Abstract
Objective:
Aspects of ANCA-associated vasculitis (AAV) prioritized by patients with AAV were described using the International Classification of Function, Disability, and Health.
Methods:
Items identified during 14 individual interviews were incorporated into ICF-based questionnaire administered to participants of 2 vasculitis patient symposia, 36 in UK and 63 in USA.
Results:
Categories identified as at least “moderately relevant” by ≥ 5% of subjects included 44 body functions, 14 body structures, 35 activities and participation, 31 environmental factors, and 38 personal factors.
Conclusion:
Identified categories differ from those captured by the current OMERACT core set and those prioritized by vasculitis experts.
Keywords: OMERACT, vasculitis, outcome assessment
INTRODUCTION
The vasculitides are a group of heterogeneous conditions characterized by inflammation of blood vessels. ANCA-associated vasculitis (AAV) are small vessel vasculitides that include granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (EGPA). AAV can affect practically any organ system, with manifestations ranging from disease limited to ears, nose, and sinuses to life-threatening failure of kidneys, lungs, or heart.
Outcome Measures in Rheumatology (OMERACT) is an international organization that strives to develop data-driven, optimal outcome measures for use in clinical trials (1). In 2010 OMERACT endorsed a Core Set of Outcome Measures for ANCA-Associated Vasculitis (2). Subsequently, a framework was developed for selection of areas and domains that should be assessed in clinical trials, referred to as the OMERACT Filter 2.0 framework (1,3); it calls for inclusion of a range of stakeholders, especially patients, into the process of outcome measure development.
The OMERACT Vasculitis Working Group has conducted several projects aimed at updating and expanding the existing expert-driven OMERACT Core Set for AAV(4,5), including this project which utilizes the International Classification of Function, Disability and Health (ICF). The ICF is a general health status framework (6) that views health as a broad concept shaped by the relationship between various ICF components: impairments of body functions and body structures, limitations of activities, restrictions of participation and the influence of environmental and personal contextual factors (6). ICF also offers a classification system to describe functioning and health using categories organized into a four-level hierarchically nested structure (6). The OMERACT initiative endorsed ICF as a tool to identify and classify (sub)-domains relevant to measurement of outcomes for a specific medical condition (1).
This manuscript describes the ICF-based analysis of aspects of health prioritized by patients with AAV.
METHODS
The study consisted of two parts, both overseen by a steering committee comprised of content experts, methodologists, including qualitative experts, and patients with AAV. The research received approval from the Ottawa Hospital’s Research Ethics Board (protocol # 20120604–01H and 20150189–01H), and all participating patients gave informed consent.
The first, qualitative, part of the study, consisted of a series of individual semi-structured interviews conducted with English-speaking adult patients with AAV at the Ottawa Hospital in Ottawa, Canada and the Nuffield Orthopaedic Centre in Oxford, United Kingdom (UK). A purposive sampling strategy was used to select interviews for this study and aimed at including patients with each of the 3 types of AAV, with different severities, and at different stages of disease (7); interviews were performed until saturation of the identified concepts was reached (8).
Interviews were audio recorded, professionally transcribed, and all basic concepts were “linked” to the most precise ICF category according to previously established ICF linking rules by Cieza et al (9,10), and then summarized on the 2nd ICF level, as we described previously (11). As personal factors are currently not classified by the ICF, the general scheme proposed by Geyh et al (12) was followed. It divides the personal factors into 3 broad groups: 1) “facts” about the individual’s position in the physical, social, and temporal context, 2) “experience” of the concept in question (feelings, thoughts, beliefs and motives,) and 3) “patterns” of experience and behaviour including personality traits and habits.
In the second part of the study, categories identified through the qualitative analysis were incorporated into an ICF-based questionnaire that was administered to participants of 2 vasculitis patient symposia: International Vasculitis and ANCA Workshop in London, UK, April 18, 2015 and the Vasculitis Foundation symposium in Jacksonville FL, USA, June 19–21, 2015. Participants rated the relevance of each listed category on a standardized ordinal scale associated with the ICF classification (6). The physiologic effects of vasculitis and its effects on patients’ activities were rated on a 5-level scale from 0 to 4 with the following categories: “no effect,” “mild,” “moderate,” “severe,” and “complete impairment.” The relevance of environmental and personal factors was rated on a 9-level scale from −4 (“extreme negative effect on health”), through the same gradations to 0 (“no effect”), and then to +4 (“extreme positive effect”). Factors that have at least “moderate” effect for at least 5% of respondents were selected, as suggested previously (13).
