Measurement of the impact of giant cell arteritis and treatment with glucocorticoids on health-related quality of life

Abstract

Giant cell arteritis (GCA) is the most common type of systemic vasculitis and predominantly affects women over the age of 50 years. Clinical presentation includes cranial and ocular involvement, joint and systemic features. Diagnosis is confirmed by positive temporal artery biopsy or ultrasound or large vessel vasculitis on positron emission tomography. Treatment includes high-dose glucocorticoids (GC) and immunosuppressants. Health-related quality of life (HRQoL) can be impacted by pain, fatigue, and sight loss; glucocorticoid therapy adds further physical, psychological, and social burdens. Patient-reported outcome measures (PROMs) aim to capture the impact of disease from the patient’s perspective. A range of PROMs have been used to highlight the impact of GCA. The generic 36-Item Short Form Health Survey (SF-36) has demonstrated the effectiveness of novel treatments including Tocilizumab in clinical trials, the symptom-specific Hospital Anxiety and Depression scale has demonstrated 50% of people with GCA suffer with anxiety and a third depression, and condition-specific (GCA PRO) and treatment-specific (Steroid PRO) PROMs have been developed and validated in people with GCA for clinical practice and trials. Shared decision-making (SDM) is essential in GCA management, ensuring patients understand treatment options and decisions reflect their preferences and “what matters to them.” PROMs support SDM and enhance the patient-clinician dialogue. Early evidence from use of the GCAPRO demonstrated that PROMs have helped patients explain their symptoms, providing clinicians with clearer insights into the patient’s health. There is a growing role for electronic PROMs (E-PROMs) to support patient and clinician communication and facilitate effective clinical triage. Considerations about E-PROMs include potential for digital exclusion in older patients, data governance with online apps, and clinical safety (i.e., for people reporting active disease). To fully implement E-PROMS in clinical practice and gain the maximum benefit, full integration into intuitive clinical and patient systems will be required.

Plain language summary

Measuring the impact of giant cell arteritis and steroids from the patients’ perspective

Giant cell arteritis (GCA) is the most common type of vasculitis. It is most common in women and those over 50 years old. GCA causes the blood vessels of the head and neck to become inflamed and narrowed. People can have headaches and pain and it can affect chewing and cause loss of vision. Some people can also get internal inflammation of the blood vessels around the aorta. A type of steroid called glucocorticoids are used to treat GCA. GCA and treatment with steroids can cause side effects and reduce quality of life. Fifty percent of people can feel anxious and a third have depressive symptoms. Patient reported outcome measures (PROMs) are questionnaires that ask people about their quality of life. They have been used in GCA in clinical trials to show the benefit of new treatments that do not have the side effects of steroids. They can also be used in clinic to improve understanding between patients and doctors and nurses. They do this by raising awareness of what is important to patients–e.g fatigue or pain or how the condition is affecting peoples’ life. A plan can then be made together to help with this. Electronic PROM questionnaires are now being used to help patients and their clinical team. These can help communication and efficiency. It is however important to think about people who may not be confident with digital questionnaires, e.g because of visual problems or older age.

Introduction to giant cell arteritis (presentation, incidence, and treatment)

Giant cell arteritis (GCA) is a chronic granulomatous vasculitis affecting large- and medium-sized vessels; most commonly the temporal artery but also affecting the aorta and its major branches. ¹ It is the most common type of primary systemic vasculitis, with a United Kingdom (UK) incidence of approximately 2.2 per 10,000 person-years. ² See Figure 1 for the clinical manifestations of GCA.

Figure 1.

Clinical manifestations of giant cell arteritis.

