Ab0998 severe Iga Vasculitis: Cyclophosphamide in Question

Abstract

BackgroundCyclophosphamide (CYC) has been a mainstay in the treatment of severe childhood primary systemic vasculitis. Although major organ involvement is rare in IgA vasculitis (IgAV), CYC is among therapeutic options in moderate and severe IgAV nephritis.(1) No specific recommendations are available for cerebral vasculitis of IgAV.ObjectivesTo describe disease course and treatment response in 2 patients with severe IgAV with CNS and renal involvement.MethodsCase reports with disease assessments using Paediatric Vasculitis Activity Score (PVAS).(2)ResultsBoth children were Caucasian boys with unremarkable previous history aged 4 (Patient 1) and 8 (Patient 2) years. In both cases the initial typical presentation included purpura and abdominal pain in both, arthritis in Patient 1 and haematuria in Patient 2. After 2 weeks of prednisone therapy for severe gastrointestinal (GIT) symptoms Patient 1 was admitted for right-sided weakness and facial palsy. His brain MRI revealed an ischaemic lesion. His PVAS reached 21/63 for skin, abdominal, renal (haematuria) and CNS systems. When Patient 2 was admitted for abdominal symptoms he developed a focal epileptic paroxysm. His brain MRI was compatible with cerebral vasculitis. His nephritic-nephrotic syndrome prompted renal biopsy showing mesangioproliferative nephritis with IgA deposits. His PVAS was 27/63 for skin, abdominal, renal (hypertension, haematuria, proteinuria) and CNS systems. Both patients received 3 doses of intravenous methylprednisolone 30 mg/kg followed by oral prednisone 1 mg/kg with subsequent tapering and 3 doses of CYC 500mg/m2(monthly i.v.). At follow-up 3 months after the 1st CYC dose both patients fully recovered neurologically. Patient 2 was on prednisone (0.2 mg/kg) and ACE inhibitor and his urine was negative for the first time, PVAS=0. Patient 1 had been developing significant proteinuria during prednisone withdrawal and his PVAS counted 10/63 for renal disease. Prednisone was re-instituted and ACE inhibitor added while renal biopsy has been pending.ConclusionIn both cases therapy with corticosteroids and CYC led to the full recovery of CNS disease but did not prevent progression of nephritis in Patient 1. Response of Patient 2 nephritis to CYC appeared satisfactory at 3 months visit but would require confirmation after full prednisone withdrawal. Although generally benign, when IgAV presents with major organ involvement its treatment is challenging. Further research is needed in order to gather better evidence to support treatment recommendations.References[1] özen S, et al. Europen consensus-based recommendations for diagnosis and treatment of IgA vasculitis – the SHARE initiative. Rheumatology (Oxford) 2019, in print[2] Doležalová P, et al. Disease activity assessment in childhood vasculitis: development and preliminary validation of the Paediatric Vasculitis Activity Score (PVAS). Ann Rheum Dis 2013 72(10):1628-33Disclosure of InterestsNone declared