RESULTS
Fourteen individual interviews (10 from Ottawa, Canada and 4 from Oxford, UK) were available for the qualitative portion of the study. Participants identified a wide range of important aspects of AAV that were linked to 159 ICF categories.
The underpinning qualitative findings were incorporated into a questionnaire administered to approximately 100 participants at each of the 2 vasculitis patient symposia described above; 51 and 74 patients from the UK and USA returned the questionnaire, and 36 and 63 (respectively) had AAV and were included in this analysis. Participants’ demographic and clinical characteristics are summarized in Supplementary Table 1; the respondents’ disease spectrum was representative of a general population of patients with AAV.
All of the categories presented in the questionnaire were rated as at least “moderately relevant” by at least 5% of subjects. This includes 44 second-level categories in the ICF component body functions, 14 in body structures, 35 in activities and participation, 31 in environmental factors, and 38 in personal factors. A subset of the most relevant categories, ranked as at least “moderately relevant” by at least 30% of participants, is summarized in Tables 1–3.
Table 1.
Categories from the International Classification of Function (ICF) compenents Body Functions, Body Structures, and Activities and Participation identified as at least moderately important by at least 30% of participants.
| ICF CODE# |
ICF Category description | % ranking category as ≥moderately impaired |
|---|---|---|
| BODY FUNCTIONS | ||
| b1 | Mental Functions | |
| b130 | Energy and drive | |
| b1300 | Energy | 87% |
| b1301 | Motivation (wanting to do things) | 59% |
| b1302 | Appetite | 47% |
| b134 | Sleep (quality and quantity) | 68% |
| b147 | Agitation, feeling “jittery” | 31% |
| b152 | Emotions such as feeling down or depressed, anxious, frustrated | 46% |
| b180 | Body image (one’s satisfaction with their look) | 42% |
| b2 | Sensory Functions, Pain, Voice and Speech | |
| b230 | Hearing | 40% |
| b240 | Sensations related to the ears - ringing, irritation, pressure | 38% |
| b279 | Numbness, decreased sensation (peripheral sensory neuropathy) | 47% |
| b280 | Pain | 80% |
| b4 | Cardiovascular, hematological, immunological, respiratory systems | |
| b420 | Blood pressure | 35% |
| b435 | Functions of immune system: protection against infections, hypersensitivity reactions (e.g. allergies) | 50% |
| b440 | Lung function (breathing, results of lung function tests) | 50% |
| b455 | Exercise tolerance | 67% |
| b460 | Shortness of breath, wheezing, chest tightness / other sensations associated with lungs or heart | 56% |
| b5 | Digestive, metabolic, and endocrine functions | |
| b530 | Weight issues (weight loss or gain) | 64% |
| b535 | Sensations related to digestive system: nausea, feeling bloated, heartburn, abdominal cramps | 40% |
| b550 | Fever, chills, sweats | 46% |
| Other body functions | ||
| b310 | Ability to speak, quality of voice | 30% |
| b610 | Functions of kidney and bladder (making urine, peeing) | 37% |
| b710 | Joint functions (moving joints etc.) | 44% |
| b840 | Sensation related to the skin: itching, burning, and tingling | 34% |
| BODY STRUCTURES | ||
| s310 | Nose | 55% |
| nc-bs* | Sinuses | 59% |
| s430 | Lungs and airways (breathing tubes) | 53% |
| s6100 | Kidneys and urinary bladder | 37% |
| s7701 | Joints (includes arthritis) | 42% |
| s810 | Skin - rashes, scarring, thinning and breaking down | 34% |
| ACTIVITIES AND PARTICIPATION | ||
| d4 | Mobility | |
| d430 | Lifting and carrying objects | 37% |
| d450 | Walking | 42% |
| d4551 | Climbing (e.g. stairs) | 52% |
| Other activities and participation | ||
| d920 | Recreation and leisurely activities | 65% |
ICF code is a numeric identifier that uniquely identifies each ICF category; it starts denotes the ICF component (b: body functions; s: body structures; d: activities and followed by numbers that specify progressively more detailed categories; for example, category b1300(energy) is a sub-category of the broader category b130 (energy and drive).
