Treatment recommendations for the systemic pharmacological treatment of systemic sclerosis digital ulcers: Results from the World Scleroderma Foundation Ad Hoc Committee.

Introduction: Digital ulcers are an important disease manifestation of systemic sclerosis and are associated with significant morbidity. As such, there is an urgent need for the development of evidence-based recommendations to guide clinicians in the treatment of digital ulcers.

Methods: A steering committee of international experts was established.

'Am I doing this right?' Physician perceptions of the global assessment in clinical trials of systemic sclerosis.

Objectives: Physician global assessments (PhyGAs) are commonly performed in randomised controlled trials (RCTs) in systemic sclerosis (SSc). However, there is no single PhyGA applied across RCTs. We performed an exploratory qualitative study to explore perceptions of the PhyGA, its role in RCTs and how physicians perform their own assessment.

Recommendations for the local management of digital ulcers in systemic sclerosis: A report from the World Scleroderma Foundation (WSF) 'Ad hoc committee'.

Introduction: Digital ulcers (DUs) stand out as one of the most prevalent and clinically meaningful manifestations of systemic sclerosis (SSc) and are associated with significant morbidity. While systemic (pharmacological) therapy is currently established as the 'standard of care', effective local ulcer management remains crucial for all cases of DUs. This is particularly true for patients who cannot tolerate systemic treatments or in the case of refractory SSc-DUs.

The minimal clinically important difference of the scleroderma clinical trials consortium damage index.

Objective: The Scleroderma Clinical Trials Consortium Damage Index is an index of global damage in systemic sclerosis. The objective of this study is to determine the minimal clinically important difference of the Scleroderma Clinical Trials Consortium Damage Index.

Methods: Patients in the Canadian Scleroderma Research Group registry and the Australian Scleroderma Cohort Study who completed Scleroderma Clinical Trials Consortium Damage Index scores and the SF36v2 at baseline and the first full follow-up visit were studied.

Improvement in Skin Fibrosis and Lung Function with Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis.

Systemic sclerosis (SSc) is a severe, progressive disease with limited treatment options. Autologous hematopoietic stem cell transplantation (AHSCT) has been shown to be an effective treatment for rapidly progressive SSc. The objective of this study was to evaluate the effectiveness of AHSCT for SSc compared to real-world clinical care.

Immunosuppressive Drugs in Early Systemic Sclerosis and Prevention of Damage Accrual.

Objective: Organ damage in patients with systemic sclerosis (SSc) in individual organs such as the lungs may be prevented by receiving immunosuppressive drugs (ISs). A new measure of global organ damage, the Scleroderma Clinical Trials Consortium Damage Index (SCTC-DI), has allowed us to investigate whether receiving ISs may reduce global organ damage accrual in patients with early SSc.

Methods: This was a retrospective study of patients with two or less years of disease duration in Canadian and Australian cohorts with SSc.

The burden and determinants of fatigue in incident and prevalent systemic sclerosis.

Objectives: To investigate the burden and clinical associations of fatigue in systemic sclerosis (SSc) as measured by FACIT-Fatigue scores.

Methods: Australian Scleroderma Cohort Study participants with ≥1 FACIT-Fatigue score were included. Participants were divided into those with incident SSc (≤5 years SSc duration at recruitment and FACIT-Fatigue score recorded within 5 years of disease onset) or prevalent SSc (first FACIT-Fatigue score recorded >5 years after SSc onset).

Investigating the trajectory of functional disability in systemic sclerosis: group-based trajectory modelling of the Health Assessment Questionnaire-Disability Index.

Objectives: To identify the trajectories and clinical associations of functional disability in systemic sclerosis (SSc).

Methods: Australian Scleroderma Cohort Study (ASCS) participants meeting ACR/EULAR criteria for SSc recruited within 5 years of disease onset, with ≥2 Health Assessment Questionnaire-Disability Index (HAQ-DI) scores were included. Group based trajectory modelling (GBTM) was used to identify the number and shape of HAQ-DI trajectories.

Epidemiology of systemic sclerosis in Quebec, Canada: a population-based study.

Background: Systemic sclerosis (SSc) is a systemic life-threatening autoimmune rheumatic disease. We aimed to assess the incidence, prevalence, mortality and spatiotemporal trends of SSc in Quebec, Canada with stratification by sex and age.

Methods: SSc cases were identified from Quebec populational databases from 1989 to 2019.

Development and Initial Validation of the Novel Scleroderma Clinical Trials Consortium Activity Index.

Objective: Accurate measurement of disease activity in systemic sclerosis (SSc) remains a significant clinical challenge. The Scleroderma Clinical Trials Consortium (SCTC) convened an Activity Index (AI) Working Group (WG) to develop a novel measure of disease activity (SCTC-AI).

