Lakshmi N. Moorthy

Physical function assessment tools in pediatric rheumatology

Pediatric rheumatic diseases with predominant musculoskeletal involvement such as juvenile idiopathic arthritis (JIA) and juvenile dermatomyositis(JDM) can cause considerable physical functional impairment and significantly affect the childrens quality of life (QOL). Physical function, QOL, health-related QOL (HRQOL) and health status are personal constructs used as outcomes to estimate the impact of these diseases and often used as proxies for each other. The chronic, fluctuating nature of these diseases differs within and between patients, and complicates the measurement of these outcomes. In children, their growing needs and expectations, limited use of age-specific questionnaires, and the use of proxy respondents further influences this evaluation. This article will briefly review the different constructs inclusive of and related to physical function, and the scales used for measuring them. An understanding of these instruments will enable assessment of functional outcome in clinical studies of children with rheumatic diseases, measure the impact of the disease and treatments on their lives, and guide us in formulating appropriate interventions.

Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's): An ARChiVe Cohort Study

To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegeners) (GPA).

Comparing presenting clinical features of 48 children with microscopic polyangiitis (MPA) against 183 having granulomatosis with polyangiitis (GPA). An ARChiVe study

To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegeners) (GPA).

Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

To characterize the early disease course in childhood‐onset antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (AAV) and the 12‐month outcomes in children with AAV.

Novel Method to Collect Medication Adverse Events in Juvenile Arthritis: Results From the Childhood Arthritis and Rheumatology Research Alliance Enhanced Drug Safety Surveillance Project

Few data are available regarding the rates of serious adverse events (SAEs) and important medical events (IMEs) outside of product‐based registries and clinical trials for juvenile idiopathic arthritis (JIA). The Enhanced Drug Safety Surveillance Project (EDSSP) was developed to pilot a novel system to collect SAEs/IMEs in children with JIA. This analysis reports the results from this 4‐year (2008–2012) EDSSP.

Efficacy of an Interinstitutional Mentoring Program Within Pediatric Rheumatology.

The small size of many pediatric rheumatology programs translates into limited mentoring options for early career physicians. To address this problem, the American College of Rheumatology (ACR) and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed a subspecialty‐wide interinstitutional mentoring program, the ACR/CARRA Mentoring Interest Group (AMIGO). We sought to assess the impact of this program on mentoring within pediatric rheumatology.

Efficacy of the AMIGO inter‐institutional mentoring program within pediatric rheumatology

The small size of many pediatric rheumatology programs translates into limited mentoring options for early career physicians. To address this problem, the American College of Rheumatology (ACR) and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed a subspecialty‐wide interinstitutional mentoring program, the ACR/CARRA Mentoring Interest Group (AMIGO). We sought to assess the impact of this program on mentoring within pediatric rheumatology.

Health related quality of life measure in systemic pediatric rheumatic diseases and its translation to different languages: an international collaboration

BackgroundRheumatic diseases in children are associated with significant morbidity andpoor health-related quality of life (HRQOL). There is no health-relatedquality of life (HRQOL) scale available specifically for children with lesscommon rheumatic diseases. These diseases share several features withsystemic lupus erythematosus (SLE) such as their chronic episodic nature,multi-systemic involvement, and the need for immunosuppressive medications.HRQOL scale developed for pediatric SLE will likely be applicable tochildren with systemic inflammatory diseases.FindingsWe adapted Simple Measure of Impact of Lupus Erythematosus in Youngsters(SMILEY©) to Simple Measure of Impact of Illness in Youngsters(SMILY©-Illness) and had it reviewed by pediatric rheumatologists forits appropriateness and cultural suitability. We tested SMILY©-Illnessin patients with inflammatory rheumatic diseases and then translated it into28 languages.Nineteen children (79% female, n=15) and 17 parents participated. The meanage was 12±4 years, with median disease duration of 21 months (1-172months). We translated SMILY©-Illness into the following 28 languages:Danish, Dutch, French (France), English (UK), German (Germany), German(Austria), German (Switzerland), Hebrew, Italian, Portuguese (Brazil),Slovene, Spanish (USA and Puerto Rico), Spanish (Spain), Spanish(Argentina), Spanish (Mexico), Spanish (Venezuela), Turkish, Afrikaans,Arabic (Saudi Arabia), Arabic (Egypt), Czech, Greek, Hindi, Hungarian,Japanese, Romanian, Serbian and Xhosa.ConclusionSMILY©-Illness is a brief, easy to administer and score HRQOL scale forchildren with systemic rheumatic diseases. It is suitable for use acrossdifferent age groups and literacy levels. SMILY©-Illness with itsavailable translations may be used as useful adjuncts to clinical practiceand research.

Successful Treatment of Henoch-Schönlein Purpura With Recurrent Gastrointestinal Involvement With Mycophenolate Mofetil A Brief Report

An 8-year old boy, previously healthy, presented with an upper respiratory tract infection and wheezing, for which he was treated with cefdinir, prednisone, and albuterol. He recovered, and 3 days after completing treatment, “spots” appeared over his legs and buttocks. Rapid strep test returned positive and the patient was started on antibiotic therapy. The patient subsequently developed nausea, bilious vomiting, and abdominal pain and returned to the emergency room where he was treated with intravenous antibiotics and fluids and was discharged in stable condition. He was admitted the next day with severe abdominal pain, vomiting and rectal bleeding. Exam was notable for a widespread petechial and purpuric rash and right elbow arthritis. He was diagnosed with Henoch-Schönlein purpura (HSP). Imaging (abdominal ultrasound and computed tomography) was negative for intussusception. He improved symptomatically with intravenous corticosteroids and was discharged on esomeprazole and an oral prednisolone 1.25 mg/kg/d tapered over 7 days. Three days later, he developed joint pain in his knees and ankles, lower abdominal pain with bloody diarrhea, and continued purpura. He improved over time with supportive care and steroids. Abdominal ultrasound showed no evidence for intussusception. He was discharged on esomeprazole and advised to avoid nonsteroidal anti-inflammatory drugs. Following discharge he continued to have weekly episodes of joint pain and purpuric rash, lasting 1 or 2 days at a time.

Abdominal Pain, Rash, and Arthritis:

A 13-year-old Caucasian female with no significant medical or surgical history presented to the emergency department with a 5-month history of polyarthralgias, 2-week history of abdominal pain, and 4-day history of severe right ankle arthritis. The patient reported that the right ankle pain was associated with swelling, erythema, and warmth of her right posterior ankle. Approximately 2 weeks prior to presentation, the patient began to feel severe fatigue and malaise while on a family vacation. At that time, she also developed lower abdominal pain with nausea. Since that time, the patient reported that she had been unable to eat solid foods secondary to pain and had lost 5 pounds. She denied vomiting, diarrhea, and constipation. At that time, patient’s primary care physician had ordered outpatient labs. Complete blood count, comprehensive metabolic panel, antinuclear antibody, rheumatoid factor, anti-doublestranded DNA antibodies, erythrocyte sedimentation rate, C-reactive protein, and complement components 3 and 4 were all within normal limits. Approximately 1 week prior to presentation, she developed an erythematous and maculopapular scaly rash over her elbows and fevers ranging from 100.4°F to 100.7°F. She also developed left subconjunctival hemorrhage. The patient denied changes in hearing or vision, recent illness, recent travel, headache, vomiting, diarrhea, constipation, hematochezia, melena, and dysuria. Laboratory values on admission (Table 1) were significant for an elevated erythrocyte sedimentation rate, hematuria, and proteinuria.