Real-world effectiveness and rheumatologist satisfaction with secukinumab in the treatment of patients with axial spondyloarthritis.

OBJECTIVES: To assess the effectiveness of secukinumab in patients with axSpA treated in routine clinical settings in 5 European countries. METHODS: Retrospective analysis of a cross-sectional survey to assess real-world effectiveness of secukinumab in the management of axSpA and rheumatologist satisfaction with treatment in France, Germany, Italy, Spain and the UK from March to December 2018. Outcomes collected included patient demographics, clinical characteristics and rheumatologist- and patient-reported satisfaction with secukinumab treatment. RESULTS: Five hundred thirty-five patients receiving secukinumab for more than 4 months were assessed, 359 of whom were diagnosed with AS and 178 with nr-axSpA. Rheumatologist assessment of disease status at treatment initiation indicated that 39 (7.3%) had stable/improving disease. Secukinumab treatment for 4 months or longer resulted in 515 (95.9%) patients judged as stable/improving. Treatment was associated with benefits from initiation to assessment in terms of BASDAI (6.2 vs 2.8), 44-joint count score (9.7 vs 6.6), rheumatologist global VAS score (56.9 vs 23.0) and patient global VAS scores (64.4 vs 25.5). These benefits for key clinical outcomes were sustained for periods of 12 months or longer. Patient-reported outcomes on health status using EQ-5D, global functioning using the ASAS health index and overall work impairment via WPAI were sustained over the treatment period, while patient and rheumatologist satisfaction with secukinumab treatment remained very high at 80.2 and 91.2%, respectively. CONCLUSION: Consistent benefits across multiple clinical and patient-reported outcomes were seen with secukinumab treatment in patients with AS and nr-axSpA treated in routine clinical settings across five European countries. Key Points • In routine clinical settings across five European countries, secukinumab treatment resulted in improvements in a wide range of clinical outcomes including physician-reported disease severity, disease status, pain, BASDAI, 44-joint count score and global VAS scores. • Key clinical and patient reported outcomes were sustained for a 12-month period or longer with secukinumab treatment. • Rheumatologist- and patient-reported treatment satisfaction was high with secukinumab.

Evaluation of the meaningfulness of health-related quality of life improvements as assessed by the SF-36 and the EQ-5D VAS in patients with active Crohn's disease.

BACKGROUND: Crohn's disease (CD) is a chronic inflammatory illness characterized by episodic abdominal pain, diarrhoea, fever, bleeding and obstruction. While the Crohn's Disease Activity Index (CDAI) remains the most commonly accepted measure for assessing the disease status in clinical trials, patient-reported outcome (PRO) instruments are being utilized more frequently to provide information about health-related quality of life (HRQOL). To facilitate interpretation of results, it is common to identify a meaningful unit of PRO score change, such as a minimal clinically important difference (MCID). AIM: To define and apply MCID estimates for the SF-36 and EuroQol-5D visual analogue scale (EQ-5D VAS) for use in CD treatment evaluation. METHODS: Data from two phase III randomized controlled trials of certolizumab pegol were utilized. MCID estimates were computed from one trial using anchor-based and distribution-based methods. These estimates were applied to data from the other trial. RESULTS: SF-36 PCS and MCS MCID estimates ranged from 1.6 to 7.0 and 2.3 to 8.7 respectively, depending on approach. EQ-5D VAS MCID estimates ranged from 4.2 to 14.8. CONCLUSIONS: For the first time, the MCID values provided interpretation guidelines for PRO results in CD. This research demonstrates that patients treated with certolizumab pegol benefit from meaningful and sustained HRQOL improvements.

Development of a revised version of the Women's Health Questionnaire.

OBJECTIVES: The Women's Health Questionnaire (WHQ) includes 36 items assessing nine domains of physical and emotional experiences of mid-aged women. The primary aim of the current research was to examine the psychometric properties of the WHQ across linguistic versions in view of the increased need for reliable health-related quality of life (HRQL) measures in multinational studies. METHODS: In this paper, we examine the hypothesized structure of the questionnaire in a UK sample, to develop and verify a revised model to be used in multicenter, international studies. RESULTS AND CONCLUSIONS: Content analysis and evaluation of missing data led to exclusion of 'Menstrual symptoms' and 'Sexual behavior' domains, retaining these as optional modules of the core questionnaire. Additionally, item 13 was excluded because it does not investigate the same concepts as other domains and the deletion of five additional items appeared to improve the questionnaire's factor structure. The revised WHQ comprises 23 items, investigating six domains. The cross-sectional psychometric properties of the 23-item WHQ were good and better than those of the 36-item version. The 23-item WHQ was assessed with multinational data, to evaluate cross-cultural equivalence of linguistically adapted versions. In addition, its reproducibility and responsiveness need to be documented.

A comparative review of generic quality-of-life instruments.

The assessment of health-related quality of life (HR-QOL) is an essential element of healthcare evaluation. Hundreds of generic and specific HR-QOL instruments have been developed. Generic HR-QOL instruments are designed to be applicable across a wide range of populations and interventions. Specific HR-QOL measures are designed to be relevant to particular interventions or in certain subpopulations (e.g. individuals with rheumatoid arthritis). This review examines 7 generic HR-QOL instruments: (i) the Medical Outcomes Study 36-Item Short Form (SF-36) health survey; (ii) the Nottingham Health Profile (NHP); (iii) the Sickness Impact Profile (SIP); (iv) the Dartmouth Primary care Cooperative Information Project (COOP) Charts; (v) the Quality of Well-Being (QWB) Scale; (vi) the Health Utilities Index (HUI); and (vii) the EuroQol Instrument (EQ-5D). These instruments were selected because they are commonly used and/or cited in the English language literature. The 6 characteristics of an instrument addressed by this review are: (i) conceptual and measurement model; (ii) reliability; (iii) validity; (iv) respondent and administrative burden; (v) alternative forms; and (vi) cultural and language adaptations. Of the instruments reviewed, the SF-36 health survey is the most commonly used HR-QOL measure. It was developed as a short-form measure of functioning and well-being in the Medical Outcomes Study. The Dartmouth COOP Charts were designed to be used in everyday clinical practice to provide immediate feedback to clinicians about the health status of their patients. The NHP was developed to reflect lay rather than professional perceptions of health. The SIP was constructed as a measure of sickness in relation to impact on behaviour. The QWB, HUI and EQ-5D are preference-based measures designed to summarise HR-QOL in a single number ranging from 0 to 1. We found that there are no uniformly 'worst' or 'best' performing instruments. The decision to use one over another, to use a combination of 2 or more, to use a profile and/or a preference-based measure or to use a generic measure along with a targeted measure will be driven by the purpose of the measurement. In addition, the choice will depend on a variety of factors including the characteristics of the population (e.g. age, health status, language/culture) and the environment in which the measurement is undertaken (e.g. clinical trial, routine physician visit). We provide our summary of the level of evidence in the literature regarding each instrument's characteristics based on the review criteria. The potential user of these instruments should base their instrument selection decision on the characteristics that are most relevant to their particular HR-QOL measurement needs.