Navigating through uncertainty-Experience from the UK national VEXAS MDT.

French cohort of children and adolescents with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas: CASSIOPEA study.

BackgroundMultidisciplinary team meetings formulate guideline-based individual treatment plans based on patient and disease characteristics and motivate reasons for deviation. Clinical decision trees could support multidisciplinary teams to adhere more accurately to guidelines. Every clinical decision tree is tailored to a specific decision moment in a care pathway and is composed of patient and disease characteristics leading to a guideline recommendation.ObjectiveThis study investigated (1) the concordance between multidisciplinary team and clinical decision tree recommendations and (2) the completeness of patient and disease characteristics available during multidisciplinary team meetings to apply clinical decision trees such that it results in a guideline recommendation.MethodsThis prospective, multicenter, observational concordance study evaluated 17 selected clinical decision trees, based on the prevailing Dutch guidelines for breast, colorectal and prostate cancers. In cases with sufficient data, concordance between multidisciplinary team and clinical decision tree recommendations was classified as concordant, conditional concordant (multidisciplinary team specified a prerequisite for the recommendation) and non-concordant.ResultsFifty-nine multidisciplinary team meetings were attended in 8 different hospitals, and 355 cases were included. For 296 cases (83.4%), all patient data were available for providing an unconditional clinical decision tree recommendation. In 59 cases (16.6%), insufficient data were available resulting in provisional clinical decision tree recommendations. From the 296 successfully generated clinical decision tree recommendations, the multidisciplinary team recommendations were concordant in 249 (84.1%) cases, conditional concordant in 24 (8.1%) cases and non-concordant in 23 (7.8%) cases of which in 7 (2.4%) cases the reason for deviation from the clinical decision tree generated guideline recommendation was not motivated.ConclusionThe observed concordance of recommendations between multidisciplinary teams and clinical decision trees and data completeness during multidisciplinary team meetings in this study indicate a potential role for implementation of clinical decision trees to support multidisciplinary team decision-making.

To report and synthesize patterns of disease-modifying antirheumatic drug (DMARD) use reported in observational studies of patients with established and early rheumatoid arthritis (RA) after publication of the American College of Rheumatology guidelines promoting universal DMARD use. We searched PubMed for full-length articles in English published between January 1, 2002 and October 1, 2012 that examined DMARD use. The data abstracted from articles included the patient characteristics, country of study, time period studied, patient source, and treating physician type. Study quality was assessed using a modified Newcastle-Ottawa Quality Assessment Scale. We reviewed 1,287 abstracts; 98 full-length articles were selected for additional review and 27 studies describing 28 cohorts of patients were included. Twelve studies described data from cohorts of patients with established RA, and DMARD use in this group of studies ranged from 73-100%. Five studies described data from patients sourced through administrative data and demonstrated consistently lower DMARD use, ranging from 30-63%. Three studies conducted population-based surveys to define cases of RA where DMARD use ranged from 47-73%. Eight studies investigated patients with early RA. DMARD use among patients followed by rheumatologists ranged from 77-98%, whereas DMARD use reported for patients seen by a mix of physicians was significantly lower (39-63%). DMARD use in studies from RA cohorts or registries (in which patients were followed by rheumatologists) ranged from 73-100%, compared with 30-73% in studies from administrative data or population-based surveys (in which patients were not necessarily receiving rheumatology subspecialty care).

Objective: To assess the risk of hospitalized infection among initiators of disease-modifying anti-rheumatic drugs (DMARDs) and/or anti-tumour necrosis factor (anti-TNF) agents in ankylosing spondylitis (AS).Method: We studied AS patients, new users of anti-TNF drugs and/or DMARDs between 1 January 2001 and 31 December 2011. Cohort entry was defined as the date of first prescription of any of these drugs. We used Cox regression with three time-varying drug exposures: current use of DMARDs without biologics, current use of anti-TNF agents alone or in combination with DMARDs (anti-TNF ± DMARDs), and current non-use. Models were adjusted for baseline patient sociodemographic characteristics, comorbidity, outpatient visits and procedures, previous infection, non-steroidal anti-inflammatory drugs, and corticosteroids. Hospitalized infection was defined on the basis of hospitalization discharge diagnoses (primary or non-primary) coding for infection.Results: The cohort included 747 AS patients, with a mean age of 51.1 years (sd 14.6), and 466 (62.4%) were men. During the median follow-up of 1.98 years, 57 hospitalized infections occurred, for an incidence rate of 2.9/100 person-years. The adjusted hazard ratio of infection (relative to unexposed) was 1.00 [95% confidence interval (CI) 0.47–2.11] for the anti-TNF ± DMARDs group and 0.96 (95% CI 0.45–2.04) for DMARDs alone. Use of healthcare, corticosteroids, and previous hospitalized infections were associated with infection.Conclusion: We found no clear evidence that the risk of hospitalized infection was linked to DMARD and/or anti-TNF drug use. Because of scarce published literature on infection risk in AS patients, our results have important implications for clinicians.

