EUSTAR Registration Impacts Favourably on Clinical Practice

Dear Editor,

We read with interest the article “Subsets in systemic sclerosis: one size does not fit all” (1) and wish to emphasize the importance of collaborative studies. Disease registries, in particular, collect uniform data and are traditionally regarded as beneficial because they resolve limitations posed by clinical trials through inclusion of the entire spectrum of patient populations, providing information on real-life experience (2). A potential added value to patient registries is the effect that it may have on clinical practice, an aspect that has remained largely unexplored.

The European Scleroderma Trials and Research group (EUSTAR) was formed in 2004 and aims to foster the awareness and research of systemic sclerosis (SSc). Our Unit became affiliated to EUSTAR in 2015. The affiliation process started with a request to the Portuguese Data Protection Authority (Comissão Nacional de Protecção de Dados – CNPD). Once obtained, the CNPD permit was submitted to the Ethics Committee (EC) of our Hospital Centre, together with specifically written patient information and informed consent forms. EUSTAR online registration was completed after the EC [160 (2014)] and the CNPD [0342 (2014)] approvals. Informed consent was obtained from all individual participants included in the study.

Our database was created in April 1994 and included 70 patients up to September 2016. From these, the current study identified 9 patients that were better classified by other disease entities, 13 had been lost to follow-up and Very Early Diagnosis of Systemic Sclerosis (VEDOSS) was identified in 6 patients. SSc was confirmed in 19 patients under regular care (100% fulfilment of current American College of Rheumatology/European League against Rheumatism joint 2013 classification criteria) (3) who were prospectively recruited and categorized according to the LeRoy (4) into 5 diffuse cutaneous (dcSSc), 11 limited cutaneous (lcSSc) and 3 limited (lSSc) subtypes, followed for a median period of 5, 12 and 6 years, respectively. Of note, Raynaud's phenomenon and abnormal nailfold capillaries were universally present. Interstitial lung disease (ILD) was absent in lcSSc but present in 100% of dcSSc patients. Pulmonary Artery Hypertension by echocardiography was excluded in those patients who underwent right heart catheterization. Therapy reflected prior and current need for immunosuppressive therapy and endothelin receptor antagonists are currently used in 4 dsSSc and 6 lcSSc patients. Likewise, there was heterogeneity of clinical phenotype and disease progression within each subtype. The mortality was 55% (23/42) over 21 years.

EUSTAR registration provided us with a practical framework for patient phenotyping, an update of screening procedures and an internal review of therapy suitability on an individual basis. The identification of anti-RNA polymerase III antibodies also allowed us to participate in a EUSTAR study (accepted for publication in Journal of Rheumatology).

We encourage EUSTAR registration as we demonstrate its favourable impact on participation in clinical research, patient screening and treatment. Cohort descriptions of SSc are recent and very scarce in Portugal (5). We anticipate it will facilitate further characterization of SSc across healthcare settings.