Rituximab Treatment in Myasthaenia Gravis: Report of two paediatric cases

Myasthaenia gravis (MG) is an autoimmune disease involving the postsynaptic receptors in the neuromuscular junction. The condition is characterised by fatigable weakness of the skeletal muscles and is uncommon in children. Acetylcholinesterase inhibitors and immune-modifying medications are usually considered the mainstay of treatment. However, these medications have to be given on a lifelong basis so that patients remain in remission; furthermore, drug-related side-effects can have a major impact on quality of life. We report two paediatric cases who were treated for MG at the Sultan Qaboos University Hospital, Muscat, Oman, in 2007 and 2008, respectively. Rituximab was eventually administered to each patient after their condition failed to improve despite several years of standard treatment with acetylcholinesterase inhibitors and immune-modifying medications. Overall, rituximab resulted in complete remission in one case and significant clinical improvement in the other case. Keywords: Myasthenia Gravis; Rituximab; Children; Cholinergic Receptors; Case Report; Oman

The Omani Arabic version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR)

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Omani Arabic language. The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their parents. The statistical validation phase explored descriptive statistics and the psychometric issues of the JAMAR: the 3 Likert assumptions, floor/ceiling effects, internal consistency, Cronbach\u2019s alpha, interscale correlations, test\u2013retest reliability, and construct validity (convergent and discriminant validity). A total of 57 JIA patients (22.8% systemic, 28.1% oligoarticular, 35.1% RF negative polyarthritis, 14.0% other categories) and 85 healthy children, were enrolled in two centres. Notably, none of the enrolled JIA patients is affected with enthesitis-related arthritis or undifferentiated arthritis. The JAMAR components discriminated well healthy subjects from JIA patients. All JAMAR components revealed satisfactory psychometric performances. In conclusion, the Omani Arabic version of the JAMAR is a valid tool for the assessment of children with JIA and is suitable for use both in routine clinical practice and clinical research

An adolescent with both Wegener's Granulomatosis and chronic blastomycosis

We report a case of Wegener's Granulomatosis (WG) associated with blastomycosis. This appears to be the first case report of WG co-existing with a tissue proven blastomycosis infection. The temporal correlation of the two conditions suggests that blastomycosis infection (and therefore possibly other fungal infections), may trigger the systemic granulomatous vasculitis in a predisposed individual; a provocative supposition warranting further study

Proceedings of the 24th Paediatric Rheumatology European Society Congress: Part three

From Springer Nature via Jisc Publications Router.Publication status: PublishedHistory: collection 2017-09, epub 2017-09-0

Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: Analysis by the Pharmachild Safety Adjudication Committee

Background: To derive a list of opportunistic infections (OI) through the analysis of the juvenile idiopathic arthritis (JIA) patients in the Pharmachild registry by an independent Safety Adjudication Committee (SAC). Methods: The SAC (3 pediatric rheumatologists and 2 pediatric infectious disease specialists) elaborated and approved by consensus a provisional list of OI for use in JIA. Through a 5 step-procedure, all the severe and serious infections, classified as per MedDRA dictionary and retrieved in the Pharmachild registry, were evaluated by the SAC by answering six questions and adjudicated with the agreement of 3/5 specialists. A final evidence-based list of OI resulted by matching the adjudicated infections with the provisional list of OI. Results: A total of 772 infectious events in 572 eligible patients, of which 335 serious/severe/very severe non-OI and 437 OI (any intensity/severity), according to the provisional list, were retrieved. Six hundred eighty-two of 772 (88.3%) were adjudicated as infections, of them 603/682 (88.4%) as common and 119/682 (17.4%) as OI by the SAC. Matching these 119 opportunistic events with the provisional list, 106 were confirmed by the SAC as OI, and among them infections by herpes viruses were the most frequent (68%), followed by tuberculosis (27.4%). The remaining events were divided in the groups of non-OI and possible/patient and/or pathogen-related OI. Conclusions: We found a significant number of OI in JIA patients on immunosuppressive therapy. The proposed list of OI, created by consensus and validated in the Pharmachild cohort, could facilitate comparison among future pharmacovigilance studies. Trial registration: Clinicaltrials.gov NCT 01399281; ENCePP seal: awarded on 25 November 2011

Challenges of Childhood Uveitis

Chronic uveitis is a rare, but potentially sight-threatening disease. The most common cause of chronic non-infectious uveitis is “idiopathic uveitis”. However, some systemic diseases are associated with chronic uveitis in children and are discussed. Chronic uveitis merits special consideration in children. The unique differences in children are highlighted with special consideration for the diagnostic and therapeutic challenges encountered in their management. While corticosteroids remain the mainstay of initial therapy, a wide range of immunosuppressive agents have been used with variable success. The role of immonomodulatory agents such as methotrexate, cyclosproin and some of the new biologic agents such as etanecept, infliximab, adalimumab are reviewed. Successful outcomes may be achieved with appropriate immunosuppressant therapy when given early in the disease, although clinical trials are required to define the true efficacy of this strategy

Challenges of Childhood Uveitis

Chronic uveitis is a rare, but potentially sight-threatening disease. The most common cause of chronic non-infectious uveitis is “idiopathic uveitis”. However, some systemic diseases are associated with chronic uveitis in children and are discussed. Chronic uveitis merits special consideration in children. The unique differences in children are highlighted with special consideration for the diagnostic and therapeutic challenges encountered in their management. While corticosteroids remain the mainstay of initial therapy, a wide range of immunosuppressive agents have been used with variable success. The role of immonomodulatory agents such as methotrexate, cyclosproin and some of the new biologic agents such as etanecept, infliximab, adalimumab are reviewed. Successful outcomes may be achieved with appropriate immunosuppressant therapy when given early in the disease, although clinical trials are required to define the true efficacy of this strategy

Growth Pattern in Children with Systemic Lupus Erythematosus

Objectives: Children with childhood-onset systemic lupus erythematosus (cSLE) enter adulthood with considerable morbidity. Of the recognized morbidities, growth failure is unique to cSLE. The aim of this study was to evaluate the growth pattern in children with cSLE longitudinally and identify possible risk factors. Methods: Serial anthropometric measurements of cSLE patients were obtained over two years and expressed as z-scores. Parental heights were obtained to calculate target height. Parent-adjusted height z-score was calculated as the difference between height z-score and target height. Growth failure was defined as parent-adjusted height z-score < -1.50. Risk factors that might have contributed to growth failure were evaluated including the presence of growth failure at baseline, disease activity, disease duration, and cumulative steroid doses. Results: Twenty-five patients were included in the study. Growth failure was observed in eight patients with an overall incidence of 32.0% (95% confidence interval (CI): 14–50%). When comparing the cohort with and without growth failure, the factors that determined growth failure was the pre-existence of growth failure at the time of diagnosis (z-score < -1.95 vs. 0.35; p < 0.001); higher cumulative steroid dose (15.8 vs. 9.1 g ; p = 0.061); and tendency for longer disease duration (5.4 vs. 3.7 years; p = 0.240). However, the severity of disease activity at the time of diagnosis was not a significant contributing factor (12 vs. 14; p = 0.529). Conclusions: Children with cSLE are at risk of having a negative effect on height including patients with pre-existing growth failure, high cumulative steroid dose, and longer disease duration. However, longitudinal prospective studies are needed to examine damage over time to improve health-related quality of life