Aim: Sulphasalazine is used widely as a second-line treatment of ankylosing spondylitis (AS) after non-steroidal anti-inflammatory drugs (NSAIDs) in Finland. The objective of this study is to evaluate the use of disease-modifying anti-rheumatic drugs (DMARDs) and drug survival in incident AS patients at Helsinki University Central Hospital (HUCH). Method: We identified all incident patients with AS in the hospital register from 1 Jan 2005 to 31 Dec 2009. The index day was defined as the date of AS diagnosis. Medication and clinical data were evaluated until the end of 2010. Results: 176 patients were identified. For 165 of them a DMARD was started. In 9 patients with low disease activity the drug treatment consisted only of NSAIDs. Sulphasalazine was the first synthetic DMARD for 157 (95%) patients. No one were prescribed a biologic drug as the first DMARD. The mean follow-up time was 3.8 years. The mean synthetic DMARD survival was 80%. Bath AS Disease Activity Index (BASDAI) available from 46 patients was 4.1 (1.8) at baseline and decreased by 1.6 (95% CI 2.2-1.1, p<0.001) during the DMARD treatment. Because of continuing disease activity, 28 (17%) patients became eligible for reimbursement of biological DMARDs and a TNF inhibitor was instituted. This was predicted by peripheral disease, higher ESR, and CRP at the baseline. Conclusion: Most patients with incident AS do fairly well with synthetic DMARDs but the proportion of the patients needing biological DMARD treatment grows over time. Use of synthetic DMARDs may reduce or postpone the need for biological DMARD treatment in AS.

To analyze the characteristics, outcomes, and predictive factors of disease-modifying antirheumatic drug (DMARD) use in 48 patients with antisynthetase syndrome [characterized by myositis, interstitial lung disease (ILD), arthritis, Raynaud's phenomenon (RP), and/or mechanic's hands] and the presence of anti-histidyl-transfer RNA synthetase (anti-Jo1) autoantibodies. Forty-eight patients (33 women, 15 men) who were anti-Jo1-positive referred to one center between 1998 and 2008 were analyzed retrospectively. The median age of disease onset was 43 years [interquartile range (IQR) 33-53 yrs]. The median followup was 5 years (IQR 2-8 yrs). At diagnosis, 81% of patients presented with myositis, 80% ILD, 77% arthralgia, 48% RP, and 21% mechanic's hands. During the followup, 14 patients (29%) had no need for DMARD, while 34 (71%) required DMARD. Patients with mechanic's hands (p=0.02) and higher creatine phosphokinase at diagnosis (median 6070 IU/l vs 1121 IU/l; p=0.002) were more likely to need DMARD. ILD, noted on computed tomography scan by a nonspecific interstitial pneumonia score, was lower in the group of patients with no DMARD need (4 vs 7; p=0.04). Twenty patients (44%) presented with a pulmonary aggravation (worsening of radiologic score of ILD and/or pulmonary function test results) leading to DMARD use. Nonspecific interstitial pneumonia score (7 vs 5; p=0.05) and total lung volume (57.5% vs 70%; p=0.02) values predicted pulmonary aggravation. Our study outlines the burden of chest involvement for the prognosis of antisynthetase syndrome in terms of patients' requirement for DMARD therapy.

