Rheumatology and Therapy最新文献

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: Celiac disease (CD) affects the small intestine, leading to a progressive disappearance of intestinal villi, and can be found in association with several other autoimmune and inflammatory conditions. The main objective of this study was to determine the prevalence and the clinical significance of anti-transglutaminase and anti-endomysium antibodies in patients diagnosed with early rheumatoid arthritis (RA) and spondyloarthritis (SpA).

Methods: We measured anti-transglutaminase and anti-endomysium antibodies in biobanked serum samples at inclusion in two French prospective multicenter cohorts of patients with suspected early rheumatoid arthritis (ESPOIR, n = 713) and spondyloarthritis (DESIR, n = 709). Results were compared with the clinical, laboratory, and radiographic findings obtained in patients during a 10-year follow-up period.

Results: In the DESIR cohort, anti-transglutaminase antibodies were evidenced at low levels (less than three times the upper limit of normal) in 2/709 (0.42%) patients and anti-endomysium antibodies in 0/709 (0%). In the ESPOIR cohort, anti-transglutaminase antibodies were evidenced in 6/713 (0.84%) patients and anti-endomysium antibodies in 1/713 (0.14%). Only the latter patient was confirmed to have celiac disease. Interestingly, this patient was ultimately diagnosed with Sjögren's disease, an autoimmune condition known to be associated with an increased risk of celiac disease.

Conclusion: The very low identified prevalence of anti-transglutaminase and anti-endomysium antibodies suggests a negligible risk of celiac disease in patients with early-stage RA or SpA, which are among the most common inflammatory rheumatic conditions. Consequently, routine screening for celiac disease via these antibodies in patients presenting with early inflammatory rheumatic conditions should not be performed except in case of clinical suspicion of celiac disease.

Introduction: Axial spondyloarthritis (axSpA) is a chronic inflammatory condition associated with considerable pain and impaired health-related quality of life (HRQoL) for affected patients. Despite the documented increase in healthcare resource utilization (HRU) related to axSpA, few studies have explored the impact of diagnostic delays on these outcomes. This study sought to determine the association between diagnostic delay of axial spondyloarthritis (axSpA) and costs in the 3 years after diagnosis.

Methods: This is a retrospective, observational study based on routine follow-up data from adult patients with confirmed axSpA diagnosis in three tertiary Spanish hospitals. Sociodemographic and clinical variables were collected at diagnosis. Direct and indirect healthcare costs were estimated from healthcare resource use (HRU) and productivity losses. The correlation between diagnostic delay and total healthcare costs was analyzed.

Results: Eighty-two patients (62.2% men; mean age: 39.3 years at diagnosis) were included, mostly with radiographic axSpA (r-axSpA) (67.1%). The mean (standard deviation, SD) diagnostic delay was 10.1 (9.3) years, with a median (interquartile range, IQR) of 5.4 (2.3, 17.2) years. The mean total healthcare cost per patient accumulated over 3 years was €25,812.6 (direct: €16,384.7; indirect: €9427.9). Patients with longer diagnostic delay (> 5.4 years) had 57% higher total healthcare cost (€31,717.7 vs. €20,188.7, p = 0.029) and higher disease activity at diagnosis (BASDAI score 4.7 vs. 3.4, p = 0.007) and after 3 years (3.9 vs. 2.9, p = 0.042) compared to those with shorter delay (≤ 5.4 years).

Conclusions: The diagnostic delay in axSpA remains high and is associated with an increase in healthcare costs post-diagnosis. Actions to reduce diagnostic delay should be prioritized by healthcare systems to potentially improve outcomes and reduce long-term costs.

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: Eosinophilic granulomatosis with polyangiitis (EGPA) is an eosinophil-associated disease (EAD) characterized by inflammation in small- to medium-sized blood vessels. In the REal-world inVestigation of Eosinophilic-Associated disease overLap (REVEAL) study, overlap among 11 EADs was assessed. In the present sub-study, we evaluated EGPA overlap with other EADs, all-cause EAD- and EGPA-related healthcare resource utilization (HCRU) and costs, and their relationship with blood eosinophil count and treatments received.

Methods: REVEAL, a retrospective study, used Optum's de-identified Clinformatics^® Data Mart Database. In this sub-study, eligibility criteria included an age of ≥ 12 years, ≥ 1 EAD, continuous health-plan eligibility, and compliance with the EGPA/GPA case definition per International Classification of Diseases Ninth/Tenth Revision diagnostic codes between 1 January 2015 and 30 June 2018. Patients were grouped based on whether they had received immunomodulators/cyclophosphamide/mepolizumab (ICM) or not (non-ICM).

