Multi-centre national audit of juvenile localised scleroderma:Describing current UK practice in disease assessment and management

OBJECTIVE: To describe current United Kingdom practice in assessment and management of patients with juvenile localised scleroderma (JLS) compared to Paediatric Rheumatology European Society (PRES) scleroderma working party recommendations. METHODS: Patients were included if they were diagnosed with JLS and were under the care of paediatric rheumatology between 04/2015-04/2016. Retrospective data was collected in eleven UK centres using a standardised proforma and collated centrally. RESULTS: 149 patients were included with a median age of 12.5 years. The outcome measures recommended by the PRES scleroderma working party were not utilised widely. The localised scleroderma cutaneous assessment tool was only used in 37.6% of patients. Screening for extracutaneous manifestations did not meet recommendations that patients with head involvement have regular screening for uveitis and baseline magnetic resonance imaging (MRI) brain: only 38.5% of these patients were ever screened for uveitis; 71.2% had a MRI brain. Systemic treatment with disease-modifying anti-rheumatic drugs (DMARDs) or biologics was widely used (96.0%). In keeping with the recommendations, 95.5% of patients were treated with methotrexate as first-line therapy. 82.6% received systemic corticosteroids and 34.2% of patients required two or more DMARDs/biologics, highlighting the significant treatment burden. Second-line treatment was mycophenolate mofetil in 89.5%. CONCLUSION: There is wide variation in assessment and screening of patients with JLS but a consistent approach to systemic treatment within UK paediatric rheumatology. Improved awareness of PRES recommendations is required to ensure standardised care. As recommendations are based on low level evidence and consensus opinion, further studies are needed to better define outcome measures and treatment regimens for JLS

Management and treatment of children, young people and adults with systemic lupus erythematosus: British Society for Rheumatology guideline scope

Lay Summary: Systemic lupus erythematosus (SLE) is a lifelong condition in which the immune system damages the body’s own tissues, causing various symptoms including rashes, hair loss, mouth ulcers, joint pain and overwhelming tiredness. It can also affect major organs including the kidneys, heart, lungs and brain. SLE can present during childhood, but most commonly affects young and middle-aged females. It is approximately nine times more common in females than males. The disease can lead to disability, poor quality of life and even death in severe cases. Treatments can often be difficult to tolerate and can cause both short- and long-term side effects. Guidelines developed by the British Society for Rheumatology aim to provide guidance for diagnosing and treating people with SLE. This is necessary to ensure that the most up-to-date approach is followed, utilising the safest and most effective treatments. This article describes the plan for a guideline in SLE that is being updated to cover new evidence that has been published since 2017 relating to the treatment and management of SLE. The guideline will take a whole life course approach, from childhood to adulthood, and is being undertaken by a working group consisting of paediatric and adult rheumatologists and nephrologists, SLE experts, general practitioners, specialist nurses and other healthcare professionals, together with people with SLE and representatives from patient organizations. The guideline will be developed using the methods and processes outlined in the British Society for Rheumatology document ‘Creating Clinical Guidelines: Our Protocol’

Unilateral urinary flow impairment at the pelviureteral junction: outcome of renal function with respect to therapeutic strategy.

OBJECTIVES: To evaluate the renal function outcome in children with unilateral hydronephrosis and urinary flow impairment at the pelviureteral junction with respect to the therapeutic strategy. METHODS: We retrospectively selected 45 children with iodine-123-hippuran renography performed at diagnosis and after 3 or more years of follow-up. All children had bilateral nonobstructive pattern findings on diuretic renography at follow-up. Eleven children were treated conservatively, and 34 underwent unilateral pyeloplasty. Split and individual renal function, measured by an accumulation index, was computed from background-corrected renograms for the affected and contralateral kidneys at diagnosis and the follow-up examination. RESULTS: Of 11 children treated conservatively, 9 had normal bilateral function at diagnosis, all had reached normal function at follow-up. Of the 34 operated kidneys, 12 (38%) had initially normal function that remained normal at the follow-up examination, and 22 had impaired function that had normalized at the follow-up examination in 15 (68%). The function of the contralateral kidneys was increased in 5 of 8 children with persistently abnormal affected kidneys. Pyeloplasty was performed in 23 children (68%) and 11 children (32%) younger and older than 1 year, respectively. The function of the affected kidneys increased in both groups, but normalization occurred only in the younger children. CONCLUSIONS: Of the children selected for conservative treatment, 82% had normal bilateral renal function at diagnosis that was normal in all at the follow-up examination. Of the children treated surgically, 65% had initially impaired function of the affected kidney that improved in 87% after pyeloplasty. Normalization of function was observed only in children who were younger than 1 year old at surgery. Persistently low function of the affected kidney was compensated for by the contralateral one regardless of the age at surgery