosis as baseline predictors of class 2 membership, with estimated odds ratios, controlling for the other two variables, of 1.91 for presence of abnormal respiration, 1.92 for lipodystrophy and 1.32 for time since diagnosis. GMM analysis of modified DAS identified three classes of patients. Patients in classes 1 (16%) and 2 (12%) had higher levels of modified DAS at diagnosis that improved or remained high, respectively. Patients in class 3 (72%) began with lower DAS levels that improved more quickly. Higher proportions of patients in PGA class 2 were in DAS class 2 (19%, compared with 16 and 10%).ConclusionGMM analysis identified novel JDM phenotypes based on longitudinal PGA and modified DAS.
Highlights
Uncertainty around medium- to long-term outcomes is a major burden of disease for patients and families, as highlighted by a recent qualitative study of children and young people with JDM [1]
In addition to characteristic features of proximal muscle weakness and pathognomonic skin rash, many patients develop a heterogeneous range of additional features, including calcinosis, lipodystrophy, treatment-resistant rash and internal organ involvement
All patients were recruited to the UK-wide JDM Cohort and Biomarker Study (JDCBS) [17], with ethical approval (UK Northern and Yorkshire Medical Research and Ethics Committee MREC/1/3/22)
Summary
Uncertainty around medium- to long-term outcomes is a major burden of disease for patients and families, as highlighted by a recent qualitative study of children and young people with JDM [1]. Patients can have heterogeneous responses to treatment, which poses a challenge for clinical management. Longer-term cross-sectional follow-up studies have indicated that some patients have ongoing disease activity and damage [7,8,9,10,11,12,13,14], underscoring the need to collect and understand data on long-term outcomes in JDM beyond the first 2 years. An example is a recent study that applied longitudinal methods to understand disease evolution using data from 95 patients at The Hospital for Sick Children, Toronto, Canada [15]
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