Abstract
Systemic sclerosis (SSc) is a rare, chronic autoimmune connective tissue disease affecting 60,000 – 100,000
people in the US. One main driver of physical disability in patients with SSc is upper extremity (UE) limitations
due to vascular and skin changes. These disease changes reduce joint movement, cause pain, and when not
treated early, can lead to permanent UE functional loss and rigidity, in the form of nonmalleable joint contractures.
Along with conventional pharmacological treatments to counteract disease severity, rehabilitation is
recommended to improve UE function and prevent long-lasting disability. Unfortunately, rehabilitation is only not
highly utilized and there are several barriers to receiving rehabilitation services. Access and travel to SSc
specialty centers are barriers, and traveling to repeated in-person sessions is not often logistically possible for
patients. In addition, the evidence base for rehabilitation does not consist of replicable protocol-based treatments
and relies on therapist expertise which results in variable care. The proposed study is designed to plan a multisite
randomized clinical trial with specialty centers and community sites to assess the efficacy of a telerehab
approach to improve UE function in patients with SSc. This is the result of several years of pilot testing and
preliminary work establishing a standard protocol for therapy and testing telerehab methods of treatment. The
planning for this multisite efficacy trial will result in adequate preparation and submission of a U01 application to
conduct the trial. We hypothesize that a supervised and tailored UE telerehab program will lead to improved UE
function in patients with diffuse cutaneous SSc compared to general education about symptom management.
We propose the following aims: 1. Establish our multisite research team that includes occupational therapists
who will serve as interventionists for the trial. 2. Complete the necessary milestones that will prepare us for a
successful U01 project application: 1) Design and refine a multisite clinical trial protocol; 2) Develop a Manual of
Operations and Procedures; 3) Refine telehealth platform and content; 4) Develop site-specific recruitment plans;
5) Develop a Data Safety and Monitoring Plan; 6) Establish our Data Management Plan; 7) Prepare and submit
single-site IRB application; 8) Create training materials for the interventionists and therapist assessor; and 9)
Conduct intervention fidelity training and test study processes. The proposed plan is the first step in examining
the efficacy of UE telerehab intervention that has the potential to be scalable and translatable into clinical
practice. Results of the trial would have implications for ongoing reimbursement for telerehab services and
potentially provide broader access to treatment of this rare condition. In addition, these methods could be applied
to other rheumatological conditions such as rheumatoid arthritis and lupus.
