Rheumatoid arthritis, RA, Difficult-To-Treat Rheumatoid arthritis, D2T-RA rheumatoid arthritis
Conditions
Interventions
None listed
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: For eligibility to participate in this study, a subject must meet all of the following criteria:Male or female subjects, aged = 18 years. Diagnosis of RA by a rheumatologist according to the 1987 ACR classification criteria or the 2010 EULAR/ACR classification criteriaD2T RA according to the EULAR definition (all 3 criteria a-c below need to be present to be classified as D2T RA): 5Treatment according to European League Against Rheumatism recommendation and failure of multiple lines of anti-rheumatic treatment (=2 biological(b)/targeted synthetic (ts) Disease Modifying Anti-Rheumatic Drugs (DMARDs) with different mechanisms of action (MOA) after failing classical synthetic (cs)DMARD therapy (unless contraindicated). Signs suggestive of active/progressive disease, defined as =1 of: At least moderate disease activity (according to validated composite measures including joint counts, for example, DAS28-ESR>3.2 or CDAI>10). Signs (including acute phase reactants and imaging) and/or symptoms suggestive of active disease (joint related or other). Inability to taper glucocorticoid treatment (below 7.5mg/day prednisone or equivalent). Rapid radiographic progression (with or without signs of active disease) Well-controlled disease according to above standards, but still having RA symptoms that are causing a reduction in quality of life. The management of signs and/or symptoms is perceived as problematic by the rheumatologist and/or patient. Capable of writing and reading in the local language of the participating center or capable of writing and reading proficiently in English. Able and willing to give written informed consent and comply with the requirements of the study protocol as described.
Exclusion criteria
Exclusion criteria: A potential subject who meets any of the following criteria will be excluded from participation in this study:Insufficient mastering of local language of the participating center and EnglishUnable to give informed consentUnable to conclude the study due to e.g. life expectancy < 2 years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome of the study is a patient-centered general outcome, namely general health-related quality of life (HRQoL) as measured by EQ-5D-5L, measured at 0, 3, 6, 9 and 12 months, and expressed as quality-adjusted life-years (QALY).EQ-5D-5L captures five dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression, all rated at five levels of severity. Through well-established valuation techniques, utility scores are assigned to these health states, indicating their preference-based value on a scale from 0 (representing death) to 1 (perfect health). QALYs are calculated by multiplying the utility score at each time point by the duration spent in that health state and summing these products across all timepoints – essentially calculating the area under the utility-time curve. Such general utility-based scores also make it possible to estimate the impact of interventions or treatments by integrating both quantity of life and life expectancy into a single measure. Since QALYs use societal valuation of health states, they provide a standardized approach for comparing health outcomes across different diseases and serve as a valuable tool for decision-making in healthcare policy and clinical practice. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary study parameters/endpoints (if applicable)Physician’s idea regarding underlying problems of a patient, phenotype (e.g., real refractory, limited treatment options due to comorbidities, pain syndromes/obesity, non-adherence, dissatisfaction with care, other (specify) – if relevant, >1 may apply) Changes in D2T RA criteria 6 (see inclusion criteria; 4.2)Radiographic progression (if available) ComorbiditiesAdverse events, toxicity or treatment intolerance Glucocorticoid toxicity index (GTI)Anti-rheumatic treatment received (DMARDs, glucocorticoids) and dose Reason(s) for treatment adjustmentOther pharmacological/non-pharmacological treatments received (for RA or comorbidities)Disease activity (DAS28/CDAI/SDAI, and their components)Health-related quality of life (utility) at individual visits (according to EQ-5D-5L)Health Assessment Questionnaire Disability Index (HAQ-DI) 8Neuropathic pain (painDETECT)Fatigue (FACIT Fatigue Scale)Work productivity (WPAI:RA) Individualized self-reported treatment goals (Goal Attainment Scaling, GAS) Treatment satisfaction by patient and physician (NRS)Patient Acceptable Symptom State (PASS)Morisky Medication-Taking Adherence Scale (MMAS, 4-item)Beliefs about Medicine Questionnaire (BMQ): BMQ-specific and BMQ-generalSocioeconomic status (level of education, income and profession)Diagnostic measures performed and their results/scores (e.g. Ultrasound)Immunologic parameters (e.g. lymphocyte subpopulations, Anti-citrullinated protein antibodies (ACPA): IgA/IgG/IgM, Anti-mutated citrullinated vimentin antibodies (anti-MCV) and Single cell RNA sequencing to be finalised at moment of central measurement) | — |
Countries
Austria, Germany, Hungary, Netherlands, Portugal, Sweden
Contacts
Public ContactA.C.A. Marijnissen
Universitair Medisch Centrum Utrecht
Outcome results
None listed