Results: Of 701 patients with EGPA, 29.5% were in the ICM group. Overall, 72.2% had ≥ 1 overlapping EAD. The number of overlaps was similar for the ICM and non-ICM groups. In patients with blood eosinophil counts ≥ 300 cells/µL, 22.8% had ≥ 1 overlapping EAD. The mean annual all-cause cost was $98,644, 54.1% of which was from outpatients and 33.6% from inpatients. The mean annual EAD- and EGPA-related costs were $23,820 and $9,306, respectively. Patients in the non-ICM group versus the ICM group had higher all-cause ($101,560 vs $91,684) but lower EAD-related ($22,733 vs $26,412) and EGPA-related ($6,171 vs $16,786) costs. All-cause HCRU and costs increased with increasing overlap.

Conclusions: EGPA was associated with substantial HCRU and costs, driven by outpatient and inpatient settings. More overlapping EADs were associated with higher HCRU and costs, highlighting the need for treatment to reduce healthcare expenditure in these patients. Infographic available for this article.

Introduction: To conduct a literature review exploring the humanistic burden, costs, and guideline recommendations for non-surgical management of moderate-severe pain in knee osteoarthritis (KOA).

Methods: Published studies (2018-25 April 2023) assessing the burden of moderate-severe pain in KOA were identified by searching Medline, Embase, EconLit, and Cochrane database, supplemented with grey literature hand searches and reference list snowballing. Treatment guidelines were also identified for key countries.

Results: This review included 106 publications and 37 treatment guidelines. Patients with moderate-severe pain were found to experience a low quality of life (QoL) and an impaired ability to perform daily tasks. The economic burden of KOA was substantial, including cost of medical visits, non-operative treatment (physical therapy and hyaluronic acid [HA] being key drivers) and productivity losses. Non-steroidal anti-inflammatory drugs (NSAIDs) were among the most frequently used pharmacological treatments, with intra-articular (IA) injections used to varying degrees. Opioid use was also frequently reported. Guidelines universally recommended NSAIDs, albeit with limited dose and duration for oral NSAIDs. IA-corticosteroids were conditionally/moderately recommended for short-term use by most guidelines, while IA-HA and opioids were rarely recommended. Guidelines are not specific to patients with moderate-severe pain and do not distinguish between different KOA phenotypes.

Conclusions: KOA with moderate-severe pain is associated with substantial humanistic and economic burden. Real-world data suggest that some treatments are regularly used at high cost regardless of the lack of evidence-based recommendations. There remains a need for new treatment options that successfully relieve pain, improve QoL and delay the need for surgery. Graphical abstract available for this article.

Introduction: Routine care studies of psoriatic arthritis (PsA) and ankylosing spondylitis (AS) demonstrated attenuated responses to tumor necrosis factor inhibitors in current/past versus never smokers. This post hoc analysis assessed tofacitinib efficacy and safety in patients with PsA or AS by cigarette smoking status at trial screening.

Methods: Pooled data from phase 3 and long-term extension (safety only) PsA trials and phase 2 and 3 AS trials were assessed by current/past versus never smoker status. Analysis included efficacy and safety data for tofacitinib 5 (PsA/AS) and 10 (PsA only) mg twice daily (BID) or placebo, and safety data in AS for tofacitinib 2 and 10 mg BID. Efficacy outcomes included American College of Rheumatology ≥ 50% responses (ACR50) and minimal disease activity (MDA) responses to month (M)6/M3 (tofacitinib/placebo) in PsA; and ≥ 40% improvement in Assessment of SpondyloArthritis international Society responses (ASAS40) and AS Disease Activity Score (ASDAS) < 2.1 responses to week (W)16 in AS. Safety was assessed to M48/W48 (PsA/AS), adjusted for treatment/smoking status/median body mass index (BMI) status/sex/trial/treatment-smoking status interaction.

Results: PsA/AS cohorts included 342/178 current/past and 572/194 never smokers. Tofacitinib efficacy was generally greater versus placebo to M3/W6 (PsA/AS), and comparable in current/past and never smokers to M6/W16 (PsA/AS). In patients receiving ≥ 1 tofacitinib dose, adjusted treatment-emergent adverse event (TEAE)/serious AE (SAE)/discontinuation due to AE incidence rates (IRs) to M48 in PsA were higher in current/past versus never smokers; adjusted IRs to W48 in AS were higher in current/past versus never smokers for TEAEs, but similar for SAEs/discontinuation due to AEs.

Conclusions: In both patients with PsA and AS, tofacitinib efficacy was greater versus placebo, and comparable across smoking categories. Adjusted IRs were higher in current/past versus never smokers for TEAEs, SAEs, discontinuation due to AEs in PsA, and for TEAEs in AS, complementing reports of associations between smoking and comorbidities in spondyloarthritis. Findings support increased surveillance/caution for patients with PsA or AS with smoking history.

Trial registration: ClinicalTrials.gov: NCT01877668/NCT01882439/NCT03486457/NCT01976364/NCT01786668/NCT03502616.