Novel PRO Tool Assesses Impact, Severity of Raynaud Phenomenon in Systemic Sclerosis

A novel patient-reported outcome (PRO) instrument — the Assessment of Systemic Sclerosis-Associated Raynaud’s Phenomenon (ASRAP) questionnaire — was developed to evaluate the severity and impact of Raynaud phenomenon in systemic sclerosis (SSc-RP). The study was published in Arthritis Care & Research (Hoboken).

It is well known that Raynaud phenomenon is the most common disease manifestation in patients with SSc. In fact, SSc-RP is a major cause of disease-related morbidity, which is ranked high by patients with regard to severity and impact. Symptoms of SSc typically include ischemic pain, sensory impairment, and decreased function of fingers. These symptoms are often associated with emotional distress, feelings of body image dissatisfaction, and embarrassment linked to discoloration of digits.

The researchers evaluated the use of the ASRAP questionnaire both in clinical practice and research/trial protocols. The items on the ASRAP were developed with patient insight partner support and were based on the lived patient experience of SSc-RP. All items on the ASRAP underwent formal qualitative evaluation and linguistic testing. 

The 37 ASRAP items were designed to capture domains including “physical symptoms,” “emotional distress,” “impact on daily life,” “exacerbating factors,” “self-management,” “adaptation,” and “uncertainty.” All item response options were designed to conform to the standards developed for the Patient-Reported Outcome Information System initiative. The preliminary 37-item ASRAP questionnaire was supplemented with 2 additional items in an effort to enhance content coverage prior to undergoing formal linguistic testing to optimize the readability.

Cognitive debriefing interviews were conducted with English-speaking patients with SSc to evaluate patient perceptions regarding comprehensibility, language, relevance, cognitive difficulty, ambiguity, and content coverage of the items. All interviews were held for approximately 90 minutes, with a comfort break. The cognitive debriefing was also conducted to ensure that the ASRAP items met accepted criteria for optimal translatability into non-English languages.

The researchers used factor analysis with item response theory to remove redundant and poorly fitting items. The 27-item long-form ASRAP questionnaire was scored and calibrated using the graded response model. Further, a fixed 10-item short-form ASRAP (ASRAP-SF) was designed using computerized adaptive testing simulations.

The study included 438 patients with SSc from scleroderma centers in the UK and US from February to March 2019 and November 2019 to March 2020. Overall, 79.8% of the cohort were women and 86.5% were White. The disease subsets of the participants included 59.4% with limited scleroderma, 34.7% with diffuse scleroderma, and 5.9% with sine scleroderma. The mean patient age was 58.9 (SD, 12.4) years. Disease duration since onset of Raynaud phenomenon symptoms and onset of first non-Raynaud phenomenon SSc symptom were 15.4±12.1 years and 11.8±9.7 years, respectively.

Additional research is underway to explore the construct validity, reliability, and responsiveness of the ASRAP and the ASRAP-SF questionnaires to enable them to be applied in both clinical trials and routine clinical practice for evaluating the severity of SSc-RP.

According to the study authors, “The ASRAP questionnaire has been developed with extensive SSc patient input, with items grounded in the lived experience of SSc-RP to ensure strong content validity, with a focus on how patients ‘feel’ and ‘function.’ An advanced psychometric approach with expert input has removed redundant and/or poorly fitting items, without eroding content validity.”

Disclosure: Some of the study authors have declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures. 

Reference

Yu L, Domsic RT, Saketkoo L-A, et al. The Assessment of Systemic sclerosis-associated RAynaud’s Phenomenon (ASRAP) questionnaire: item bank and short form development. Arthritis Care Res (Hoboken). Published online October 10, 2022. doi:10.1002/acr.25038