Symptoms of GCA may include headaches, scalp tenderness, swelling and tenderness of the temporal artery, jaw claudication, and visual loss. There may also be non-specific effects of systemic inflammation such as anemia, fever, weight loss, and malaise. ³ GCA predominantly affects individuals aged 50 and over (average age of diagnosis ~79 years), with a marked female predominance and highest incidence in Northern European populations.^4–6 GCA can present as one of two broad recognizable patterns: cranial-GCA (c-GCA) and large vessel-GCA (LV-GCA); some patients have features of both. ⁷ Imaging evidence of large-vessel involvement is present in over 80% of patients with GCA, although limb claudication is less common. ⁸ GCA is also closely associated with polymyalgia rheumatica (PMR), an inflammatory rheumatic disease, causing pain and stiffness in the shoulders and pelvic girdle; around 40%–60% of patients with GCA also have PMR.^5,9 The 2022 American College for Rheumatology (ACR) classification criteria for GCA, developed primarily for clinical trials, include positive investigation results such as raised erythrocyte sedimentation rate or C-reactive protein, positive temporal artery biopsy or “halo sign” on temporal artery ultrasound, and positron-emission tomography activity throughout the aorta. ¹⁰ However, no single definitive test exists, and clinical diagnosis requires interpretation of test results in the light of the overall clinical picture.

The pathophysiology of GCA is a multi-step cascade, resulting in vessel inflammation. ¹¹ The response to vascular injury includes neointima hyperplasia, which may result in vascular stenosis or occlusion and downstream tissue ischemia.^11,12 Involvement of the arterial supply to eye or brain can cause blindness or stroke. Scalp or tongue necrosis can also occur. Aortitis causes structural damage to the aorta, and this can be complicated by arterial dissection, aneurysm, or rupture. While the cranial complications tend to occur during periods of active inflammation, the aortic complications may occur many years later.

Newly diagnosed GCA is treated with high-dose glucocorticoids (GC), with or without adjuvant immunomodulation, to suppress vascular inflammation; after which the glucocorticoid dose is tapered to achieve the minimal dose that will maintain disease control.^13,14 The balance between over- and under-treatment is made harder by the myriad side-effects of glucocorticoids. ¹⁵

The Outcome Measurement in Rheumatology (OMERACT) non-profit organization is an international initiative that builds consensus on which patient and disease-specific areas should be measured (domains), and which measurement instruments should be used, in different clinical trial contexts. Global Working Groups comprising patient research partners, clinicians, methodologists work together using rigorous methodological steps to develop Core Outcome Sets outlining domains and core-sets. There are two OMERACT working groups (WG) that have explored the impact of GCA and its treatment from the patient’s perspective: the OMERACT Glucocorticoid-Related Adverse Events Working Group and the Large Vessel Vasculitis Working Group.^16,17 The OMERACT initiative classifies outcome measures into Core Areas of Pathophysiological Manifestations and Life Impact. Here, we focus on measurement of (life) impact of GCA and its treatment.

The impact of GCA on health-related quality of life

Health-related quality of life (HRQoL) is a complex and subjective term with various definitions. ¹⁸ It is defined by the WHO as “a person’s goals, expectations, standards, or concerns about their overall health and health-related domains.” ¹⁹

GCA has a significant and multifaceted impact on HRQoL, caused by the disease burden itself and the consequences of long-term treatment. Patients frequently report fatigue, pain, and weight gain among other systemic symptoms, significantly impairing physical function and impacting sense of self. ²⁰ Cranial manifestations such as headache and jaw claudication can impact daily living, whilst visual impairment can threaten independence and participation within society. Sight loss has the greatest correlation to psychological distress in GCA, with sight-related symptoms yielding higher disease-impact scores. ²¹ Beyond disease-related symptoms, the prolonged use of high-dose glucocorticoids imposes a considerable burden on HRQoL; this impact is explored in more detail below.

Psychological and social dimensions of HRQoL are also affected with increased levels of anxiety, depression, and social isolation. ²² Patients must live with the uncertainty of when or if they will relapse or develop more severe complications. GCA patients can be an older demographic at onset compared with other rheumatic diseases, and factors leading to increased frailty, for example multi-morbidity and polypharmacy, are often relevant. ²³ Conversely, some patients can be of working age or in active retirement and be very well prior to developing GCA; patients in this situation have the shock of a new diagnosis and dealing with a broad range of new impacts on HRQoL due to the disease and its treatment. ²⁰ Capturing patient’s perspectives on the impact of GCA is important for clinical care and trials. Qualitative and quantitative studies have examined the impact of GCA on patients’ HRQoL using a range of patient-reported outcome measures (PROMs; see Table 1).

Table 1.

Measurement of health-related quality of life in GCA: overview of qualitative and quantitative studies.

Title and type of study	Author, population, year	Outcome measures or analysis	Summary of results
Visual function and quality of life among patients with giant cell (temporal) arteritis.	Kupersmith et al., 21 New York, 2001	• SF36	• SF-36 baseline scores did not correlate with the presence or absence of visual loss
Quantitative, randomized placebo-controlled study (methotrexate vs placebo)		• ADVS	• Mental distress was worse in patients with vision loss at baseline
		• Survey (N = 20)
Domains of health-related quality of life important to patients with giant cell arteritis.	Hellmann et al., 24 American, France, Spain, 2003	• Focus groups to identify domains (e.g., losing sight in both eyes permanently, being able to shower, shaving)	• Vision ranked as number 1 QoL concern for patients
Mixed methods study, qualitative followed by quantitative questionnaires		• IRQ survey (N = 214)	• Maintaining control over body and movement ranked highly
What is the impact of giant cell arteritis on patients’ lives?	Liddle et al., 25 2017, UK primary care and via PMRGCAUK	• Structured telephone interviews (N = 24)	• GCA described as an ongoing experience over time:
Qualitative study.			• Symptoms (pain, fatigue, sight loss)
			• The impact of symptom duration
			• The impact of GCA on life circumstances (e.g., impact on relationships, hobbies, finances)
Quality of life of patients treated for giant cell arteritis: a case-control study	Jobard et al., 26 France, 2017	• Individual semi-structured telephone interviews	• Skin fragility most common complication of GCs
Mixed methods, qualitative phone interview & quantitative questionnaires		• Questionnaire including 7 QoL domains from Hellmann’s study	• 56.7% of patients felt improved after steroid withdrawal
		• SF-36 (N = 30 patients, 60 controls)	• SF-36—patients did not demonstrate statistical difference from controls
			• Vitality score from the SF-36 was lower in patients who gained weight.
Health-related quality of life in patients with giant cell arteritis treated with tocilizumab in a phase III randomized controlled trial (GiACTA)	Strand et al., 27 2019, North America, Europe.	• SF-36	• Lower FACIT-fatigue scores, SF-36 PCS, MCS in all GCA groups compared with population.
Quantitative study		• VAS	• Significant improvement in physical and general health domains, FACIT-fatigue scores in tocilizumab + Pred26 treated groups compared with pred alone.
		• FACIT-Fatigue score (N = 251).	• In Toci + Pred 26 group HRQoL improved to at least population controls at week 52.
Vision-related and health-related quality of life in patients with giant cell arteritis	Ni Mhealoid et al., 28 Ireland, 2020	• SF-12	• Mental disability significantly worsened by GCA-related ophthalmic impairment
Quantitative study		• 30-item GDS (N = 116)	• Altered QoL scores with GCA diagnosis, particularly in >80s
Impact of giant cell arteritis and its treatment on the patient’s quality of life.	De Boysson et al., 29 France, 2021	• SF-12	• Moderate-severe physical disability in 46%
Quantitative study		• 30-item GDS (N = 90)	• Physical disability associated with reduced muscle mass/strength, walking difficulties, and age >80
			• Moderate-severe mental disability in 33%
			• Mental disability associated with depressive mood, attribution to glucocorticoids, and immunosuppressants.
Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome.	Robson et al., 20 UK and Australia, 2021	• Structured interviews and cognitive interviews (N = 36)	• 39 individual themes within 5 domains:
Qualitative study			• Physical symptoms
			• Activities of daily living and functioning
			• Participation
			• Psychological impact
			• Impact on sense of self and health perception
Patient-reported outcomes provide evidence for increased depressive symptoms and increased mental impairment in giant cell arteritis.	Froehlich et al., 30 Germany, 2023	• SF-36	• 40% evidence of major depression disorder
Cross-sectional survey		• PHQ-9 Physician and patient VAS (N = 100)	• Patient VAS correlated with PHQ-9 and SF-36
			• Physician VAS only correlated with physical categories of SF-36.
Validation of a patient-reported outcome measure for giant cell arteritis.	Ndosi et al., 31 UK, 2024	• Candidate items for GCA-PRO	• Final scale structure of GCA PRO (30-item with 4 domains: acute symptoms, activities of daily living, psychological impact, participation).
Cross-sectional survey		• EQ-5D-5L
		• ICECAP-A
		• CAT-PROM5
		• Self-report disease activity (N = 428)
Anxiety and depression in patients with giant cell arteritis.	Martins-Martinho et al., 22 Portugal, 2024	• SF-36	• GCA patients: 48.6% anxiety and 33.3% depression
Cross-sectional survey		• HADS (N = 72 patients and 288 controls)	• Glucocorticoid therapy was a predictor of high anxiety scores (OR 10.4)
			• Older age was a predictor of high depression scores (OR 1.2)

ADVS, Activities of Daily Vision Scale; EQ-5D-5L, EuroQol-5 Dimension 5 Level; FACIT, Functional Assessment of Chronic Illness Therapy; GCs, glucocorticoids; GCA, giant cell arteritis; GDS, geriatric depression scale; HADS, Hospital Anxiety and Depression Scale; HRQoL, Health-related quality of life; IRQ, Importance Rating Questionnaires; OR, odds ratio; PHQ-9, Patient Health Questionnaire; PRO, patient-reported outcome; QoL, quality of life; VAS, visual analogue scale.

Impact of glucocorticoids on HRQoL

The mainstay of treatment for GCA remains prolonged courses of high-dose glucocorticoids over at least 1–2 years, this can be longer if patients suffer relapses, which can occur in up to 50% of cases.^13,14 Glucocorticoids play a key role in controlling inflammation, reducing pain, and systemic symptoms, and can prevent irreversible damage from active GCA, but they do impact HRQoL.^15,32–38 Patients report psychological impact including mood disturbance, sleep, anxiety, irritation, paranoid thoughts, and difficulties in thinking clearly.^33,36–38 Changes in appearance and weight gain from glucocorticoids can impact self-esteem and choices with clothing and activities and lead to unwanted comments from friends and family.^35,39 Mood changes and irritability experienced with glucocorticoid therapy can also affect relationships with friends, family, and work colleagues.^15,33,35

Patients report positive aspects to treatment with glucocorticoids: they are effective in controlling disease at diagnosis and during flares, ³² and there is a sense of relief in starting effective treatment. ³⁵ In a survey of patients taking glucocorticoids (n = 150) by Black et al. ³² over half of the cohort felt that the benefits of glucocorticoids outweighed the adverse effects. Healthcare professionals can often be cautious about prescribing glucocorticoids, and this can put pressure on interactions between patients and their clinicians. ³⁴ There is a balance between effective treatment with GCs to control symptoms and reduce damage, and adverse effects experienced, including impact on HRQoL.

Patient-reported outcome measures

PROMs are defined by the Food and Drug Administration (FDA) as “a report that comes directly from the patient . . . about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.” ⁴⁰ Unlike biomarkers or clinician-reported outcomes, PROMs capture the “lived experience” of the condition by providing unique insights into the unobservable, subjective burden of disease including pain intensity, fatigue, and psychological distress.^41–43 PROMs are designed for use in different contexts: (i) as validated outcome measurements in clinical trials and observational studies to capture the impact from the patient’s perspective, and (ii) as tools to improve communication between patients and clinicians. The rigorous development of all types of measures is critical to ensure they accurately reflect the constructs they intend to measure and are sensitive enough to detect change in clinical trials and are meaningful in practice. ⁴⁴

There are different types of PROMs for use in different contexts, often more than one type of PROM is chosen as they can be complementary to each other. Generic instruments assess global health concepts, and allow for comparisons across different populations and diseases, for example the EuroQol-5 Dimension 5 Level (EQ-5D-5L) ⁴⁵ and the 36-item Short-form survey (SF36) questionnaires. ⁴⁶ PROMIS^® (Patient-Reported Outcomes Measurement Information System) is a set of generic measures developed in the United States of America (USA) that can be used to measure physical, mental, and social health in people with different conditions and the general population. ⁴⁷ PROMIS also uses Computer Adapted testing, whereby the test adapts to patients’ ability (or severity) level, using targeted questions to reduce responder burden and increase precision. ⁴⁴ Condition-specific instruments are developed and validated with people with a specific disease, to maximize relevance to patients with that condition, for example the condition-specific GCA PRO. ³¹ Greater relevance and specificity can improve measurement properties—for example by increasing sensitivity to change in the context of a clinical trial ³¹ and increase acceptability to patients. Symptom-specific PROMs can also be used across diseases or populations as generic measures, for example the Functional Assessment of Chronic Illness Therapy—Fatigue Scale (FACIT-Fatigue). ⁴⁸ An overview of the different types of PROMs used in GCA, and contexts of use, is shown in Figure 2. Table 1 outlines the key studies on HRQoL in GCA, and the outcome measures used.

Figure 2.

Patient-reported outcome measures to capture the impact of giant cell arteritis and glucocorticoids.

Methodologically, PROMs can be used in a range of different types of research studies. Within epidemiological studies, PROMs can be used to measure the disease burden of a condition on HRQoL and identify the prevalence of specific patient-reported symptoms. An example of this is the 2024 study by Martins-Martinho et al., ²² which demonstrated that almost half of GCA patients have high levels of anxiety and a third have high levels of depression, through the use of the Hospital Anxiety and Depression Scale. In contrast, within interventional clinical trials, PROMs can be used to evaluate treatment effects by demonstrating a statistical difference in HRQoL between different arms of a treatment trial. For example, the GiACTA trial used SF-36 to demonstrate a significant improvement in HRQoL for patients receiving Tocilizumab compared to the placebo arm. ²⁷ Although PROMs are usually included as secondary outcomes in GCA trials, they are increasingly scrutinized by regulatory bodies such as the European Medicines Agency and the US FDA, during the approval process for novel therapies. ⁴⁰

Development of PROMs

The development and validation of robust PROMs is a complex and iterative process, involving a mixed-methods process. The process is underpinned by patient involvement at all stages and overseen by steering committee of researchers, methodologists, patient partners, and clinicians. To ensure global applicability and regulatory acceptance, best practice entails a hybrid approach, informed by the FDA Guidance for Industry ⁴⁰ to govern the qualitative development phase (ensuring content validity), and the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) taxonomy ⁴⁹ to guide the quantitative validation phase.

Item generation and content validity

To guide the development of PROMs, a conceptual framework is first developed, based on the literature review, patient partners, and stakeholder input to define the scope of the PROM. Underpinning qualitative work includes patients with a range of disease features and demographics to explore the impact of the condition under study from their perspective. These qualitative themes are then operationalized into candidate items (questions) through a collaborative process between researchers and patient partners. To establish content validity, in-depth cognitive interviews with patients are conducted to evaluate the relevance, comprehensiveness, and comprehensibility of the items, ensuring they are interpreted as intended and reducing respondent burden (duplication/redundancy). Early stage linguistic appraisal evaluates the structural and conceptual equivalence of the items to ensure the PROM is amenable to future cross-cultural adaptation and international translation. ²

Psychometric validation and item reduction

Following item generation, a large-scale validation survey is conducted to evaluate the structural validity and measurement properties of the PROM. This phase aims to produce a parsimonious, feasible, and clinically acceptable questionnaire for patients to use. Modern psychometric techniques, specifically Rasch analysis ¹ are used to evaluate how well items adhere to a mathematical model of fundamental measurement. Items can be removed due to significant deviation from the Rasch model (indicating the item does not contribute to a unidimensional construct), item redundancy (identified through local dependency), or ceiling and/or floor effects (where a lack of item difficulty prevents the detection of variance at the extremes of a construct). ²

Evaluation of measurement properties

Validity is the degree to which a PROM instrument measures what it is intending to measure. In accordance with COSMIN guidelines, the final scale is assessed for (i) construct validity, including convergent validity (testing hypotheses regarding correlations with other validated measures) and discriminative validity (testing the PROM against known groups), (ii) reliability including internal consistency (item interrelatedness) and test–retest (temporal stability), and (iii) responsiveness, that is, the ability to detect clinically meaningful change over time, which is essential for its utility in longitudinal research. ³

Development and validation of the GCAPRO

The OMERACT Large Vessel Vasculitis group first highlighted the need for a disease-specific PROM to measure the impact of GCA in their report from OMERACT 2016, and this was developed over subsequent meetings.^50,51 This initiative was taken forward by a collaboration of researchers, patient partners, and methodologists from the UK and Australia who formed a steering committee and developed a conceptual framework to develop a disease-specific PROM for GCA.

Qualitative phase and item generation

Underpinning qualitative interviews were held with patients with clinician-confirmed GCA, recruited from rheumatology and ophthalmology departments in the UK and Australia (n = 36). Themes identified during the qualitative interviews were recast into a long-list of 69 candidate GCA-PRO items, these were then piloted and refined with input from patient partners, cognitive interviews with patients from the UK and Australia, and a linguistic translation assessment to reduce to a list of 40 items.

Quantitative validation

The long list of candidate items was then reduced further by use of a large-scale validation study in the UK. Patients with clinician-confirmed GCA were recruited from 38 UK National Health Service rheumatology and ophthalmology departments (n = 428).^50,51

Patients completed the original 40 candidate GCA-PRO items, the EQ-5D-5L, CAT-PROM5, and self-report of disease activity. Rasch and factor analysis were used to determine internal validity and factor structure. A final PRO consisting of 30 items in 4 domains was confirmed: Acute symptoms (8 items), Activities of daily living (7 items), Psychological (7 items), and Participation (8 items). See Figure 3 for domain and individual items of the GCA PRO. Measurement properties including convergent validity with EQ-5D-5L, known groups validity, and test-retest reliability were demonstrated. See Figure 4 for an overview of the development stages of the GCA PRO. The GCA PRO has been translated into German, Italian, and French and validation studies in these countries are currently underway in clinical trials and practice.

Figure 3.

GCA PRO domains and items.

GCA, giant cell arteritis; PRO, patient-reported outcome.

Figure 4.

(a) Qualitative development of the GCA PRO. (b) Validation of the GCAPRO scale structure and measurement properties.

GCA, giant cell arteritis; PRO, patient-reported outcome.

Development and validation of the Steroid PRO

The OMERACT GC Impact WG is an international collaboration including patients, methodologists, and researchers with an interest in the measurement of the impact of steroids within clinical trials. The WG has completed a package of work included: a systematic review of qualitative and quantitative studies of the impact of GCs, ¹⁵ a patient and physician Delphi of the key domains to measure in clinical trials in relation to GC impact, ⁵² and the final GC Impact core domain set—which defines the key domains to be measured in clinical trials where patients are treated with steroids.^36,53 A series of international qualitative studies including people with GCA, and ANCA-associated vasculitis, rheumatoid arthritis, systemic lupus erythematosus, and idiopathic inflammatory myositis were undertaken to explore the multiple patient impacts from GCs.^34,35,37,54 The group identified that a data-driven PROM to capture the impact of systemic GC use was needed. ⁵⁵ Two previous systematic reviews by the European Alliance for Associations for Rheumatology (EULAR)^56,57 also highlighted the need to identify adverse effects of GCs in a standardized way.

