Assessment of the Systemic Sclerosis-Associated Raynaud's Phenomenon Questionnaire: Item Bank and Short-Form Development.


Journal

Arthritis care & research
ISSN: 2151-4658
Titre abrégé: Arthritis Care Res (Hoboken)
Pays: United States
ID NLM: 101518086

Informations de publication

Date de publication:
08 2023
Historique:
revised: 12 09 2022
received: 24 06 2022
accepted: 04 10 2022
medline: 28 7 2023
pubmed: 11 10 2022
entrez: 10 10 2022
Statut: ppublish

Résumé

To develop, refine, and score a novel patient-reported outcome instrument to assess the severity and impact of Raynaud's phenomenon (RP) in systemic sclerosis (SSc). The Assessment of Systemic Sclerosis-Associated Raynaud's Phenomenon (ASRAP) questionnaire items were developed with patient insight partner support and grounded in the lived patient experience of SSc-RP. ASRAP items underwent formal qualitative assessment and linguistic testing. An international multicenter study was undertaken to field test the preliminary ASRAP questionnaire. A preliminary 37-item ASRAP questionnaire was supplemented with 2 additional items following expert review to enhance content coverage before undergoing formal linguistic testing to optimize readability. Patient cognitive debriefing interviews were undertaken to enhance comprehension, ambiguity, cognitive difficulty, relevance, and content coverage of both the ASRAP items and instructions. We enrolled 420 SSc patients from scleroderma centers in the UK and US over 2 consecutive winters. Factor analysis with item response theory was undertaken to remove redundant and poorly fitting items. The retained 27-item long-form ASRAP questionnaire was calibrated and scored using the graded response model. A fixed 10-item short-form ASRAP questionnaire was developed using computerized adaptive testing simulations. 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. Long- and short-form ASRAP questionnaires have been calibrated and scored to permit formal validation.

Identifiants

pubmed: 36214062
doi: 10.1002/acr.25038
doi:

Types de publication

Multicenter Study Journal Article Research Support, U.S. Gov't, Non-P.H.S. Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1725-1734

Informations de copyright

© 2022 American College of Rheumatology.