Collective Wisdom and Multidisciplinary Tumor Boards

Use of nonbiologic disease-modifying antirheumatic drugs (DMARDs) and/or biologic DMARDs is generally recommended to improve the prognosis of patients with rheumatoid arthritis (RA). The objective of this study was to describe the changing trends in DMARD use for RA over the past 2 decades. We analyzed data from an open longitudinal cohort of RA patients recruited from rheumatologists' practices in northern California. We examined baseline demographic and clinical characteristics of the participants and their long-term DMARD use through annual comprehensive structured telephone interviews. A total of 1,507 established RA patients were recruited through 5 enrollment periods between 1983 and 2009. Between 1983 and 2009, the use of any DMARD increased from 71% of all patients to 83% (P for trend < 0.0001). In 2009, 43% received a biologic DMARD, 34% were on both nonbiologic and biologic DMARDs, and 40% were treated with only nonbiologic DMARDs. The 4 most commonly used nonbiologic DMARDs in 2009 were methotrexate (49%), hydroxychloroquine (30%), leflunomide (13%), and sulfasalazine (7%). Etanercept (20%) was the most commonly used biologic DMARD in 2009, followed by infliximab (10%), adalimumab (9%), and abatacept (6%). Use of oral steroids was common (40-50%) and remained similar throughout the study period. There has been a significant increase in the use of DMARDs for RA over the past 2 decades. However, 15% of the individuals with a clinical diagnosis of RA were not receiving DMARDs in 2009. Future research should focus on sociodemographic and clinical factors associated with DMARD use for RA.

Background:Disease-modifying anti-rheumatic drug (DMARD) use in older adults with late-onset rheumatoid arthritis (LORA) is suboptimal despite their high efficacy and tolerability in this population. In contrast, long-term glucocorticoid (GC) use...

BackgroundThe use of non-biologic disease-modifying anti-rheumatic drugs (DMARDs) has been scarcely explored in patients with IgG4-related disease (IgG4-RD). There is only one small series reporting the use of methotrexate.ObjectivesThis paper...

Background Severe long-term consequences of rheumatoid arthritis (RA) are well known, while the existing evidence suggests that early and active use of disease modifying anti rheumatic drugs (DMARDs) may improve the outcomes. Objectives To study the impact of disease on the outcomes of patients with RA, and to describe their use of DMARDs over time. Methods In August 2000, a questionnaire was sent to 1500 patients with RA who had been seen in Jyvaskyla Central Hospital since 1995 and were included in the RA database of the hospital. A total of 1093 (73%) patients replied and were included in the study. The overall impact of RA was assessed by asking “How much does RA impact your life?”. Response alternatives were 1)”Not at all”, 2)”Some”, 3)”Much”, and 4)”Very much”. The questionnaire also assessed functional capacity by Health Assessment Questionnaire (HAQ, range 0–3), pain and general health by 10 cm visual analogue scale (VAS, range 0–100), and current usage of DMARDs. Data concerning demographics, “diagnostic delay”, and previous use of DMARDs were available through the RA database. The intensity of the use of DMARDs was calculated as the percentage of the time on DMARDs from the total time elapsed since the diagnosis to August 2000. Results A total of 13% of the patients did not experience any impact from RA, while 48% experienced “some”, 27% “much”, and 12% were impacted “very much”. Those experiencing “very much” level of impact by RA were older, had longer disease duration, had less education, were more often rheumatoid factor positive, had worse HAQ and general health scores, had more pain, and also had longer diagnostic delay, and less use of DMARDs over time than those experiencing “no” or “some” impact by the disease (Table 1: mean values for variables are shown except median for DMARD%.) Conclusion In spite of extensive therapy with DMARDs, RA causes considerable impact in the majority of patients in the long term. Shortening of diagnostic delay (improvement in the cooperation between primary and specialist care) and continuous active therapy with available DMARDs should be emphasised to decrease disease impact.

BackgroundRecent-onset inflammatory arthritis (IA) may represent a broad range of diseases. Few studies have examined the full spectrum of diagnostic outcomes in an unselected cohort of recent-onset IA patients.ObjectivesTo describe...