Not covered - body structures; category not covered by the current ICF classification.
Table 3:
International Classification of Function (ICF) Personal Factors identified as at least moderately relevant by at least 30% of participants
| ICF Code | ICF Category description | % ranking category as ≥ moderately relevant |
|---|---|---|
| p1 | Facts about individual and his/her personal life | |
| Demographic characteristics | ||
| Marital status | 40% | |
| Health literacy - one’s ability to understand the nature of vasculitis and prescribed treatments | 76% | |
| Occupation - job or profession | 37% | |
| Personal history and biography | ||
| Personal and family circumstances - specific events in one’s personal life | 37% | |
| Roles and responsibilities in one’s family (dependants to look after, etc.) | 40% | |
| Financial situation | 48% | |
| p2 | One’s experience of vasculitis | |
| General well-being (how well do you feel overall compared to how you were before the diagnosis of vasculitis) | 67% | |
| Mental / psychological well-being | 35% | |
| Feelings / emotions /mood related to vasculitis or its treatments | 68% | |
| Thoughts and beliefs related to vasculitis or its treatments | 93% | |
| Change of potential and goals | 61% | |
| p3 | Personality, habits and patterns of behavior | |
| Personality | 73% | |
| Habits | 81% | |
| Lifestyle, interests and hobbies before diagnosis | 68% | |
| Health-related habits (tendency to look after oneself, compliance with treatments, interest in own health) | 72% | |
| Adaptation to chronic illness | 75% |
A greater proportion of participants from the USA compared to the UK reported financial situation and healthcare system issues, such as access to medications, as relevant to their health (38% and 60% versus 19% and 44%, respectively). Similarly, more American participants reported positive effects of having vasculitis (personal factors -> experience of vasculitis), including increased appreciation of life, increased sensitivity to other people’s misfortunes, and increasing activities related to helping others (72%, 73%, and 64% versus 42%, 36%, and 39%, respectively). In contrast, the effect of the vasculitis on patients’ ability to travel (activities and participation -> recreation and leisurely activities) was reported as at least moderately relevant by 61% of participants from the UK compared to 29% of Americans.
Similar analyses by type of AAV demonstrated expected trends: more respondents with EGPA reporting effect of their disease on the heart and lungs and a prominent impact of air quality (consistent with asthma being a central feature of the disease); subjects with MPA had the most difficulty with blood pressure and kidneys. Furthermore, subjects with MPA seemed to be most affected by the various aspects of their psychosocial functions, including mental functions (motivation, appetite, concentration, and emotions); domestic, community, and social life (activities and participation), and interpersonal interactions and relationships (environmental factors.)
DISCUSSION
In this analysis mainly limited by the narrow geographic sampling, the ICF was a useful framework for describing aspects of AAV relevant to patients. Comparisons of prioritized items by country of residence and by diagnosis revealed that while the majority of differences between the 3 different forms of AAV were in keeping with the expected differences in the frequencies of specific organ involvement, most variability between the 2 participating countries was seen in the importance of various contextual factors. As the role of contextual factors in interpreting outcome measures continues to be clarified (14), considering some key contextual factors will deepen the ability to fully assess the impact of AAV and other diseases.
