Search

Background Granulomatosis with polyangiitis (GPA) is a rare disease in childhood. Treatment strategies and clinical approach are still mostly derived from adult GPA studies. Objectives The present study was aimed to compare disease onset and course, therapeutic approach and clinical outcome of two cohorts of children and adults affected by GPA. Methods This retrospective study included children and adult patients selected on the basis of complete data set and follow up of at least one year, followed at the Pediatric and Adult Rheumatology Centers, University Hospital of Padua, over a period of 20 years (1993-2013). GPA was diagnosed according to the ACR and EULAR/PRES criteria. Clinical features, instrumental findings, laboratory parameters and therapeutic regimens of both cohorts were analyzed at diagnosis, six months later (T6) and at the last follow-up visit. Disease activity was assessed by the Birmingham Vasculitis Activity Score (BVAS) modified for GPA. Results 9 children with mean age at disease onset of 10.3 years (range 3-15) entered the study, 6/9 were female. Mean follow-up time was 8.4 years (range 2.5-18). Of the 23 adults 65.3% were female, mean age at onset 53 years (range 37-71) and mean follow-up 1.9 years (range 1-3.5). At disease onset, BVAS, clinical features and laboratory parameters of the two cohorts were not significantly different. BVAS decreased more slowly in children (p 0.002). As for internal organs involvement, renal disease was significantly higher at T6 and persisted over time in children (p 0.003). Similarly, pulmonary disease remained elevated at T6 while decreased over 50% in adults (p 0.01). At the last F/U visit, eye involvement was present in 44% of children while no adult showed signs of ocular disease (p 0.004). Regarding to treatment strategies, immunosoppressors were more widely used in children at diagnosis (p 0.06) and biological agents were used at an earlier stage of the disease than adults. Despite the longer follow up children were still undergoing treatment at the last visit while 17% of adults were off therapy. Conclusions According to the BVAS results, adults and children had similar disease activity at onset. However, childhood GPA had a more severe-course due to persistent renal, pulmonary and eye involvement. Lower disease activity was obtained with a more aggressive treatment approach although no pediatric patients reached a treatment-free remission. References Ozen et al. EULAR/PRINTO/PRES criteria for Henoch–Schonlein purpura, childhood polyarteritis nodosa, childhood Wegener granulomatosis and childhood Takayasu arteritis . Ann Rheum Dis 2010;69:798–806. Cabral et al. Classification, Presentation, and Initial Treatment of Wegener’s Granulomatosis in Childhood. Arthritis and Rheum 2009; 60: 3413–3424. Stegmayr et al. Wegener granulomatosis in children and young adults. Ped Nephr 2000; 14. Demirkaya et al. Performing of Birmingham Vasculitis Activity Score and disease extent index in childhood vasculitides . Clin Exp Rheumatol.2012; 30(1 Suppl 70):S162-8. Akikusa et al. Clinical features and outcome of Pediatric Wegener’s Granulomatosis. Arthritis & Rheum 2007;57:837-844. Disclosure of Interest None Declared

To compare the efficacy of oral antibiotic treatment alone with treatment started parenterally and completed orally in children with a first episode of acute pyelonephritis.Multicentre, randomised controlled, open labelled, parallel group, non-inferiority trial.28 paediatric units in north east Italy.502 children aged 1 month to7 years with clinical pyelonephritis.Oral co-amoxiclav (50 mg/kg/day in three doses for 10 days) or parenteral ceftriaxone (50 mg/kg/day in a single parenteral dose) for three days, followed by oral co-amoxiclav (50 mg/kg/day in three divided doses for seven days). Main outcomes measures Primary outcome was the rate of renal scarring. Secondary measures of efficacy were time to defervescence (37 degrees C), reduction in inflammatory indices, and percentage with sterile urine after 72 hours. An exploratory subgroup analysis was conducted in the children in whom pyelonephritis was confirmed by dimercaptosuccinic acid (DMSA) scintigraphy within 10 days after study entry.Intention to treat analysis showed no significant differences between oral (n=244) and parenteral (n=258) treatment, both in the primary outcome (scarring scintigraphy at 12 months 27/197 (13.7%) v 36/203 (17.7%), difference in risk -4%, 95% confidence interval -11.1% to 3.1%) and secondary outcomes (time to defervescence 36.9 hours (SD 19.7) v 34.3 hours (SD 20), mean difference 2.6 (-0.9 to 6.0); white cell count 9.8x10(9)/l (SD 3.5) v 9.5x10(9)/l (SD 3.1), mean difference 0.3 (-0.3 to 0.9); percentage with sterile urine 185/186 v 203/204, risk difference -0.05% (-1.5% to 1.4%)). Similar results were found in the subgroup of 278 children with confirmed acute pyelonephritis on scintigraphy at study entry.Treatment with oral antibiotics is as effective as parenteral then oral treatment in the management of the first episode of clinical pyelonephritis in children.Clinical Trials NCT00161330 [ClinicalTrials.gov].