Faculty Bibliography

Behcet's syndrome (BS) shows a peculiar distribution, with a much higher prevalence in countries along the ancient Silk Road compared with rest of the world. BS also seems to follow a more severe course in ethnic groups with higher prevalence. Diagnosis depends on clinical findings. Criteria sets may not help in patients with less frequent types of involvement. Management strategies should be modified according to the age and sex of the patient and the organs involved. Being a serious health problem in endemic areas, BS also attracts global attention as a model to study inflammatory diseases of unknown cause.

OBJECTIVES: The Behcet's Syndrome Activity Score (BSAS) is the first patient reported outcome measure developed to assess the global disease activity in patients with Behcet's syndrome (BS). We aimed to evaluate the reliability and validity of the Turkish version of BSAS for measuring disease activity in BS. We further investigated the performance of Routine Assessment of Patient Index Data (RAPID)3, a patient-reported index originally developed for rheumatoid arthritis, in BS patients. METHODS: Patients seen consecutively at a tertiary Rheumatology Centre were requested to complete BSAS and multidimensional health assessment questionnaire (MDHAQ). Besides, all attending physicians filled the Behcet's Disease Current Activity Form (BDCAF). Descriptive statistics and Pearson correlation coefficients were calculated accordingly for the reliability and validity assessments of BSAS. RESULTS: A total of 104 patients completed all three assessments. The test-retest reliability of BSAS has a good level (ICC=0.84, 95% CI [0.69-0.94]). The mean scores for BSAS, BDCAF and RAPID3 were 39+/-20.8, 3.2+/-1.4 and 9.2+/-5.6, respectively. BSAS was correlated with BDCAF moderately (r=0.587), while it was moderately correlated with RAPID3 (r=0.648). The correlation between the RAPID3 and BDCAF was moderate (r=0.403), but lower as compared to the correlations between the other instruments. CONCLUSIONS: We found that the BSAS has modest correlation with BDCAF and is a reliable and valid patient reported measure of disease activity that can be used to assess patients with BS. An outcome score composed of only patient-derived observations may have the additional advantage of being easier to use in a routine care setting. Demonstration of a moderate level of correlation between RAPID3 and BDCAF (close to the level of weak relationship), suggests that RAPID3 likely needs more investigations before recommending its use in BS.

OBJECTIVE: To analyze a patient self-report joint count from the Rheumatoid Arthritis Disease Activity Index (RADAI) on a Multidimensional Health Assessment Questionnaire (MDHAQ) in a cohort of consecutive patients seen in usual rheumatology care with rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), osteoarthritis (OA), psoriatic arthritis (PsA), and gout. METHODS: Each patient completed an MDHAQ, which included a RADAI, at each visit in one usual care setting. In order to include a physician measure, a random visit at which there was a recorded physician global estimate was selected for each of 465 patients (174 patients with RA, 75 with SLE, 113 with OA, 53 with PsA, and 50 patients with gout). The RADAI was analyzed for total scores (range 0-48), number of involved joint groups (range 0-16), and each specific joint group, and then compared in the 5 diagnostic groups to one another and to other MDHAQ measures and the Routine Assessment of Patient Index Data 3 (RAPID3). RESULTS: In patients with RA, SLE, OA, PsA, and gout, mean RADAI scores (range 0-48) were 12.4, 6.5, 10.1, 6.7, and 2.7, respectively. The mean numbers of involved joint groups (range 0-16) were 6.9, 3.8, 4.8, 4.5, and 1.7, respectively, and the median numbers were 6, 2, 4, 4, and 1, respectively. RADAI scores were correlated significantly with the physician global estimate, except in SLE, and at higher levels with the MDHAQ and RAPID3 scores in all diagnostic groups. CONCLUSION: The RADAI self-report joint counts can be used to record self-report involvement of specific joints and joint groups in patients with SLE, OA, PsA, and gout, with minimal effort on the part of the rheumatologist.

ABSTRACT: Rheumatoid arthritis treatment has seen major changes in the last decade, one of which is the concept of treating to target. Various composite outcome measures have been developed, and the latest is the new American College of Rheumatology/European League Against Rheumatism remission criteria. Zhang and colleagues test the predictive validity of the new criteria in an observational cohort and show that they work as well as other definitions of remission. Our main challenge remains getting rheumatologists to use one of the outcome measures rather than developing new measures that are basically no different from already available measures in predicting functional and radiographic changes, the two most important long-term outcomes of rheumatoid arthritis.

