Rheumatology and Therapy最新文献

Introduction: Ozoralizumab (OZR) is a novel tumor necrosis factor (TNF) inhibitor that was launched in Japan for treating patients with rheumatoid arthritis (RA) who have had an inadequate response to existing therapies. This post-hoc analysis aimed to compare the efficacy of OZR administered without methotrexate (MTX) with placebo or OZR administration in combination with MTX.

Methods: We analyzed the OZR group (30 mg) in the NATSUZORA trial (non-MTX, open trial) (OZR group; n = 94) and the placebo group (MTX group; n = 75) and the 30-mg OZR group (OZR + MTX group; n = 152) in the OHZORA trial (combined MTX, double-blind trial), and the covariates were adjusted by propensity score matching. Subsequently, the American College of Rheumatology (ACR) 20/50/70 response rates from baseline to 24 or 52 weeks were compared. Furthermore, to compare longitudinal data on disease activity indicators, a mixed-effects model for repeated-measures analyses was used.

Results: Comparing the OZR and MTX groups, 52 patients were matched in each group. The OZR group showed improvements in the ACR20 (OZR group, 67.3% vs. MTX group, 34.6%, p = 0.001), ACR50 (51.9% vs. 17.3%, p < 0.001), and ACR70 (26.9% vs. 11.5%, p = 0.047) response rates compared to those in the MTX group. Comparing the OZR and OZR + MTX groups, 77 patients were matched in each group. No significant difference was observed in the ACR20 response rate (OZR group, 58.4% vs. OZR + MTX group, 70.1%, p = 0.130). However, the OZR + MTX group showed higher ACR50 (44.2% vs. 62.3%, p = 0.024) and ACR70 (29.9% vs. 45.5%, p = 0.046) response rates.

Conclusion: OZR administration without MTX was associated with an improvement in the signs and symptoms of RA compared to placebo administration (continuation of MTX monotherapy). OZR and MTX administration showed better efficacy than OZR administration alone.

Introduction: This study evaluated the prevalence and incidence of systemic lupus erythematosus (SLE) in Germany and explored real-world data on sequence of therapy (SOT; sequence of drugs as prescribed in clinical practice).

Methods: This retrospective, observational, longitudinal cohort study using German claims data from the WIG2 GmbH Scientific Institute for Health Economics and Health System Research database (January 2011-December 2019), extrapolated to the statutory health insurance (SHI)-insured population, evaluated prevalence and incidence in an epidemiological analysis group and SLE treatment patterns in an incident cohort (subgroup ≥ 18 years of age with incident disease and ≥ 24-month follow-up post index date). Analyses were descriptive.

Results: Based on the epidemiological analysis (N = 3017), annual SLE prevalence per 100,000 gradually increased from 40.47 in 2012 to 59.87 in 2019 in the SHI population. In contrast, annual SLE incidence was relatively stable, ranging from 8.83 in 2012 to 8.86 in 2019. In the incident cohort (n = 941), based on SOT analysis (n = 681), treatment gaps of > 60 days were common: 67.1%, 51.2% and 54.9% in SOT1, SOT2 and SOT3, respectively. Corticosteroids were the most frequent monotherapy in SOT1 (31.0% vs 0% in SOT2/SOT3); 30.0-70.0% of patients received a corticosteroid combination therapy across SOTs. Over 50% of patients in each SOT received an antimalarial therapy (combination or monotherapies). The use of biologic disease-modifying drugs was low, ranging from 0.4% in SOT1 to 9.7% in SOT3.

Conclusions: Our data demonstrate an increased prevalence of SLE with stable incidence in Germany, suggesting improved survival of affected patients. Nevertheless, suboptimal treatment patterns, including limited use of biologics, reflect a high unmet need for optimised and personalised therapies in patients with SLE.

Introduction: It is important to understand the differences in patient-physician perceptions and factors affecting satisfaction with treatment in patients with systemic sclerosis (SSc).

Methods: This web-based survey (conducted in Japan in March 2023) targeted patients aged ≥ 18 years with SSc and physicians in hospitals with ≥ 20 beds and seeing ≥ 3 patients with SSc monthly. Physicians and patients answered similar questions.

Results: Responders were 301 patients (63.8% female; 47.5% limited cutaneous SSc; 44.9% diffuse cutaneous SSc) and 129 physicians (51.2% rheumatologists; 20.9% dermatologists). The most common problematic symptoms reported by patients having each symptom were Raynaud's phenomenon (RP) (59.5%), skin tightening (47.4%), and malaise (45.5%). Physicians also perceived RP as the common problematic symptoms (46.5%). Conversely, there was a large gap in the perception of malaise as problematic (5.4%). There was a ≥ 20% difference in the percentage of respondents who felt that treatments improved symptoms of reflux esophagitis (48.8% in patients vs. 76.7% in physicians), dysphagia (25.0% vs. 52.7%), constipation (35.1% vs. 62.8%), diarrhea (36.1% vs. 62.8%), and pain (47.6% vs. 69.0%). Patient characteristics associated with high satisfaction with treatment included treatment responsiveness, age ≥ 50 years, being anti-topoisomerase I antibody positive, having dermatological or digestive symptoms as problematic symptoms, and not feeling they should have seen their physician earlier.

