ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 2909

Attitudes Toward Patient-Reported Outcome Instruments for the Assessment of Raynaud’s Phenomenon in Systemic Sclerosis

John Pauling1,2, Tracy M. Frech3,4, Michael Hughes5, Jessica K. Gordon6, Robyn T. Domsic7, Francesca Ingegnoli8, Neil J. McHugh1,9, Sindhu R. Johnson10, Marie Hudson11, Francesco Boin12, Voon Ong13, Marco Matucci Cerinic14, Nezam Altorok15, Marina Scolnik16, Mandana Nikpour17, Ankoor Shah18, Janet E. Pope19, Dinesh Khanna20 and Ariane L. Herrick21, 1Department of Pharmacy and Pharmacology, University of Bath, Bath, United Kingdom, 2Department of Rheumatology, Royal National Hospital for Rheumatic Diseases, Bath, United Kingdom, 3Internal Medicine, Salt Lake City VAMC, Salt Lake, UT, 4Internal Medicine-Division of Rheumatology, University of Utah School of Medicine, SLC, UT, 5Centre for Musculoskeletal Research, The University of Manchester, Manchester, United Kingdom, 6Rheumatology, Hospital for Special Surgery, New York, NY, 7Medicine - Rheumatology, Univ of Pittsburgh Med Ctr, Pittsburgh, PA, 8Department of Rheumatology, Istituto Gaetano Pini, University of Milano, Italy, Milano, Italy, 9Rheumatology, Royal National Hospital for Rheumatic Diseases, Upper Borough Walls, United Kingdom, 10Medicine, Division of Rheumatology, Toronto Western Hospital, University Health Network Pulmonary Hypertension Programme, Toronto General Hospital, Mount Sinai Hospital and University of Toronto, Toronto, ON, Canada, 11Department of Medicine, McGill University and Jewish General Hospital, Montreal, QC, Canada, 12Rheumatology, University California San Francisco, San Francisco, CA, 13Rheumatology, University College London Medical School, Royal Free Hospital, London, UK, London, United Kingdom, 14Department of Medicine, Division of Rheumatology, University of Florence, Italy, Florence, Italy, 15Rheumatology, University of Toledo Medical Center,, Toledo, OH, 16Rheumatology Section, Rheumatology Unit, Internal Medicine Service, Hospital Italiano de Buenos Aires, Instituto Universitario Hospital Italiano de Buenos Aires, and Fundacion PM Catoggio, Buenos Aires, Argentina, 17Department of Medicine (St Vincent's Hospital), The University of Melbourne, Melbourne, Australia, 18Rheumatology and Immunology, Duke University Medical Center, Durham, NC, 19Monsignor Roney Bldg/Rheum, University of Western Ontario, St Joseph Health Care, London, ON, Canada, 20Division of Rheumatology, University of Michigan Medical School, Ann Arbor, MI, 21Centre for Musculoskeletal Research, University of Manchester, Manchester Academic Health Science Centre, Manchester, United Kingdom

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: ,

Session Information

Date: Tuesday, November 15, 2016

Title: Systemic Sclerosis, Fibrosing Syndromes, and Raynaud's – Clinical Aspects and Therapeutics - Poster III

Session Type: ACR Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose:  The Raynaud’s Condition Score (RCS) diary is a clinician-derived validated patient-reported outcome (PRO) instrument for assessing the frequency, duration and severity of Raynaud’s phenomenon in systemic sclerosis (SSc-RP).

Methods: The Scleroderma Clinical Trials Consortium Vascular Working Group (SCTC-VWG) has been assembled to develop better methods for assessing and managing peripheral vascular manifestations of SSc. SCTC-VWG members (n=28) were invited to participate in a survey gauging attitudes towards the RCS diary and the perceived need for novel tools for assessing SSc-RP.

Results:  Nineteen SCTC-VWG members (68% response rate) completed the survey. All were practicing rheumatologists with an interest in SSc (79% treating >15 SSc patients per month) affiliated with academic units based in North America (n=9), Europe (n=8), South America (n=1) and Australasia (n=1). The majority of respondents (95%) had participated in clinical trials of SSc and most (83%) had experience of SSc-RP endpoints. There were mixed views from respondents regarding the extent to which the RCS diary captures its intended conceptual framework (Table). There was broad consensus that RCS diary returns could be influenced by seasonal variation in weather, efforts made by patients to avoid or ameliorate attacks of RP, habituation to RP symptoms, the potential evolution of RP symptom characteristics with progressive obliterative microangiopathy, patient coping strategies, respondent burden and placebo effect (Table). There was consensus that the RCS diary might be a barrier to drug development (79% of respondents agree/strongly agree), that a novel PRO instrument for SSc-RP might aid drug development for SSc-RP (95% agree/strongly agree) and that a novel PRO instrument for SSc-RP should be developed with the combined input of clinicians and patients (84% agree/strongly agree).

Conclusion:  The chief limitations of this pilot work are the relatively small survey size and selection bias derived from targeting SCTC-VWG members. Nevertheless, the SCTC-VWG benefits from its composition of highly experienced clinicians affiliated with specialized SSc centers across four continents. A number of perceived limitations of the RCS diary have been highlighted along with concerns that these factors might impede drug development programs for SSc-RP. There is support within the scleroderma community for the development of a novel PRO instrument for SSc-RP. Table 1. Responses obtained from SCTC-VWG members in survey

Extent to which respondents agreed with each statement:
Unable to offer opinion Strongly disagree Disagree Neither disagree or agree Agree Strongly agree
The RCS diary accurately reflects: Frequency of RP attacks 1 (5) 1 (5) 2 (11) 6 (32) 8 (42) 1 (5)
Duration of RP attacks 1 (5) 1 (5) 3 (16) 8 (42) 5 (26) 1 (5)
Overall severity and impact of RP 1 (5) 1 (5) 2 (11) 6 (32) 7 (37) 2 (11)
The RCS diary returns are influenced by: Difficulty recognizing attacks of RP 0 (0) 0 (0) 7 (37) 2 (11) 7 (37) 3 (16)
Seasonal variation in weather 1 (5) 0 (0) 0 (0) 0 (0) 9 (47) 9 (47)
Efforts made to avoid attacks of RP 1 (5) 0 (0) 0 (0) 3 (16) 10 (53) 5 (26)
Efforts made to ameliorate attacks of RP 1 (5) 0 (0) 1 (5) 3 (16) 11 (58) 3 (16)
Habituation to RP symptoms over time 1 (5) 0 (0) 0 (0) 0 (0) 12 (63) 6 (32)
Evolution of morphological digital microvascular disease 1 (5) 0 (0) 1 (5) 4 (21) 9 (47) 4 (21)
Patient coping strategies 1 (5) 0 (0) 0 (0) 1 (5) 13 (69) 4 (21)
Excessive respondent burden 1 (5) 0 (0) 1 (5) 2 (11) 9 (47) 6 (32)
Placebo effect 2 (11) 0 (0) 1 (5) 2 (11) 8 (42) 6 (32)
The RCS diary: Might impede drug development in SSc-RP 2 (11) 0 (0) 2 (11) 0 (0) 9 (47) 6 (32)
Is satisfactory and no further research is required in this area 0 (0) 7 (37) 10 (53) 1 (5) 0 (0) 1 (5)
A novel PRO instrument for SSc-RP: Might aid drug development in SSc-RP 0 (0) 0 (0) 0 (0) 1 (5) 11 (58) 7 (37)
Should be primarily PATIENT-derived 0 (0) 0 (0) 1 (5) 3 (16) 9 (47) 6 (32)
Should be primarily CLINICIAN-derived 0 (0) 0 (0) 9 (47) 8 (42) 2 (11) 0 (0)
Should be CLINICIAN and PATIENT-derived 0 (0) 0 (0) 1 (5) 2 (11) 7 (37) 9 (47)
Disclosure: J. Pauling, None; T. M. Frech, None; M. Hughes, None; J. K. Gordon, None; R. T. Domsic, None; F. Ingegnoli, None; N. J. McHugh, None; S. R. Johnson, None; M. Hudson, None; F. Boin, None; V. Ong, None; M. Matucci Cerinic, None; N. Altorok, None; M. Scolnik, None; M. Nikpour, None; A. Shah, None; J. E. Pope, None; D. Khanna, None; A. L. Herrick, None.

To cite this abstract in AMA style:

Pauling J, Frech TM, Hughes M, Gordon JK, Domsic RT, Ingegnoli F, McHugh NJ, Johnson SR, Hudson M, Boin F, Ong V, Matucci Cerinic M, Altorok N, Scolnik M, Nikpour M, Shah A, Pope JE, Khanna D, Herrick AL. Attitudes Toward Patient-Reported Outcome Instruments for the Assessment of Raynaud’s Phenomenon in Systemic Sclerosis [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/attitudes-toward-patient-reported-outcome-instruments-for-the-assessment-of-raynauds-phenomenon-in-systemic-sclerosis/. Accessed .

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