Faculty Bibliography

Juvenile idiopathic arthritis (JIA) includes several forms of chronic arthritis in childhood with no apparent cause. JIA is the most common rheumatic disease in children, and may result in pain, joint deformity, and growth im- pairment, with possible persistent active arthritis into adulthood. Prior treatment involved non-specific agents, several with significant adverse effects. The recent use of biologics now provides target-specific therapy, which may be better tolerated. Through continued translational research and clinical trials, one better understands the biology mediating disease, with the hope of offering safer, more effective medicine, and potential cure. This review will outline the clinical features of JIA, as well as provide the latest updates in treatment.

OBJECTIVE: To use consensus methods and the considerable expertise contained within the Childhood Arthritis and Rheumatology Research Alliance (CARRA) organization to extend the 3 previously developed treatment plans for moderate juvenile dermatomyositis (DM) to span the full course of treatment. METHODS: A consensus meeting was held in Chicago on April 23-24, 2010, involving 30 pediatric rheumatologists and 4 lay participants. Nominal group technique was used to achieve consensus on treatment plans that represented typical management of moderate juvenile DM. A preconference survey of CARRA, completed by 151 (56%) of 272 members, was used to provide additional guidance to the discussion. RESULTS: Consensus was reached on timing and rate of steroid tapering, duration of steroid therapy, and actions to be taken if patients were unchanged, worsening, or experiencing medication side effects or disease complications. Of particular importance, a single consensus steroid taper was developed. CONCLUSION: We were able to develop consensus treatment plans that describe therapy for moderate juvenile DM throughout the treatment course. These treatment plans can now be used clinically, and data collected prospectively regarding treatment effectiveness and toxicity. This will allow comparison of these treatment plans and facilitate the development of evidence-based treatment recommendations for moderate juvenile DM.

ABSTRACT: OBJECTIVE: Behcet's is an idiopathic multi-organ syndrome, which may have onset during childhood. Vascular involvement is uncommon, with rarely reported coronary aneurysm formation. We present a case report of a teenager girl who developed recalcitrant life-threatening Behcet's vasculitis, involving both small and large venous and arterial systems including a giant coronary aneurysm. CASE REPORT: De-identified data were collected retrospectively in case report format. Although our sixteen year old female with Behcet's vasculitis had resolution of many arterial aneurysms, she had persistent venous thrombosis of large vessels, as well as persistent, giant arterial aneurysms requiring intra-arterial coiling of a lumbar artery and coronary bypass grafting despite intensive immunosuppression including glucocorticoids, cyclophosphamide, infliximab, methotrexate, azathioprine and intravenous immunoglobulin. CONCLUSIONS: Vascular manifestations may be seen in Behcet's syndrome, including asymptomatic coronary aneurysm, which may be refractory to immunosuppression and ultimately require surgical intervention. Increased awareness is essential for prompt diagnosis and management.

A 20-year-old woman presented with mental retardation and a history of stroke related to moyamoya disease at the age of 8 years. She had cognitive impairment which became more pronounced after the stroke. This patient's parents were first cousins and six close family relatives had strokes in their 60s or 70s. The patient's 16-year-old sister had learning disability, chronic muscle pain, and an ECG suggestive of previous hypoxemic heart injury. The two sisters had similar dysmorphic facial appearance including a prominent philtrum, bulbous nose, and severe acne. They both had increased subcutaneous tissue in their faces, whereas their bodies were slim. Both sisters were found to have elevated levels of rheumatoid factor, C-reactive protein, and erythrocyte sedimentation rate on repeat measurements. Partial autoimmunity screening in one of the patients was negative. Chromosome analysis and array comparative genomic hybridization analyses were also normal. Nerve conduction findings in the younger sister were consistent with distal, predominantly motor, demyelinating neuropathy localized to the lower extremities. We propose that these two sisters suffer from a new autosomal recessive syndrome. Carrier status for this condition may predispose to later onset stroke.

Juvenile idiopathic arthritis (JIA) comprises a collection of all forms of chronic arthritis in childhood with no apparent cause. JIA is the most common rheumatic disease in children, and may result in significant pain, joint deformity, and growth impairment, with persistence of active arthritis into adulthood. Prior to the mid 1990s, the therapeutic armamentarium for JIA was more limited, utilizing non- specific agents, many with significant adverse effects. With the relatively recent use of biologics, one can provide more target-specific therapy, which may be better tolerated. Through continued translational research and clinical trials, one better understands the biology mediating disease, with the hope of offering safer, more effective medicine, and potential cure. This review will outline the clinical features of JIA, as well as provide the latest updates in current and future pharmacotherapy.