Conclusions: Patients and physicians had different perceptions of symptoms and treatment response. Patients' perception of improvement affected their satisfaction with treatment. Reviewing treatment goals and content between patients and physicians is necessary to improve treatment satisfaction.

Trial registration: UMIN000050368.

Introduction: Postmenopausal osteoporosis (PMO) increases the risk of fragility fractures (FF), leading to disability, higher mortality, and elevated healthcare costs. Despite available treatments, osteoporosis (OP) remains undertreated, especially in women over 50 years at high risk for FF. Real-world data on OP care in Spain are limited. This study aims to assess the OP treatment gap, healthcare resource utilisation (HCRU), and costs among Spanish women following a first FF or PMO diagnosis.

Methods: This retrospective study used data from the BIG-PAC^® administrative database on women aged ≥ 50 years with a first FF (cohort 1) or newly diagnosed PMO (cohort 2) between 2014 and 2018. Patients were followed for 2 years after the index event. The primary outcome was the proportion of women not prescribed OP medication within 6 months after the index event (treatment gap). Secondary outcomes included fracture incidence, mortality, HCRU, and costs.

Results: The study included 22,142 women: 3190 in cohort 1 and 18,952 in cohort 2. The OP treatment gap was higher in cohort 1 vs cohort 2 (41.5% vs 23.6%). In cohort 1, 59.2% were diagnosed with PMO after the first FF, with 88% experiencing subsequent fracture(s). OP treatment persistence decreased over time in both cohorts. Fracture rates were lower in women prescribed OP treatment vs those who were not (8.35 vs 13.8 per 1000 patient-years) and in those who showed 24-month-persistence and 12-month adherence to treatment vs those who did not (8.98 and 7.66 vs 10.79 and 10.76). The 2-year mean cost per patient was higher in cohort 1 (€10,601) than in cohort 2 (€1659), with the highest costs incurred for hip (€15,833) and vertebral (€10,593) fractures.

Conclusion: This study highlights a significant treatment gap in Spanish women aged ≥ 50 with a first FF or newly diagnosed PMO. Costs are particularly high for those with a first FF, especially for hip or vertebral fractures. Improving treatment adherence could reduce fracture risk, healthcare costs, and resource utilisation.

Introduction: Prescribable digital health applications (DiGAs) present scalable solutions to improve patient self-management in rheumatology, however real-world evidence is scarce. Therefore, we aimed to assess the effectiveness, usage, and usability of DiGAs prescribed by rheumatologists, as well as patient satisfaction.

Methods: The DiGAReal registry includes adult patients with rheumatic conditions who received a DiGA prescription. Data at baseline (T0) and the 3-month follow-up (T1) were collected through electronic questionnaires. Study outcomes included DiGA-specific outcome assessments as well as generic outcome assessments, including the Patient Global Impression of Change (PGIC), Patient Activation Measure (PAM®), and the German Telehealth Usability and Utility Short Questionnaire (TUUSQ). Changes between T0 and T1 were analyzed using descriptive statistics and paired tests.

Results: A total of 191 patients were included between June 2022 and April 2023. Of these, 127 completed the 3-month follow-up, and 114 reported using the prescribed DiGA, with 66% reporting weekly use and 15% completing the full DiGA program. The most commonly prescribed DiGAs targeted pain management (53%). Symptom improvement was reported by 51% of patients using a DiGA, with significant reductions in exhaustion levels (p = 0.03). Significant DiGA-specific improvements were observed for DiGAs addressing back pain (p = 0.05) and insomnia (p = 0.006). However, no overall significant changes were detected in patient activation, health literacy, pain, overall health, or disease activity. Back pain and weight management DiGAs were the most effective, frequently used, and best-rated DiGAs, with symptom improvements reported by 50% to 82% of patients.

Conclusion: The findings suggest that DiGAs can improve symptom management in rheumatic patients, especially for conditions like back pain and weight control. Further real-world evidence is needed and may support value-based digital health efforts and reimbursement frameworks.

Introduction: Fatigue is a key symptom in patients with ankylosing spondylitis (AS). The objective of this analysis was to estimate the median time to initial and stable improvement events in fatigue in patients with AS receiving tofacitinib.