Références

Walker UA, Tyndall A, Czirjak L, et al. Geographical variation of disease manifestations in systemic sclerosis: a report from the EULAR Scleroderma Trials and Research (EUSTAR) group database. Ann Rheum Dis 2009;68:856-62.
Pauling JD, Saketkoo LA, Matucci-Cerinic M, et al. The patient experience of Raynaud's phenomenon in systemic sclerosis. Rheumatology (Oxford) 2019;58:18-26.
Pauling JD, Domsic RT, Saketkoo LA, et al. Multinational qualitative research study exploring the patient experience of Raynaud's phenomenon in systemic sclerosis. Arthritis Care Res (Hoboken) 2018;70:1373-84.
Bassel M, Hudson M, Taillefer SS, et al. Frequency and impact of symptoms experienced by patients with systemic sclerosis: results from a Canadian National survey. Rheumatology (Oxford) 2011;50:762-7.
Black CM, Halkier-Sorensen L, Belch JJ, et al. Oral iloprost in Raynaud's phenomenon secondary to systemic sclerosis: a multicentre, placebo-controlled, dose-comparison study. Br J Rheumatol 1998;37:952-60.
Wigley FM, Korn JH, Csuka ME, et al. Oral iloprost treatment in patients with Raynaud's phenomenon secondary to systemic sclerosis: a multicenter, placebo-controlled, double-blind study. Arthritis Rheum 1998;41:670-7.
Merkel PA, Herlyn K, Martin RW, et al. Measuring disease activity and functional status in patients with scleroderma and Raynaud's phenomenon. Arthritis Rheum 2002;46:2410-20.
Pauling JD. The challenge of establishing treatment efficacy for cutaneous vascular manifestations of systemic sclerosis. Expert Rev Clin Immunol 2018;14:431-42.
Roustit M, Blaise S, Allanore Y, et al. Phosphodiesterase-5 inhibitors for the treatment of secondary Raynaud's phenomenon: systematic review and meta-analysis of randomised trials. Ann Rheum Dis 2013;72:1696-9.
Khouri C, Lepelley M, Bailly S, et al. Comparative efficacy and safety of treatments for secondary Raynaud's phenomenon: a systematic review and network meta-analysis of randomised trials. Lancet Rheumatol 2019;1:e237-46.
Pauling JD, Frech TM, Domsic RT, et al. Patient participation in patient-reported outcome instrument development in systemic sclerosis. Clin Exp Rheumatol 2017;35 Suppl 106:184-92.
Pauling JD, Frech TM, Hughes M, et al. Patient-reported outcome instruments for assessing Raynaud's phenomenon in systemic sclerosis: a SCTC Vascular Working Group report. J Scleroderma Relat Disord 2018;3:249-52.
Pauling JD, Reilly E, Smith T, et al. Evolving symptom characteristics of Raynaud's phenomenon in systemic sclerosis and their association with physician and patient-reported assessments of disease severity. Arthritis Care Res (Hoboken) 2019;71:1119-26.
Pauling JD, Saketkoo LA, Domsic RT. Patient perceptions of the Raynaud's condition score diary provide insight into its performance in clinical trials of Raynaud's phenomenon: comment on the article by Denton et al [letter]. Arthritis Rheumatol 2018;70:973-4.
DeWalt DA, Rothrock N, Yount S, et al. Evaluation of item candidates: the PROMIS qualitative item review. Med Care 2007;45 Suppl 1:S12-21.
Reise SP, Yu JY. Parameter recovery in the graded response model using MULTILOG. J Educ Meas 1990;27:133-44.
Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity: establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2--assessing respondent understanding. Value Health 2011;14:978-88.
Van den Hoogen F, Khanna D, Fransen J, et al. 2013 classification criteria for systemic sclerosis: an American College of Rheumatology/European League Against Rheumatism collaborative initiative. Arthritis Rheum 2013;65:2737-47.
Muthén LK, Muthén BO. Mplus: statistical analysis with latent variables. User's guide. 6th ed. 2010. URL: https://www.statmodel.com/download/usersguide/Mplus%20Users%20Guide%20v6.pdf.
Samejima F. Estimation of latent ability using a response pattern of graded responses. Psychometrika 1969;34 Suppl 1:1-97.
Cai L, Thissen D, du Toit SH. IRTPRO for Windows. Lincolnwood (IL): Scientific Software International; 2011.
Holland P, Wainer H, editors. Differential item functioning. Hillsdale (NJ): Routledge; 1993. p. 67-113.
Choi SW. Firestar: computerized adaptive testing simulation program for polytomous item response theory models. Appl Psychol Meas 2009;33:644-5.
Murphy SL, Lescoat A, Alore M, et al. How do patients define Raynaud's phenomenon? Differences between primary and secondary disease. Clin Rheumatol 2021;40:1611-6.

Auteurs

Lan Yu (L)

University of Pittsburgh, Pittsburgh, Pennsylvania.

Robyn T Domsic (RT)

University of Pittsburgh, Pittsburgh, Pennsylvania.

Lesley-Ann Saketkoo (LA)

University of Tulane Medical Center, New Orleans, Louisiana.

Jane Withey (J)

Royal National Hospital for Rheumatic Diseases, Royal United Hospitals NHS Foundation Trust, Bath, UK.

Tracy M Frech (TM)

Vanderbilt University, Nashville, Tennessee.

Ariane L Herrick (AL)

The University of Manchester, Northern Care Alliance NHS Foundation Trust, Manchester Academic Health Science Centre, and NIHR Manchester Biomedical Research Centre, Central Manchester NHS Foundation Trust, Manchester, UK, and Salford Royal NHS Foundation Trust, Salford, UK.

Laura K Hummers (LK)

Johns Hopkins University School of Medicine, Baltimore, Maryland.

Ami A Shah (AA)

Johns Hopkins University School of Medicine, Baltimore, Maryland.

Dinesh Khanna (D)

University of Michigan, Ann Arbor.

John D Pauling (JD)

Royal National Hospital for Rheumatic Diseases, Royal United Hospitals NHS Foundation Trust, Bath, UK, and North Bristol NHS Trust and University of Bristol, Bristol, UK.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH