Session Type: ACR Poster Session C
Session Time: 9:00AM-11:00AM
Background/Purpose: The patient perspective captured using Patient-Reported Outcome (PRO) instruments provide valuable insight into the patient condition not always captured by physician-derived assessment tools. Target patient population involvement is considered an essential component of PRO instrument design and is assessed by regulatory bodies when considering labelling claims in medical product development. We have reviewed the level of patient involvement in the development of PRO instruments used in the assessment of systemic sclerosis (SSc).
Methods: A comprehensive literature review was undertaken to identify studies reporting PRO instruments in SSc. Studies were assessed to establish whether the PRO instruments had been developed specifically for SSc or adopted from other disease areas. Studies reporting PRO instruments specific for SSc were scrutinised for evidence of target patient population involvement in the development of the instrument.
Results: A total of 58 PRO instruments that have been used in SSc research were identified. Twelve (21%) of these were developed specifically for outcome assessment within SSc populations (Table). Of these, 5 (42%) had not reported any patient involvement in the development phase of the instrument. Five SSc PRO instruments (42%) involved target patient population in the domain/item generation stage. Four (33%) of SSc PRO instruments had undertaken cognitive interviewing/linguistic evaluation to ensure item wording adequately captured the intended conceptual framework. The SCTC GIT questionnaires and the Systemic Sclerosis Questionnaire had each involved SSc patients in both domain/item generation and cognitive interviewing/linguistic evaluation stages of instrument development.
Conclusion: PRO instruments are particularly valuable in SSc due to the multi-faceted nature of the disease and the paucity of effective objective methods for assessing disease status. The majority of existing PRO instruments used in SSc have not involved significant target patient involvement in their development. By involving patients in the development and design phase of novel PRO instruments in SSc, we can ensure that PRO instruments used in the clinical and research settings adequately capture experiences most relevant to our patients. Table. Patient involvement in development of SSc-specifc PRO instruments * Authors report patient involvement but no details supplied.
|Organ system||Conceptual Framework||Author, Year||PRO||
Patient involvement in:
|Conceptual Framework||Domain generation||Item generation||Cognitive interviewing||Linguistic Evaluation||Respondent Burden|
|Disability & function||Disability & Function in SSc||Steen and Medsger, 1997||Scleroderma HAQ subscales||No||No||No||No||Yes||No|
|Disability and Function in SSc||Silman et al., 1998||UK Scleroderma Functional Score||No||No *||No *||No||No||No|
|Disability and Function in SSc||Guillevin and Ortonne, 1983||Scleroderma Functional Index||No||No||No||No||No||No|
|Global Assessment of Health Status||Global disease assessment||Suarez-Almazor, et al., 2007 & Kallen et al. 2010||Symptom Burden Index||No||Yes||Yes||No||No||No|
|Global disease assessment in SSc||Ruof et al., 1999||Systemic Sclerosis Questionnaire||No||Yes||Yes||Yes||Yes||No|
|Global disease assessment in SSc||
Ostojic and Damjanov, 2006
|Scleroderma Assessment Questionnaire||No||Yes||Yes||No||No||No|
|Skin||Skin thickening, tethering and thinness in SSc||Nagy et al. 2009||Patient Skin Self Assessment Questionnaire||No||No||No||No||Yes||No|
|Body Image||Body Image in SSc||Jewett, 2015||BCSS||No||No||No||No||No||No|
|Body Image in SSc||Jewett, 2010||Brief-SWAP||No||No||No||No||No||No|
|Peripheral Vascular||Raynaud’s phenomenon in SSc||Wigley et al., 1998 & Black et al. 1998||Raynaud’s Condition Score Diary||No||No||No||No||No||No|
|Gastrointestinal||GI symptoms in SSc||Khanna et al. 2007 & Khanna et al. 2009||SCTC GIT 1.0 and 2.0||No||Yes||Yes||Yes||Yes||No|
|Mouth Handicap in SSc||Mouthon et al., 2007||MHISS||Yes Postal survey||Yes Postal survey||Yes Postal survey||No||No||No|
To cite this abstract in AMA style:Pauling J, Frech TM, Domsic RT, Hudson M. Patient Participation in Patient Reported Outcome Instrument Development in Systemic Sclerosis [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/patient-participation-in-patient-reported-outcome-instrument-development-in-systemic-sclerosis/. Accessed November 27, 2020.
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