Qualitative phase and item generation

An international steering committee, including patients, researchers, and methodologists from the UK, USA, and Australia oversaw the development of the Steroid PRO, informed by FDA guidance. Bridgewater et al. ³³ interviewed 60 patients with inflammatory conditions including GCA from the UK, USA, and Australia to explore the impact of GC therapy on HRQoL. Individual candidate questionnaire items were developed and refined via piloting with patient partners and cognitive interviewing. ⁵⁸

Quantitative validation

Scale structure and psychometric properties of the Steroid PRO were then determined through a cross-sectional international validation study (N = 946, including 128 with GCA). ³⁵ The final version of the Steroid PRO consisted of 15 items and 4 domains (social impact, impact on appearance, psychological impact, and treatment concerns), see Figure 5.

Figure 5.

Steroid PRO domains and items.

PRO, patient-reported outcome.

The development phases are shown in Figure 6. The Steroid PRO is a generic measurement tool to capture the impact of glucocorticoids from the patient’s perspective for use in clinical practice and trials including GCA. The Steroid PRO has since been shown to be relevant and acceptable for use in inflammatory respiratory, dermatological, and gastroenterological conditions by patients and clinicians in the UK and USA. ⁵⁹ Its use in clinical practice and observational studies is currently being evaluated in Australia and Germany.

Figure 6.

(a) Qualitative development of the Steroid PRO. (b) Validation of the steroid PRO scale structure and measurement properties.

PRO, patient-reported outcome.

Use of PROMs in clinical practice

The EULAR and British Society of Rheumatology GCA Management guidelines and Treat-to-target recommendations have all highlighted the need for shared decision-making between patients with GCA, and their clinicians.^13,14

Shared decision-making (SDM) includes ensuring patients with GCA understand the care, treatment, and support options available, and the risks, benefits, and consequences of different options. Deciding on next steps can then be based on good quality information and personal preferences to provide personalized care—where people have choice over their care based on “what matters to them” and their individual strengths and weaknesses. ⁶⁰

PROMs can be used to promote communication between patients and their clinicians and therefore support patient-centered care and SDM. A realist evaluation of how PROMs can work in practice to support SDM identified two theories about how PROMs can achieve this: (i) Theory 1 describes how patients completing PROMs, go through a process of reflection and empowerment, which allows them to raise issues with clinicians that they might not normally feel able to discuss, and (ii) Theory 2 describes how PROM (total or domain scores) can raise clinicians’ awareness of patients’ problems that patients ⁶¹ might not otherwise raise in their clinical appointments, ⁶² bridging the perspectives of patient and clinician within the consultation but also itself influencing what is, and can be discussed within the remit of the consultation. Rather than viewing it simply as a conduit for information, it can be viewed as a relational tool, an object that can become an integral part of the consultation.

A pilot study of use of the GCAPRO in clinical practice in rheumatology and ophthalmology departments (n = 16 appointments) in the UK supported the suggestion that PROs can support communication and SDM in practice. Fourteen patients agreed that the GCAPRO helped them to explain their condition; in 14/16 consultations, the clinicians agreed that the GCAPRO had given them greater insights into the patients’ current state. Fifty percent of patients and clinicians agreed that the GCAPRO helped in joint decision making, although they highlighted that symptoms, blood results, and stage of illness were also taken into consideration. Further evaluation will be needed in real-world settings in relation to GCA. The GCAPRO (see Figure 3 for domains and items) may be useful to help identify specific issues and target treatments appropriately for individual patients. As an example, a high score in “Acute Symptoms” domain could prompt clinical evaluation and potential increase in immunosuppression; if high domain scores in “Activities of daily living” or “Participation” are seen, patients may benefit from occupational therapy or physiotherapy; and high scores in the “Psychological” domain, could prompt referral for talking therapies, psychological support.