We aimed to quantify the rate of Mycobacterium tuberculosis disease (TB) among a cohort of patients with rheumatoid arthritis (RA) and to assess whether the independent use of disease-modifying antirheumatic drugs (DMARDs) is associated with the risk of developing TB. The study was performed using the PharMetrics Patient-Centric database (PharMetrics). The cohort consisted of all subjects with > or =1 occurrence of a diagnosis of RA during an inpatient or outpatient visit during the period of September 1998 through December 2003. Conditional logistic regression was used in a nested case-control analysis to estimate the rate ratio (RR) of TB with any use of biological or traditional DMARDs during the year before the index date. We also assessed the interaction between DMARDs and the current use of corticosteroids. The cohort consisted of 112,300 patients with RA. A total of 386 cases of TB were identified, which resulted in an overall rate of 2.19 cases per 1000 person-years. The adjusted RR of TB for biological DMARD use is 1.5 (95% CI, 1.1-1.9). Use of traditional DMARDs was also independently associated with TB (RR, 1.2; 95% CI, 1.0-1.5). RRs of developing TB disease with the use of biological or traditional DMARD were lower among current users of corticosteroids than among noncurrent users of corticosteroids. We found that the use of biological and traditional DMARDs is associated with an increased risk of developing TB in patients with RA, mainly among noncurrent users of corticosteroids.

Objectives:To demonstrate the pattern of disease-modifying antirheumatic drugs (DMARDs) use in Saudi and non-Saudi rheumatoid arthritis (RA) patients, and to evaluate the association of DMARDs use with anti-mutated citrullinated vimentin (anti-MCV) positivity and other factors.Methods:Retrospectively, for a period of 7 years (2007-2014), we studied 205 RA patients, at King Abdulaziz University Hospital (KAUH), Jeddah, Saudi Arabia. All patients used DMARDs. Pattern of use for all 6 DMARDs was almost the same among Saudis and non-Saudis with no significant difference (p>0.05) for each DMARD; MTX was the most commonly used DMARD (71-76%).Results:There was no association between anti-MCV positivity and different DMARDs use. Methotrexate was used 76 times as combination, scoring the highest in this respect. There was a significant correlation (p<0.05) between Plaquenil with Methotrexate and with Sulfasalazine; Leflunomide with anti-TNF and with Prednisolone; age with Methotrexate and with Plaquenil; anti-MCV positivity with Prednisolone. Saudi/non-Saudi status showed no correlation with all factors or drugs. There was no significant association between DMARDs and comorbidity.Conclusion:Similar to worldwide results, MTX was the most commonly used DMARD; with the addition of anti-TNF to increase the effect, and folic acid to minimize the side effects. In this cohort, the pattern of use for all DMARDs was similar among Saudis and non-Saudis; treatment depended neither on anti-MCV positivity nor on the presence of comorbid conditions. A study of the association of DMARDs with disease activity is recommended.

To determine whether the incidence of herpes zoster is elevated in patients with rheumatoid arthritis (RA) and whether herpes zoster is associated with use of disease-modifying antirheumatic drugs (DMARDs) in patients with RA. Two retrospective cohort studies were conducted using data from a US integrated managed care database (PharMetrics claims database) from 1998-2002 and the UK General Practice Research Database (GPRD) between 1990-2001. Rates of herpes zoster among patients with RA and randomly sampled non-RA patients were compared. A nested case-control analysis was performed within each RA cohort to examine the effect of current treatment on herpes zoster risk. A total of 122,272 patients with RA from the PharMetrics database and 38,621 from the GPRD were included. The adjusted hazard ratios of herpes zoster for patients with RA compared with non-RA patients were 1.91 (95% confidence interval [95% CI] 1.80-2.03) in the PharMetrics database and 1.65 (95% CI 1.57-1.75) in the GPRD. In the PharMetrics database, current use of biologic DMARDs alone was associated with herpes zoster (odds ratio [OR] 1.54, 95% CI 1.04-2.29), as was current use of traditional DMARDs alone (OR 1.37, 95% CI 1.18-1.59). In the GPRD, current use of traditional DMARDs was associated with herpes zoster (OR 1.27, 95% CI 1.10-1.48). In both data sources, use of oral corticosteroids was associated with herpes zoster regardless of concomitant therapies. Data from 2 large databases suggested that patients with RA are at increased risk of herpes zoster. Among patients with RA, DMARDs and/or use of oral corticosteroids appeared to be associated with herpes zoster.