The objective of the study was to compare clinical features, treatment and disease outcome in patients with early versus later onset of childhood-onset systemic lupus erythematosus (cSLE). A retrospective matched cohort study of cSLE patients diagnosed between 1988 and 2008 and followed for a minimum of one year was conducted. Thirty-four pre-pubertal cSLE patients with disease onset prior to their 12th birthday were matched by ethnicity and year of diagnosis to 34 pubertal cSLE patients. The most common criteria at diagnosis in both groups were malar rash, arthritis, hematologic manifestations, and renal disease. After a mean follow-up of more than six years, a similar proportion of patients in the two groups were still prescribed corticosteroids (47% and 41%); patients in the early onset group required a significantly higher daily dose (0.6 mg/kg prednisone-equivalent versus 0.2 mg/kg, p < 0.05). There were no significant differences in organ involvement, disease activity and disease damage between the two groups, and severe complications occurred at similar rates. There were a greater number of admissions to the pediatric intensive care unit (PICU) in the early onset group (18 versus 5, p = 0.01), with time-to-event analysis demonstrating a significantly shorter disease duration from diagnosis to first PICU admission in the early onset group (p < 0.001). While a similar proportion of patients in the early and later onset groups required treatment with cyclophosphamide, patients in the early onset group received treatment earlier in their disease course (mean 13.7 versus 19.9 months, p < 0.001). Early onset cSLE leads to earlier and more frequent PICU admission, earlier use of cyclophosphamide, and higher corticosteroid dose at long-term follow-up

OBJECTIVE: To assess the prevalence of neurocognitive impairment (NCI) in childhood-onset systemic lupus erythematosus (cSLE) by comparing published classification criteria, and to examine associations between NCI, disease characteristics, psychosocial well-being, and intelligence. METHODS: cSLE patients and ethnicity- and age-matched healthy controls completed a neuropsychological research battery, and results were categorized by 3 different NCI classification criteria with different cutoff scores (e.g., >2, 1.5, or 1 SD below the mean) and the number of required abnormal tests or domains. RESULTS: Forty-one cSLE subjects and 22 controls were included. Subjects were predominantly female (~70%) and Hispanic ( approximately 70%). Executive functioning, psychomotor speed, and fine motor speed were most commonly affected. Method 1 classified 34.1% of cSLE subjects with NCI compared to method 2 (14.6% with decline and 7.3% with NCI) and method 3 (63.4% with NCI). The prevalence of NCI was not significantly different between the controls and patients using any of the categorization methods. NCI was not associated with SLE disease activity or characteristics or with depression. Using method 3, patients in the cognitive impairment group reported significantly lower quality of life estimates (69.7 versus 79.3; P = 0.03). Below average intellectual functioning (intelligence quotient <90) differentiated the number of test scores >1 and >1.5 SDs, but not >2 SDs below the mean. CONCLUSION: NCI was prevalent in cSLE, but varied according to the chosen categorization method. A similar proportion of cSLE patients and controls had NCI, reinforcing the importance of studying an appropriate control group. Categorical classification (i.e., impaired/nonimpaired) may oversimplify the commonly observed deficits in cSLE

Abstract Behcet's disease is a rare autoimmune vasculitis that may cause coronary artery aneurysms. We discuss the evaluation and management decisions for a 19-year-old female with a giant rapidly expanding aneurysm of the proximal left anterior descending coronary artery and Behcet's disease. (J Card Surg 2011;26:268-270)

Juvenile Idiopathic Arthritis (JIA) consists of a collection of all forms of chronic arthritis in childhood with no apparent cause. JIA is the most common rheumatic disease in children and may result in significant pain, joint deformity, and growth impairment, with persistence of active arthritis into adulthood. The extra-articular features of JIA, such as anterior uveitis or macrophage activation syndrome, are often the greater focus of therapy. Prior to the mid 1990s, the therapeutic armamentarium for JIA was more limited, utilizing non-specific agents, many with significant adverse effects. In the current era of target-specific biologic therapy, it is possible to better tailor therapy for patients. Through continued translational research and clinical trials, the biology mediating disease is better understood, and there is the hope of safer, more effective medicine and potential cure. This review will outline the clinical features of JIA as well as provide the latest updates in current and future pharmacotherapy