Probability of Response in the First Sixteen Weeks After Starting Biologics: An Analysis of Juvenile Idiopathic Arthritis Biologics Trials.

Objectives: Most juvenile idiopathic arthritis (JIA) biologic disease-modifying antirheumatic drugs (bDMARDs) trials used an open-label run-in period followed by randomized medication withdrawal. We used data from the run-in period of 4 bDMARD trials to 1) delineate early response trajectory to bDMARDs and 2) identify predictors of early response.

Methods: Data from the first 16 weeks of 4 bDMARD trials were used.

Latent classes of early response trajectories to biologics initiation in juvenile idiopathic arthritis: an analysis of four trials.

Aims: 1) To delineate latent classes of treatment response to biologics in juvenile idiopathic arthritis (JIA) patients in the first 16 weeks after initiation. 2) To identify predictors of early disease response.

Methods: The study population was drawn from four biologics trials in polyarticular course JIA: Etanercept 2000, Abatacept 2008, TRial of Early Aggressive Therapy (TREAT) 2012 and Tocilizumab 2014.

Joint trajectories of disease activity, and physical and mental health-related quality of life in an inception lupus cohort.

Objectives: To examine for latent patterns of SLE disease activity trajectories that associate with specific latent patterns of health-related quality of life (HRQoL; Medical Outcomes Study Short Form-36), and to determine baseline predictors of class membership.

Methods: In this retrospective longitudinal inception cohort of 222 SLE adults over 10 years, trajectories of three outcomes were studied jointly: Short Form-36 physical (PCS) and mental (MCS) component summaries and adjusted mean SLEDAI-2000 (AMS). Group-based joint trajectory modelling was used to model latent classes; univariable and multivariable analyses were used to identify predictors of class membership.

Using Registry Data to Understand Disease Evolution in Inflammatory Myositis and Other Rheumatic Diseases.

Purpose Of Review: While rheumatic disease registries collect longitudinal patient information, longitudinal analytic methods are usually not applied to these data. This review will showcase advances in longitudinal designs/analyses, and ways to leverage digital technologies to recruit and retain more registry participants.

Recent Findings: We will show how the accelerated cohort and longitudinal multiform methods are more efficient than traditional longitudinal designs.

Real-World Effectiveness of Common Treatment Strategies for Juvenile Idiopathic Arthritis: Results From a Canadian Cohort.

Objective: Undervaluing the effectiveness of conventional treatments may lead to overtreatment with biologic medications in children with juvenile idiopathic arthritis (JIA). Using data from a nationwide inception cohort and strict methods to control bias, the aim of our study was to estimate the real-world effectiveness of simple JIA treatment strategies recommended in current guidelines.

Methods: Children with JIA who were recruited at 16 Canadian centers from 2005 to 2010 were followed for up to 5 years.

Alternative Design and Analytical Techniques for Longitudinal Rheumatology Studies: Improved Understanding of Outcomes.

Longitudinal cohort designs (with three or more measurement occasions) are invaluable to investigate between- and within-individual variation in outcomes. However, traditional longitudinal designs require a lengthy implementation and data collection period and impose a substantial burden on participants and investigators. We discuss alternative longitudinal designs, including planned missing data designs and retrospective cohort studies with secondary data, which require a shorter period for data accrual and reduce participant burden while maintaining statistical power.

From Childhood to Adulthood: Disease Activity Trajectories in Childhood-Onset Systemic Lupus Erythematosus.

Objective: No previous study has studied the longitudinal disease course of childhood-onset systemic lupus erythematosus (cSLE). Our objectives are to assess distinguishable differences in disease activity trajectories in cSLE patients, determine baseline factors predictive of disease trajectory membership, and assess if the different disease activity trajectories are associated with different damage trajectories.

Methods: This is a retrospective, longitudinal inception cohort of cSLE patients.

Methods for analyzing observational longitudinal prognosis studies for rheumatic diseases: a review & worked example using a clinic-based cohort of juvenile dermatomyositis patients.

Most outcome studies of rheumatic diseases report outcomes ascertained on a single occasion. While single assessments are sufficient for terminal or irreversible outcomes, they may not be sufficiently informative if outcomes change or fluctuate over time. Consequently, longitudinal studies that measure non-terminal outcomes repeatedly afford a better understanding of disease evolution.

Risk Factors for Symptomatic Avascular Necrosis in Childhood-onset Systemic Lupus Erythematosus.

Objective: To examine the frequency and risk factors for symptomatic avascular necrosis (AVN) in childhood-onset systemic lupus erythematosus (cSLE).

Methods: A single-center, nested, matched, case-control design was used. There were 617 patients with cSLE followed at the Hospital for Sick Children (SickKids) Lupus Clinic between July 1982 and June 2013 included in the study.

Longterm outcomes and damage accrual in patients with childhood systemic lupus erythematosus with psychosis and severe cognitive dysfunction.

Objective: (1) To describe the clinical course and response to treatment; and (2) to evaluate and compare damage accrual of distinct phenotypic subgroups of patients with clinically important psychiatric illness of pediatric systemic lupus erythematosus (pSLE).

Methods: A single-center cohort study of patients with pSLE followed at a pediatric lupus clinic from 1985 to July 2009. Clinical course and response to treatment were studied.

Psychiatric illness of systemic lupus erythematosus in childhood: spectrum of clinically important manifestations.

Objective: To determine the spectrum of manifestations in clinically important (i.e., requiring alterations of immunosuppressive therapy) psychiatric illness of pediatric systemic lupus erythematosus (pSLE) and to describe the laboratory and imaging features associated with psychiatric illness of pSLE (psySLE).

The risky business of studying prognosis.

Prognosis studies provide important healthcare information. Clinicians use prognostic factors to predict disease progress, thus allowing individualization of disease management. Prognosis is the issue in many translational studies that aim to identify biomarkers to predict outcomes.

Predicting longitudinal trajectory of bone mineral density in paediatric systemic lupus erythematosus patients.

Objectives: (1) To identify the average lumbar spine (LS) bone mineral density (BMD) trajectory in paediatric systemic lupus erythematosus (pSLE) patients, and (2) to identify predictors of BMD trajectory.

Methods: 68 consecutive newly diagnosed pSLE patients prospectively followed in our lupus cohort with three annual dual energy x-ray absorptiometry (DEXA) examinations were studied. Low LS BMD was defined as z-score ≤-2.