Methotrexate (MTX) is the anchor treatment for rheumatoid arthritis (RA) and has been very thoroughly studied in many different patient populations, as monotherapy and in combination with various other disease modifying antirheumatic drugs and biologic agents, as they became available. It has a well-established safety and efficacy profile and is the preferred first line agent for RA treatment. Historically, oral (PO) preparations of MTX have been used in the USA with minimal parenteral (subcutaneous, SC, and intramuscular, IM) administration. Several shortages of drug availability in a parenteral form have been possibly one of the reasons for this low level of use. Several studies have looked at the role of parenteral MTX in RA treatment, and these overall demonstrate better tolerability, bioavailability, and possible efficacy of MTX compared with PO preparation.

Behcet's syndrome (BS) is a vasculitis, seen more commonly around the Mediterranean and the Far East, and manifests with oral and genital ulcerations, skin lesions, uveitis, and vascular, central nervous system and gastrointestinal involvement. Its natural history of getting less severe over time, more severe disease in males and lack of specific diagnostic testing separates it from other commonly seen conditions in rheumatology. Most of the serious manifestations respond well to immunosuppression, and these are the mainstays of treatment for BS. BS is more prevalent in regions along the Silk Road, from the Mediterranean to the Far East. The genetic risk factor most strongly associated with BS is the human leukocyte antigen (HLA)-B51 allele. While genetic factors seem to play a role in the development of certain features of BS, there is general consensus that as yet unidentified environmental stimuli are necessary for initiation of disease. Proposed exogenous triggers include both bacterial and viral infections, which may then lead to dysregulation of the immune system, ultimately leading to the phenotypic expression of disease. The clinical manifestations of BS are protean in nature. While most patients develop mucocutaneous and genital ulcers along with eye disease, other patients may also present with arthritis, frank vasculitis, thrombophlebitis and CNS disease. Interestingly, the manifestations of this illness vary considerably based on gender and ethnicity. As the phenotypic expression among patients with BS is quite heterogeneous, pharmacological therapy is variable and dependent upon the severity of the disease as well as organ involvement. Treatment for BS overlaps considerably with therapies for other autoimmune diseases, including systemic lupus erythematosus, rheumatoid arthritis and the vasculitides. Pharmacological agents utilized for treatment of BS include corticosteroids, colchicine, azathioprine, and tumour necrosis factor (TNF).alpha inhibitors, among others. In this article, we review the salient clinical studies for each drug class along with important side effects as well as drug toxicity monitoring. Management of the patient with BS is complex and oftentimes requires a multidisciplinary approach. We discuss strategies to assess and stratify patients based on clinical manifestations and disease severity. A summary of drug toxicities as they relate to the aforementioned pharmacological agents, as well as guidelines regarding vaccinations in this patient population, are offered. Finally, we conclude with treatment strategies for the common manifestations of BS along with a discussion of the management of thrombotic disease in these patients.

OBJECTIVE: It is important to evaluate how a randomized controlled trial (RCT) sample was assembled from the general patient population in order to determine whether a patient differs from those who participated in the trial in a meaningful way. The aim of this study is to assess the adequacy of reporting of the recruitment process of rheumatoid arthritis (RA) patients participating in RCTs with biologic agents. METHODS: We searched PubMed for all reports of RCTs involving etanercept, infliximab, adalimumab, golimumab, certolizumab, abatacept, tocilizumab, and rituximab in RA patients. Data recorded were eligibility fraction, enrollment fraction, recruitment fraction, and number of patients needed to be screened (NNS) in order to randomize 1 participant. RESULTS: Of the 66 trials included in the analysis, 23 (35%) reported the number of individuals assessed by investigators for eligibility, and 18 (27%) reported the number eligible for participation. Of the studies that reported quantitative recruitment information, the median eligibility fraction was 80.6% (interquartile range [IQR] 71.8-91.1%) and the median enrollment fraction was 100% (IQR 88.4-100%). The median NNS was found to be 1.28 (IQR 1.18-1.43). CONCLUSION: A substantial majority of RCTs conducted in RA patients with biologic agents did not provide sufficient information about the patient recruitment process, which makes assessments of external validity difficult. The rate of reporting of the recruitment process in this study was found to be lower as compared to similar studies conducted in different specialties.