PROMs for systemic sclerosis (Scleroderma)

Abstract

Scleroderma (SSc, systemic sclerosis) is a rare autoimmune connective tissue disease that often involves multiple organs and thus can affect many aspects from the view point of the patient. Patient-reported outcomes (PROs) are needed to capture these features. PROs that are commonly used in SSc include patient global assessment, function as measured by the Health Assessment Questionnaire Disability Index (HAQ, HAQ-DI), pain, fatigue, Raynaud’s Condition Score (RCS), gastrointestinal tract (UCLA SCTC GIT 2.0), the Symptom Burden Index (SBI), and the Scleroderma HAQ (SHAQ), which is the HAQ with added scales. Some measures are specific to SSc and others are general. General measures include health-related quality of life measures, such as the SF-36, that allow comparison between diseases. Specific measures have been developed for SSc and are usually validated using clinic patients or trial patients with SSc. The latter includes UCLA SCTC GIT 2.0, Symptom Burden Index, Mouth Handicap Scale in Systemic Sclerosis, and the UK SSc Functional Score. The RCS can be applied to any patient with either primary or secondary Raynaud’s Phenomenon (RP) including RP related to SSc. The majority of SSc patients have hand involvement. There are multiple measures for hand function that are not specific for SSc, including the HAQ; Cochin Hand Function Scale, Michigan Hand Questionnaire; and the Disabilities of the Arm, Shoulder and Hand (DASH). It is difficult to encompass all problems that occur in SSc with only one measurement tool. However, PROs in SSc are important to aid in understanding the impact of SSc on patients.