An earlier ICF-based analysis of the current OMERACT Core Set for AAV (11) revealed that it does not measure the whole spectrum of limitations in activities and participation prioritized by patients in this study, and covers only a small number of environmental and personal factors, likely because contextual factors have only recently become recognized as relevant for interpreting the measured outcomes (14). The areas under-sampled by the core set were also found to be less important to vasculitis clinical experts in a recent ICF-based analysis (15); this is not surprising, given that the composite tools that constitute the current OMERACT Core Set for AAV were designed by the same clinical experts. Notably, the few contextual factors that clinicians did identify as important differ from those prioritized by patients: clinicians focus on hard “objective” factors such as demographics, comorbidities, availability of health services, and social support (15), while the range of factors identified by patients is much broader and dominated by the more subjective factors such as attitudes of other people and patients’ own reactions and thoughts. In contrast, several of the more severe disease manifestations of AAV, such as lung and kidney involvement, visual impairment, and hearing loss, that are prioritized by the majority of vasculitis clinical experts (15) were rated as at least “moderately relevant” by <50% of patient participants, likely reflecting the frequency of these manifestations in the cohort. Along with similar findings in other studies (16), the observed differences in perspectives of patients and clinicians support OMERACT’s recommendation to include perspectives of different stakeholders, including patients, into the process of development of core sets of domains and outcome measures (1). To comply with these standards, the OMERACT Vasculitis Working Group is working to update the current OMERACT core set for AAV.
Supplementary Material
Table 2:
International Classification of Function (ICF) Environmental Factors identified as at least moderately relevant by at least 30% of participants
| ICF Code# |
ICF Category description | % ranking category as ≥ moderately relevant |
|---|---|---|
| e1 | Products and technology | |
| e1101 | Medications | 75% |
| > Unwanted side effects of medications | 64% | |
| >Unwanted side effects of prednisone specifically | 73% | |
| e130 | Products and technology for education (books, computer hardware and software, internet) | 42% |
| e1650 | Financial assets (money), tangible assets (houses, land, owned goods and technology), other assets | 31% |
| e3 | Support and relationships | |
| e310 | Immediate family | 79% |
| e320 | Friends | 72% |
| e325 | Acquaintances, peers, colleagues, neighbours, community members | 51% |
| e350 | Health professionals | 82% |
| nc-cf* | Support from others in a similar situation - e.g. other patients with vasculitis, patients with other chronic conditions, support groups | 63% |
| eE4 | Attitudes (of family members, friends, colleagues, health professionals, etc.) | |
| e410 | Attitudes of immediate family members | 73% |
| e420 | Attitudes of friends | 61% |
| e425 | Attitudes of acquaintances, peers, colleagues, neighbours and community members | 47% |
| e450 | Attitudes of health professionals | 73% |
| Other factors that may affect health | ||
| e260 | Air quality | 32% |
| e580 | Health-related services, systems and policies including access to medical services and medications, health insurance | 55% |
| nc-cf* | Information about disease and drugs, either provided by health care providers or researched in journals, books, or internet | 71% |
| nc-cf * | Living arrangement - private house or apartment, amount of external noise, proximity to traffic, to parks or exercise facilities, etc. | 50% |
| nc-cf * | Stress - physical or psychological stress from any source | 49% |
| nc-cf * | Clinical research in teaching hospitals | 33% |
ICF code is a numeric identifier that uniquely identifies each ICF category; it starts with a letter that denotes the ICF component (e for environmental factors) and is followed by numbers that specify progressively more detailed categories; for example, category e310 (family members) is a sub-category of the broader category e3 (support and relationships).
Not covered - contextual factors; category not covered by the current ICF classification.
Acknowledgements:
The authors appreciate the assistance and advice of Susan Humphrey-Murto, Joyce Kullman, John Mills, Susan Mills, and C. Douglas Smith.
Sources of support:
The Vasculitis Clinical Research Consortium (VCRC) Outcome Measures in Rheumatology Vasculitis Working Group was supported by the Vasculitis Clinical Research Consortium, which received support from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (grants U54-AR-057319 and U01-AR-51874 04), the National Center for Research Resources (grant U54-RR-019497), the National Center for Advancing Translational Science, the Office of Rare Diseases Research, each part of the United States National Institutes of Health (NIH). The VCRC is a member of the NIH Rare Diseases Clinical Research Network. The VCRC also received a Pilot award from the Patient-Centered Outcomes Research Institute. Dr. Milman’s work was supported by a postgraduate Rheumatology Fellowship award given jointly by UCB Pharma, the Canadian Rheumatology Association, and The Arthritis Society, and a Fellowship from the Ottawa Hospital Department of Medicine. This work was further supported by a Vasculitis Foundation Research Grant.
Footnotes
Conflict of interest: none
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