Methods: Using consensus methodology, we developed a conceptual definition of disease activity.

Brentuximab vedotin for skin involvement in refractory diffuse cutaneous systemic sclerosis, an open-label trial.

Objective: We explored the efficacy and safety of brentuximab vedotin, a chimeric anti-CD30 antibody drug conjugate, in patients with severe active diffuse cutaneous systemic sclerosis (dcSSc).

Methods: This phase II proof-of-concept, single centre, open-label, single arm, investigator-initiated trial included patients ≥18 years, with dcSSc, modified Rodnan skin score (mRSS) ≥15 with <5 years since the first non-Raynaud's symptom and/or skin worsening despite immunosuppression who were treated with intravenous brentuximab vedotin 0.6 mg/kg q3 weeks for 45 weeks.

Proton pump inhibitors in systemic sclerosis: a reappraisal to optimise treatment of gastro-oesophageal reflux disease.

Gastroesophageal reflux disease (GERD) is associated with significant morbidity in patients with systemic sclerosis (SSc). Although the introduction of proton pump inhibitors (PPIs) into clinical care have represented a major achievement in the management of oesophago-gastric problems in SSc, PPIs are seldom fully effective in SSc patients, and the utilization of maximum PPI dosages is a very frequent clinical practice. However, currently there is little evidence currently to support the empiric use of PPIs in SSc which is especially relevant in regard to safety concerns of long-term exposure with have been raised in the general population.

Surgical management of digital ulcers in systemic sclerosis: A systematic literature review.

Background: There is a strong rationale to develop locally-acting surgical treatments for digital ulcers (DUs) in patients with systemic sclerosis (SSc). Our aim was to examine the safety and efficacy of local surgical management for SSc-DU.

Methods: A systematic literature review was carried out until to August 2022 using 7 different databases.

Systemic pharmacological treatment of digital ulcers in systemic sclerosis: a systematic literature review.

Objective: To evaluate the evidence concerning systemic pharmacological treatments for SSc digital ulcers (DUs) to inform the development of evidence-based treatment guidelines.

Methods: A systematic literature review of seven databases was performed to identify all original research studies of adult patients with SSc DUs. Randomized controlled trials (RCTs) and prospective longitudinal observational studies (OBSs) were eligible for inclusion.

Special Issue "Rheumatic Diseases: Pathophysiology, Targeted Therapy, Focus on Vascular and Pulmonary Manifestations 2022".

This Special Issue, titled "Rheumatic Diseases: Pathophysiology, Targeted Therapy, Focus on Vascular and Pulmonary Manifestations", aims to demonstrate recent and new advances and future trends in the field of rheumatic diseases [...

Effect of pregnancy on scleroderma progression.

Objective: To explore the trajectory of scleroderma disease activity in women who experienced a pregnancy after systemic sclerosis diagnosis compared to nulliparous women.

Methods: We analyzed data from the Canadian Scleroderma Research Group registry by identifying nulliparous women and women with ⩾1 pregnancy after systemic sclerosis diagnosis. Patient characteristics were compared between groups at registry entry.

Prediction of damage trajectories in systemic sclerosis using group-based trajectory modelling.

Objectives: Damage accrual in SSc can be tracked using the Scleroderma Clinical Trials Consortium Damage Index (DI). Our goal was to develop a prediction model for damage accrual in SSc patients with early disease.

Methods: Using patients with <2 years disease duration from Canada and Australia as a derivation cohort, and from the Netherlands as a validation cohort, we used group-based trajectory modelling (GBTM) to determine 'good' and 'bad' latent damage trajectories.

Evaluation of Patient and Physician Assessments of Gastrointestinal Disease Activity in Systemic Sclerosis.

Objective: To assess whether patient and physician global assessment of gastrointestinal tract (GIT) disease in systemic sclerosis (SSc) are associated with a meaningful change in disease status.

Methods: One hundred forty-three participants from the Australian Scleroderma Cohort Study were recruited to this study. Using logistic regression analysis, we evaluated the relationship between patient-reported and physician-assessed GIT disease status and symptoms, measures of health-related quality of life (36-item Short Form Health Survey [SF-36]) and GIT disease severity, measured by the Scleroderma Clinical Trials Consortium UCLA Gastrointestinal Tract 2.

Patient and Physician Global Assessments of Disease Status in Systemic Sclerosis.

Global assessments of disease by both patients and physicians are widely used in clinical studies of systemic sclerosis (SSc). They are commonly secondary end points in randomized controlled trials (RCTs) and are considered important items in composite measures of treatment response. A comprehensive literature review was conducted of the formats, wording, and clinimetric properties of the patient global assessment of disease status (PtGA) and physician global assessment of disease status (PhGA) used in RCTs of SSc.