Objective. To describe an enthesitis-related arthritis (ERA) inception cohort and determine which entheses and joints are most commonly affected. Methods. We reviewed a retrospective inception cohort study of children with ERA who were diagnosed and treated at The Children's Hospital of Philadelphia between November 2007 and December 2009. Results. During the study period, there were 32 newly diagnosed ERA patients. Fifty-nine percent were male, and the median age at the date of initial evaluation was 12.5 years (interquartile range [IQR] 10.2-14.3 years). The median number of tender entheses at presentation was 2 (IQR 0 -5), and 21 subjects (66%) had at least 1 tender enthesis. The most prevalent tender entheses were the patellar ligament insertion at the inferior pole of the patella, the plantar fascial insertion at the calcaneus, the Achilles tendon insertion at the calcaneus, and the plantar fascial insertion at the metatarsal heads. Enthesitis was most often symmetric. The median number of active joints was 2 (IQR 0 -4). The most commonly affected joints were the sacroiliacs, knees, and ankles. Sacroiliitis, which was defined clinically, was most often symmetric, while peripheral arthritis was most frequently asymmetric. The odds of having active enthesitis at 6 months increased significantly with each additional tender enthesis at the initial evaluation. Conclusion. Among pediatric patients with ERA, lower extremity enthesitis is prevalent at the time of diagnosis and is likely to persist 6 months later. Future studies should address standardization of the enthesitis examination, the pattern of enthesitis over time, enthesitis response to therapy, and the impact of enthesitis on quality of life.

OBJECTIVE To characterize the effect of corticosteroid exposure on clinical outcomes in children hospitalized with new-onset Henoch-Schönlein purpura (HSP). PATIENTS AND METHODS We conducted a retrospective cohort study of children discharged with an International Classification of Diseases, Clinical Modification code of HSP between 2000 and 2007 by using inpatient administrative data from 36 tertiary care children’s hospitals. We used stratified Cox proportional hazards regression models to estimate the relative effect of time-varying corticosteroid exposure on the risks of clinical outcomes that occur during hospitalization for acute HSP. RESULTS During the 8-year study period, there were 1895 hospitalizations for new-onset HSP. After multivariable regression modeling adjustment, early corticosteroid exposure significantly reduced the hazard ratios for abdominal surgery (0.39 [95% confidence interval (CI): 0.17– 0.91]), endoscopy (0.27 [95% CI: 0.13– 0.55]), and abdominal imaging (0.50 [95% CI: 0.29 – 0.88]) during hospitalization. CONCLUSIONS In the hospital setting, early corticosteroid exposure was associated with benefits for several clinically relevant HSP outcomes, specifically those related to the gastrointestinal manifestations of the disease.

Objective-To describe variation regarding inpatient therapy and evaluation of children with Henoch Schönlein purpura (HSP) admitted to children's hospitals across the United States.Study design-We conducted a retrospective cohort study of children discharged with a diagnosis of HSP between 2000 and 2007 using inpatient administrative data from 36 children's hospitals. We examined variation among hospitals in the use of medications, diagnostic tests, and intensive care services using multivariate mixed effects logistic regression models.Results-During the initial HSP hospitalization (N=1,988), corticosteroids were the most common medication (56% of cases), followed by opioids (36%), NSAIDs (35%), and anti-hypertensives (11%). After adjustment for patient characteristics, hospitals varied significantly in their use of corticosteroids, opioids, and NSAIDs; the use of diagnostic abdominal imaging, endoscopy, laboratory testing, and renal biopsy; and the utilization of intensive care services. By contrast, hospitals did not differ significantly regarding administration of anti-hypertensives or performance of skin biopsy.Conclusions-The significant variation identified may contribute to varying HSP clinical outcomes between hospitals, warrants further investigation, and represents a potentially important opportunity to improve quality of care. Keywordsopioids; corticosteroids; anti-hypertensives; non-steroidal anti-inflammatory drugs; adolescents; epidemiology © 2009 Mosby, Inc. All rights reserved.Address for correspondence: Dr. Pamela F. Weiss, Room 1539, North Campus, Division of Rheumatology, The Children's Hospital of Philadelphia, 3535 Market Street, Philadelphia, PA 19104. Fax: (215) 426-2303. weisspa@email.chop.edu.. Publisher's Disclaimer: This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final citable form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.The authors declare no conflicts of interest. Prior epidemiologic studies about the natural course and treatment of HSP are limited by incomplete follow-up over variable time intervals, selection bias, and varying definitions of disease severity and complications. These limitations are evidenced by widely disparate estimates of the short-and long-term morbidities associated with HSP and varying conclusions regarding different therapeutic algorithms. Further, there are no established guidelines for the management of HSP from professional societies. Current inpatient therapy for HSP may include one or a combination of the following: opioids, corticosteroids, non-steroidal antiinflammatory drugs (NSAIDs), and anti-hypertensive agents. The frequencies at which these medications are used and the variatio...

Much has been written about real-world evidence (RWE) in scientific papers and the lay media. Although some researchers and journalists tout its value, opponents are vocal in challenging its validity, pointing out shortcomings and downplaying any potential benefits. Despite an emphasis on the importance of RWE in the 21st Century Cures Act, a standard definition of the term has not been uniformly embraced. 1 The US Food and Drug Administration defines RWE as "data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials." 2,3 In addition, the agency's Real-world Evidence Program framework defines real-world data (RWD) as "data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources." 4(p4) To maximize the potential of RWE and define its role moving forward, limitations must be balanced with merits when considering RWD sources (Table ).Real-world evidence is obtained from RWD, which encompass data collected outside randomized clinical trials (RCTs), ideally in diverse groups such as those representing all Americans, all Europeans, or all global citizens. Real-world evidence can be derived through routine daily clinical practice or through patient interactions in their customary daily living environment. Because greater than 90% of patients with cancer are treated outside RCTs, 5 RWE presents a unique avenue for obtaining data on patients with characteristics outside those typically required for trial eligibility. Randomized clinical trials that lead to Food and Drug Administration approval of drug therapies often enroll younger patients who lack comorbidities, have adequate organ functions, possess proper psychosocial support, are able to travel to study sites, and are white. 2 Generation of RWE allows assessing how therapy affects a heterogeneous population-those who are older and those with concurrent medical problems, impaired organ function, limited social resources, and varied ethnicities/races.Moreover, the Food and Drug Administration uses RWE and RWD to monitor postmarketing safety and adverse events, which may go unnoticed during RCTs. These findings can have regulatory and strategy implications for payers, physicians, and manufacturers. RWD Sources Administrative ClaimsThese administrative data sets contain patient medical and pharmacy claims generated for health care encounters and submitted for reimbursement from government and commercial payers. Medical and pharmacy claims contain coded data that capture patient demographics, diagnosis, procedures, and medications, as well as associated dates, place of ser-vice, and costs for each claim. When these claims are linked to a given patient over time, a longitudinal history that depicts his or her journey through the health care system can be created.

We found low levels of TPE use across hospitals for key indications, including TTP, a condition for which TPE is considered a first-line and life-saving procedure. The variation identified may contribute to varying clinical outcomes between hospitals, warrants further investigation, and represents an important opportunity to improve quality of care.