Methods: This post hoc analysis used data from a phase 3 trial (NCT03502616) in patients with active AS receiving tofacitinib 5 mg twice daily or placebo. Time to improvement in fatigue was assessed using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) total score, experience domain score, and impact domain score. The rapidity of improvement was assessed by time-to-event analyses (nonparametric Kaplan-Meier models); initial improvement events (i.e., time to first week of FACIT-F improvement) and stable improvement events (i.e., time to first week of FACIT-F improvement, sustained to 16 weeks) were examined.

Results: Overall, 269 patients were assessed (mean disease duration: 14.2 [standard deviation (SD): 9.8] years; mean baseline FACIT-F total score: 27.2 [SD: 9.3]). Median times to initial and stable improvement events in FACIT-F total and domain scores were significantly shorter and occurred in more patients receiving tofacitinib than placebo. Median time to initial and stable improvement events of 6 points in FACIT-F total score were 8 and 12 weeks with tofacitinib, respectively (placebo: not reached); 70.0% versus 48.5% of patients receiving tofacitinib versus placebo, respectively, experienced initial improvements of 6 points in FACIT-F total score within 16 weeks.

Conclusions: Improvements in fatigue occurred more rapidly with tofacitinib than with placebo. These results may be useful for healthcare providers when discussing tofacitinib treatment expectations with patients.

Trial registration: ClinicalTrials.gov: NCT03502616 (June 7, 2018).

Introduction: An intriguing aspect that emerged in recent years is the transition phase from psoriasis (PsO) to psoriatic arthritis (PsA). The PRESTO instrument allows estimating a patient's risk of developing PsA based on a few clinical items. The aim of this study was to apply and evaluate the performance of the PRESTO tool in a cohort of patients with PsO.

Methods: Consecutive patients with PsO were enrolled. Dermatological and rheumatological assessment was carried out in order to evaluate clinical features of PsO, to exclude the diagnosis of PsA, and to administer the PRESTO tool.

Results: Between January 1, 2024 and April 1, 2024, 100 patients were assessed. Eight-four patients found the questionnaire to be very useful and easy. The estimated risk (median/IQR) of 1-year progression to PsA found in our group was 2.45% at 1 year (1.1-4).

Conclusions: The PRESTO instrument was feasible and well accepted by patients. The 1-year risk assessed by PRESTO tools is consistent with other reports in the literature.

Introduction: We assessed tofacitinib efficacy and safety in ankylosing spondylitis (AS) by body mass index (BMI) category.

Methods: Data were pooled from phase 2/3 trials; analyses included patients with active AS randomized (1:1) to tofacitinib 5 mg twice daily or placebo, who were stratified by baseline BMI into < 25, ≥ 25 to < 30, and ≥ 30 kg/m² categories. Efficacy was assessed at week 12 and safety to week 16.

Results: Of 370 patients, 153, 131, and 86 had a baseline BMI  of < 25, ≥ 25 to < 30, and ≥ 30 kg/m², respectively. At baseline, patients with BMI < 25 kg/m² were younger and more likely to be current smokers/Asian, and patients with BMI ≥ 30 kg/m² had higher mean waist circumference/swollen joint count (SJC) and were more likely to have enthesitis, high-sensitivity C-reactive protein (hsCRP) > 5 mg/L, an inadequate response to tumor necrosis factor inhibitors (TNFi), and prior biologic disease-modifying anti-rheumatic drug (bDMARD) use versus other categories. Across categories, tofacitinib responses/improvements were greater than with placebo, except for ≥ 40% Assessment of SpondyloArthritis international Society improvement (ASAS40), ASAS partial remission, 50% improvement from baseline in the Bath Ankylosing Spondylitis Disease Activity Index score (BASDAI50), and Ankylosing Spondylitis Disease Activity Score using C-reactive protein (ASDAS-CRP) inactive disease rates, which were similar for tofacitinib and placebo in the BMI ≥ 30 kg/m² category. Treatment effects were similar across categories, except for BASDAI50, which was smaller in the BMI ≥ 30 category versus  the < 25 kg/m² category. More adverse events (AEs) and serious adverse events (SAEs) with tofacitinib were reported in the BMI < 25 kg/m² category, which had a higher proportion of current smokers versus other categories.

Conclusions: Regardless of baseline BMI category, efficacy was greater with tofacitinib versus placebo in patients with AS, and no treatment effect differences between categories were observed, with exceptions for BMI ≥ 30 kg/m² (more active/treatment-refractory disease and a smaller sample size). Overall, tofacitinib safety was generally comparable across categories; however, AE/SAE rates with tofacitinib were higher in the BMI < 25 kg/m² category (which had more current smokers). This post hoc analysis demonstrates that tofacitinib can be considered as a treatment option for AS, regardless of baseline BMI category; however, interpretation was limited by small sample sizes and differences in sample sizes and baseline characteristics across categories.

Trial registration: ClinicalTrials.gov identifiers, NCT01786668 and NCT03502616.