The Scleroderma Patient-Centered Intervention Network Self-Management Program: Protocol for a Randomized Feasibility Trial

Background Systemic sclerosis (SSc), or scleroderma, is a rare disease that often results in significant disruptions to activities of daily living and can negatively affect physical and psychological well-being. Because there is no known cure, SSc treatment focuses on reducing symptoms and disability and improving health-related quality of life (HRQoL). Self-management programs are known to increase self-efficacy for disease management in many chronic diseases. The Scleroderma Patient-centered Intervention Network (SPIN) developed a Web-based self-management program (SPIN self-management; SPIN-SELF) to increase self-efficacy for disease management and to improve HRQoL for patients with SSc. Objective The proposed study aims to assess the feasibility of conducting a full-scale randomized controlled trial (RCT) of the SPIN-SELF program by evaluating the trial implementation processes, required resources and management, scientific aspects, and participant acceptability and usage of the SPIN-SELF program. Methods The SPIN-SELF feasibility trial will be conducted via the SPIN Cohort. The SPIN Cohort was developed as a framework for embedded pragmatic trials using the cohort multiple RCT design. In total, 40 English-speaking SPIN Cohort participants with low disease management self-efficacy (Self-Efficacy for Managing Chronic Disease Scale score ≤7), who have indicated interest in using a Web-based self-management program, will be randomized with a 3:2 ratio into the SPIN-SELF program or usual care for 3 months. Feasibility outcomes include trial implementation processes, required resources and management, scientific aspects, and patient acceptability and usage of the SPIN-SELF program. Results Enrollment of the 40 participants occurred between July 5, 2019, and July 27, 2019. By November 25, 2019, data collection of trial outcomes was completed. Data analysis is underway, and results are expected to be published in 2020. Conclusions The SPIN-SELF program is a self-help tool that may improve disease-management self-efficacy and improve HRQoL in patients with SSc. The SPIN-SELF feasibility trial will ensure that trial methodology is robust, feasible, and consistent with trial participant expectations. The results will guide adjustments that need to be implemented before undertaking a full-scale RCT of the SPIN-SELF program. International Registered Report Identifier (IRRID) DERR1-10.2196/16799


Number of Reviewers
Your application was reviewed by your peers and considered to be of exceptionally high quality. You should take great pride in this achievement, particularly given the highly competitive nature of CIHR funding.
As you know, peer review is the cornerstone of our research funding system. This process is made possible because of the volunteerism of individuals who generously gave their time to review your application. We are continuously recruiting and retaining the most accomplished innovative and creative scientists to review health research proposals. As a CIHR-funded researcher, you may be invited to serve in the peer review process for future competitions. The proposed RCT is anchored in The Scleroderma Patient-centered Intervention Network (SPIN, international) which was created to deal with challenges due to lack of N/power in such a rare condition. Importantly, the proposal follows a CIHR Team Grant which was awarded to create an infrastructure for trials of online disease-management tools for SSc (N=2000 patients in 2016, 440 will participate to the trial). SPIN uses a novel cohort-based trial design, in which patient outcome data are collected at regular intervals and trials are embedded in the cohort.
Applicants mention that the development, study of feasibility and testing of the proposed intervention has already been done. The intervention consists in an internet-based SM program on topics that were identified as relevant by the patients themselves in previous studies. The modules address 9 different targets.

Weaknesses/Faiblesses:
My main question here is with the primary outcome chosen. The objective of a selfmanagement program is ultimately to change the experience of symptoms and behaviours. Although global self-efficacy to self-manage is a relevant preliminary target, I wonder why specific clinical targets as related to modules were not included as outcomes. For example, fatigue, emotional distress, or sleeping difficulties are domains where validated measures exist.
Wouldn't it be more clinically relevant to include these measures at least as secondary outcomes? (you could pick those who are more frequently reported by patients). Doing so, you could show that you empower patients and that this translates into relevant clinical outcomes. The current secondary outcome (HRQL) is a very distal target which is likely to be influenced by many other aspects, including relevant clinical outcomes (such as fatigue) identified by patients.
Criterion/Critère: Importance of the Idea/Importance de l'idée

Strengths/Forces:
There is an important need to improve self-management in this population and to improve the way we test interventions in rare diseases. To date, only two pre-post intervention studies of SSc self-management programs have been conducted. This proposed trial will evaluate whether an online intervention can improve disease-management self-efficacy for patients with a rare disease without disease-modifying treatments. If effective, patient organization partners will disseminate the intervention to patients and healthcare providers. This could also serve as a prototype for similar programs in other rare diseases.

Rating/Cote: E+
Strengths/Forces: This is a very powerful design as the trial is embedded in the cohort. Apparently patients who consented to be part of the cohort are more or less automatically proposed the trials (high feasibility) The methods are very well written, and appropriate. This appears as a methodologically sound project. The intervention and the comparator are well defined, although the comparator could differ across countries (but this will be controlled for in analyses, the ability to control for that depends on N/site though). The measure of self-efficacy to self-manage appears a very subjective evaluation of one's capacity. With such a measure, positivity biases and expectations effects may be maximized in an unblinded trial, especially if the measure remains global and aggregates 6 different domains. Another limitation with this measure concern its relationship with the modules of the intervention. Logically one would expect direct links between the 6 items of the scale and the 9 domains of the intervention.

Weaknesses/Faiblesses
Although this issue was probably anticipated by the team, it is unclear how the applicants will deal with potential overlap of this RCT with other future RCT. You mentioned that the cohort participants will be routinely proposed enrollment in research. Here you plan to recruit roughly 25% of your cohort, so overlap is certain in the future. This could lead to important biases.