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

Luc Mouthon, Serge Poiraudeau, Margaret Vernon, Kelly Papadakis, Loïc Perchenet, Dinesh Khanna, Luc Mouthon, Serge Poiraudeau, Margaret Vernon, Kelly Papadakis, Loïc Perchenet, Dinesh Khanna

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.

Keywords: Patient perspective; Qualitative research; Systemic sclerosis; Treatment.

Conflict of interest statement

LM has received grants and consultancy fees from Actelion Pharmaceuticals Ltd., LFB Biotechnologies, CSL Behring and Roche.

MV declares that there are no competing interests.

KP and LP are employees of Actelion Pharmaceuticals Ltd.

DK has received grants from Boehringer Ingelheim, Bristol-Myers Squibb and Corbus Pharmaceuticals; has received consultancy fees from Actelion Pharmaceuticals Ltd., Acceleron Pharma, Arena Pharmaceuticals, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CSL Behring, Corbus Pharmaceuticals, Cytori Therapeautics, GlaxoSmithKline, Genentech (Roche), Galápagos NV, Merck & Co., Mitsubishi Tanabe Pharma and UCB; and holds stocks and/or shares in Eicos Sciences, Inc.

Figures

Fig. 1
Fig. 1
Schematic of the development, content validation and psychometric evaluation of the HDISS-DU instrument. CHFS, Cochin Hand Function Scale; HDDIS-DU, Hand Disability in Systemic Sclerosis-Digital Ulcers; n, number of participants; PROM, patient-reported outcome measure; SSc-DU, systemic sclerosis-related digital ulcer

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