To identify predictors of treatment changes and to evaluate the effectiveness and patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) initiating tofacitinib in a real-world setting.

The non-interventional study ESCALATE-RA included 1518 patients with RA from Germany. RA treatment, including all changes in therapy, was documented for 24 months starting from the initial intake of tofacitinib.

All patients started with tofacitinib therapy, either as monotherapy or in combination with methotrexate (MTX).

The impact of several factors of interest on the number and timing of treatment changes was assessed as primary outcome using Cox proportional hazards models. Further outcomes were tofacitinib drug survival and the use of follow-up disease-modifying antirheumatic drugs after first treatment change. We also assessed the effectiveness, concomitant glucocorticoid (GC) use, PROs (such as functional ability, patient satisfaction, pain and quality of life) and safety. Analyses were based on observed data.

‘Lack of efficacy’ (HR 3.30) and ‘intolerance’ (HR 4.43) leading to termination of tofacitinib were key factors favouring therapy changes. Higher patient satisfaction was significantly associated with a reduced likelihood of treatment changes (HR 0.82). Increasing GC doses were associated with a higher probability of step-up/switch changes (HR 1.21). The estimated tofacitinib drug survival was 48% at the end of study. Proportions of patients achieving low disease activity (both Simplified Disease Activity Index (SDAI) and Clinical Disease Activity Index (CDAI) 62%) and remission (SDAI 25%, CDAI 28%) increased from baseline under tofacitinib and were comparable between monotherapy and combination therapy with MTX. Mean concomitant GC dose decreased (2 mg/day). PROs indicated reduced pain and fatigue, while functional ability and quality of life improved. 63.9% of the patients experienced a treatment-emergent adverse event (AE), 8.8% a treatment-emergent AE of special interest and deaths occurred in 0.5%.

Key factors for therapy changes in patients with RA treated with tofacitinib were lack of efficacy and intolerance. Higher patient satisfaction was associated with a reduced probability of treatment changes, while increased GC doses led to a higher likelihood of step-ups/switches. Patients demonstrated a marked reduction in disease activity for up to 24 months, along with improvements in functional ability, pain and quality of life. Observed AEs were consistent with the known safety profile of tofacitinib.

NCT03387423.

There are little available data on the prevalence, economic and quality of life impacts of musculoskeletal disorders in sub-Saharan Africa. This lack of evidence is wholly disproportionate to the significant disability burden of musculoskeletal disorders as reported in high-income countries. Our research aimed to undertake an adequately powered study to identify, measure and value the health impact of musculoskeletal conditions in the Kilimanjaro region, Tanzania.

A community-based cross-sectional survey was undertaken between January 2021 and September 2021. A two-stage cluster sampling with replacement and probability proportional to size was used to select a representative sample of the population.

The survey was conducted in 15 villages in the Hai District, Kilimanjaro region, Tanzania.

Economic and health-related quality of life (HRQOL) questionnaires were administered to a sample of residents (aged over 5 years old) in selected households (N=1050). There were a total of 594 respondents, of whom 153 had a confirmed musculoskeletal disorder and 441 matched controls. Almost three-quarters of those identified as having a musculoskeletal disorder were female and had an average age of 66 years.

Questions on healthcare resource use, expenditure and quality of life were administered to all participants, with additional more detailed economic and quality of life questions administered to those who screened positive, indicating probable arthritis.

There is a statistically significant reduction in HRQOL, on average 25% from a utility score of 0.862 (0.837, 0.886) to 0.636 (0.580, 0.692) for those identified as having a musculoskeletal disorder compared with those without. The attributes ‘pain’ and ‘discomfort’ were the major contributors to this reduction in HRQOL.

This research has revealed a significant impact of musculoskeletal conditions on HRQOL in the Hai district in Tanzania. The evidence will be used to guide clinical health practices, interventions design, service provisions and health promotion and awareness activities at institutional, regional and national levels.