Exploring the optimal follow-up for systemic sclerosis patients: study protocol for a Dutch multicenter randomized controlled trial

BACKGROUND: Currently, evidence-based guidelines for the frequency and intensity of follow-up of systemic sclerosis (SSc) patients are not available. Based on expert opinion, an annual extensive evaluation is recommended. A multidisciplinary Care Pathway that integrates this annual extensive evaluation at the Leiden University Medical Center has shown increased patient satisfaction, decreased healthcare utilization, and improved outcomes in SSc patients (1, 2). However, for a subgroup of SSc patients with relatively mild disease, this annual extensive evaluation might be redundant. Therefore, this study aims to evaluate whether assessment in a regular outpatient clinic setting is an acceptable alternative to extensive annual evaluation in the Care Pathway in SSc patients with a low risk of disease progression. METHODS: This study is designed as a multicenter (n = 3) non-inferiority randomized controlled trial. SSc patients are categorized into three categories for risk of disease progression (low, intermediate, or high) based on a previously published risk prediction model (3). Patients with a predicted low or intermediate risk of disease progression are randomized between (1) follow-up in the outpatient clinic (intervention) and (2) follow-up via usual care according to the annual Care Pathway (control group). The year after the "study visit," all patients are evaluated in the Care Pathway. In this study, 250 patients will be recruited and randomized. The primary outcome is healthcare utilization, which will be assessed via questionnaires. Secondary outcome measures include disease progression, patients' perception of disease and care, and health-related quality of life. Healthcare utilization is defined as the number of contacts with a healthcare professional and will be analyzed using descriptive statistics and linear regression analysis. DISCUSSION: There is an unmet need for tailor-made care for SSc patients in accordance with disease activity, and evidence-based guidelines regarding the follow-up of SSc patients are lacking. This is the first randomized controlled trial evaluating the optimal follow-up for SSc patients at low risk of disease progression. Results of this study will show whether routine assessment at the outpatient clinic is an acceptable alternative to assessment in a standardized care setting including an annual 6-min walk test, an ECG, lab, mRSS, and a pulmonary function test. TRIAL REGISTRATION: ClinicalTrials.gov NCT05103553. Registered on October 11, 2021.