Electronic patient-reported outcome measures

There has been greater interest in the use of electronic PROs (E-PROMs) in clinical practice since COVID-19, with an increase in remote monitoring and virtual consultations. Within healthcare systems, there is also a determined shift from analogue to digital practice, as a central tenet of healthcare—for example, the 2025 10 Year Health Plan for England—“Fit for the Future.” ⁶³ Where ePROMs have been fully integrated into the Electronic Patient Records (EPR), their use in triage of rheumatic conditions has improved efficiency and responsiveness of clinical departments. ⁶⁴

Digital E-PROMs could potentially be used in a variety of ways in clinical practice: (i) To improve the quality of communication and understanding between clinicians and patients with GCA in telephone or video appointments as described above, (ii) To be used by clinicians to review results alongside clinical results such as inflammatory markers, during remote triage, (iii) To be used by patient to provide them with information about their own disease state, and aid self-management. ⁶¹

Digital exclusion is one of the greatest concerns with replacement of face-to-face appointments with virtual or asynchronous use of EPROs (where clinicians review results without the patient); patients who are older, with lower levels of education or higher disability may be at greater risk of exclusion—so this is of real concern in GCA. ⁶⁵ There are also some concerns that some patients with higher digital ability may be able to gain greater access to resources, via completion of higher scoring PROMs, and that this could worsen health inequalities. ⁶⁶ Use of serial E-PROMs across time, and review in combination with other clinical results and assessments should help to personalize the interpretation of PROM scores and individualize the appropriate response to negate this effect.

Implementation of E-PROMs in clinical practice

There are multiple patient and clinician factors that are important to consider in the implementation of E-PROMs. Qualitative focus groups in inflammatory arthritis have highlighted the need to maximize the perceived benefits to patients of completing ePROs to ensure high and continued patient adherence. ⁶⁷ Training of clinicians to always discuss the completed PRO is essential, to provide feedback on the patient effort in a timely and responsive manner. Intuitive application functionality and sensible tailoring of frequency of ePROs to reduce patient burden are also key. For patients who would prefer remote or telephone monitoring, using ePROs to facilitate this rather than face-to-face consultations is also of benefit. ⁶⁷ User-centered design in ePRO applications or software, for example, the choice of graphics and exactly how data is collected and presented back to patients and their clinicians is important. ⁶⁸ The commonest reason for low levels of EPRO uptake is a lack of suitable software solutions. As well as intuitive design, the software should include automatic score calculation and displays and be fully integrated within medical records to ensure it is feasible for clinical and administrative teams to use. ⁶⁹

Use of E-PROMs in patient self-management

Patients may find that regular use of ePROMs could support them in self-management: for example, correlating their own symptom changes with changes in medication, diet, and lifestyle. If patients had access to GCAPRO individual domain scores, they could explore options depending on their results—for example physical activity, or talking therapies. Some interpretation of scores would be helpful to guide patients, and it would be essential to include safeguards and warnings to contact medical personnel for assessment of any red flags of active disease—for example new jaw claudication or ocular involvement. Their use outside of routine healthcare would need to be carefully designed, tested, and validated in terms of patient feasibility, acceptability, and safety. Patient charities may have a role in this implementation. Data security and privacy of highly personal information would be essential.

Generic symptom tracking products for use in the patient-facing “wellness” space have been developed by vendors of generic large language models—their use may become more popular with patients, and clinicians should be aware of this trend and be prepared to discuss self-tracking data during consultations. The potential benefits and considerations as described above for healthcare-based uses of PROs would also be relevant to consumer-facing apps that people might choose to use—specifically around safety and data protection.

Summary

The ongoing digital transformation of healthcare offers a clear opportunity to integrate new data on life impact, including PROMs, into clinical practice, self-management, research, and health improvement. Integrating PROMs fully into clinical systems, with intuitive and informative patient and clinician-facing platforms, is key—all stakeholders need to experience a benefit of PROMs to ensure full uptake. Within clinical trials, use of generic PROMs as validated secondary outcome measures, has demonstrated real benefits to patients’ HRQoL due to novel therapies. Use of condition and treatment-specific PROMs such as the GCA PRO and Steroid PRO in clinical trials needs further evaluation; they may demonstrate greater sensitivity to change due to their design with people who have GCA.