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Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument.

ABSTRACT: BACKGROUND:We aimed to develop a patient-reported outcome measure, in accordance with the US Food and Drug Administration guidance, to capture the impact of systemic sclerosis-related digital ulcers (SSc-DUs) on hand function. Psychometric analyses were conducted to evaluate and document the measurement properties of the resulting instrument-the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®). METHODS:The HDISS-DU was developed through a series of confirmatory, qualitative concept-elicitation interviews (N?=?36) to provide supportive evidence that the instrument captures all relevant issues and functional limitations relating to SSc-DUs in this patient population. Psychometric analyses used blinded data from two randomised, controlled, phase 3 trials in patients with SSc-DUs (N?=?517). The analyses included assessment of reliability, construct validity, responsiveness and thresholds for meaningful change. RESULTS:Qualitative interviews confirmed that the HDISS-DU had good content coverage and patients understood the HDISS-DU instructions, items and response scale. The HDISS-DU demonstrated excellent internal consistency and test-retest reliability, with satisfactory construct validity. Overall, the HDISS-DU was highly responsive to change in digital ulcer severity: the no-change group (for other criterion measures) had mean differences and effect sizes close to 0, while mean differences were mostly negative (indicating improvement) for the improvement groups (for other criterion measures) and vice versa. The preliminary threshold for meaningful change was a 0.50 difference in HDISS-DU score. CONCLUSIONS:Using data from two large studies of SSc-DU patients, these psychometric analyses support the reliability, validity, discriminating ability and responsiveness to change of the HDISS-DU for evaluating treatment outcomes in future clinical studies and clinical practice.

SUBMITTER: Mouthon L

PROVIDER: S-EPMC6945532 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature

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Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument.

Mouthon Luc L Poiraudeau Serge S Vernon Margaret M Papadakis Kelly K Perchenet Loïc L Khanna Dinesh D

Arthritis research & therapy 20200106 1

<h4>Background</h4>We aimed to develop a patient-reported outcome measure, in accordance with the US Food and Drug Administration guidance, to capture the impact of systemic sclerosis-related digital ulcers (SSc-DUs) on hand function. Psychometric analyses were conducted to evaluate and document the measurement properties of the resulting instrument-the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®).<h4>Methods</h4>The HDISS-DU was developed through a series of confirmatory, qu ...[more]

PMID: 31907061

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Project description:Background Systemic sclerosis (SSc) is a rare disabling connective tissue disease with few available treatment options. Diffuse cutaneous systemic sclerosis (dcSSc) is associated with high mortality. A previous experiment has shown that JAK2 inhibitor can significantly improve skin fibrosis in bleomycin (BLM)-induced murine model, including reducing dermal thickening and collagen accumulation. We aimed to describe the efficacy of oral JAK1/2 inhibitor baricitinib in SSc patients, especially focusing on skin fibrosis and microvascular manifestations. Methods We described the different effects of oral selective JAK1, JAK2, or JAK3 inhibitor treatment in a BLM-induced skin fibrosis mouse model. Furthermore, 10 adult patients with dcSSc were treated with baricitinib. We assessed the changes in modified rodman skin score (mRSS) and digital ulcer net burden at week 12 and 24 from baseline. We also compared the absolute changes in scores on the Scleroderma Health Assessment Questionnaire (SHAQ) and a total score on the St. George's Respiratory Questionnaire (SGRQ) over a 24-week period. Results In the experimental mouse model of skin fibrosis, a JAK1 and JAK2 inhibitor ameliorated skin fibrosis, and a JAK2 inhibitor had the most obvious effect. Treatment with the JAK2 inhibitor also blunted the capillary rarefaction. We demonstrated that skin fibrosis and digital ulcers were significantly relieved in 10 SSc patients treated with baricitinib. The mRSS significantly improved at week 12 from baseline, with a mean change in mRSS of −8.3 [95% confidence interval (CI), −12.03 to −4.574; p = 0.0007] and improved greater at week 24 to −11.67 (95% CI, −16.84 to −6.496; p = 0.0008). Among the four patients with digital ulcers (DU), three were completely healed at week 24, the number of ulcers in another patient was significantly reduced, and there was no patient with new ulcers. Only one adverse event (AE) of herpes zoster was observed. Conclusions Our results indicate that selective JAK1 and JAK2 inhibitor alleviates skin fibrosis, and oral JAK1/2 inhibitor baricitinib is a potentially effective treatment for dcSSc patients with skin fibrosis and DU. Baricitinib was well-tolerated by most patients in this study. Additional large clinical trials are needed to confirm our pilot findings. Chinese Clinical Trial Registry Number ChiCTR2000030995.

Dataset Information

Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument.

Publications

Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument.

Similar Datasets

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