title: Childhood primary large vessel CNS vasculitis: single-centre experience and review of the literature.
abstract: Ischaemic brain injuries are rare conditions in the paediatric age group. Main causes include non-arteriosclerotic arteriopathies, which in childhood usually result from primary vasculitis of large or small vessels and lead to impaired perfusion and subsequent ischaemic brain lesions. In accordance with the nomenclature of systemic forms, CNS vasculitis is subdivided into groups, based on the size of affected vessels: angiography-positive primary angiitis of medium-sized and large vessels (pPACNS), and angiography-negative angiitis of small vessels (svPACNS). We report the clinical presentation, diagnostic approach, and therapy of four children with progressive pPACNS. Patients were treated with high-dose corticosteroids and anticoagulation with unfractionated heparin in the acute phase, followed by immune modulatory treatment with mycophenolate mofetil (MMF) and dual antiplatelet therapy with acetylsalicylic acid and clopidogrel. In this manuscript, we illustrate the experience gained in our hospital, resulting in significantly faster diagnosis and treatment initiation, and discuss the applied immune modulating treatment regimen in the context of the literature. Based on our observations, we conclude that immune modulating therapy with initial high-dose corticosteroids, followed by steroid-sparing maintenance treatment with MMF, may be safe and effective in childhood progressive pPACNS.
authors.affiliation: Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Sonja
authors.initials: S
authors.lastname: Walsh
authors.affiliation: Klinik- und Poliklinik für Kinder- und Jugendmedizin, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Ralf
authors.initials: R
authors.lastname: Knöfler
authors.affiliation: Institut und Poliklinik für Radiologische Diagnostik, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Gabriele
authors.initials: G
authors.lastname: Hahn
authors.affiliation: Klinik- und Poliklinik für Kinder- und Jugendmedizin, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Judith
authors.initials: J
authors.lastname: Lohse
authors.affiliation: Klinik- und Poliklinik für Kinder- und Jugendmedizin, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Reinhard
authors.initials: R
authors.lastname: Berner
authors.affiliation: Klinik- und Poliklinik für Kinder- und Jugendmedizin, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Sebastian
authors.initials: S
authors.lastname: Brenner
authors.affiliation: Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Martin
authors.initials: M
authors.lastname: Smitka
authors.affiliation: Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
authors.forename: Maja
authors.initials: M
authors.lastname: von der Hagen
authors.affiliation: Klinik- und Poliklinik für Kinder- und Jugendmedizin, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany. christian.hedrich@uniklinikum-dresden.de.
authors.forename: Christian M
authors.initials: CM
authors.lastname: Hedrich
mesh_terms: D000305:Adrenal Cortex Hormones / Q000008:administration & dosage*; D017668:Age of Onset; D000925:Anticoagulants / Q000008:administration & dosage; D001241:Aspirin / Q000008:administration & dosage; D002545:Brain Ischemia / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000209:etiology; D002533:Cerebral Angiography / Q000379:methods; D002648:Child; D002675:Child, Preschool; D000077144:Clopidogrel; D038524:Diffusion Magnetic Resonance Imaging; D004359:Drug Therapy, Combination; D005260:Female; D005858:Germany; D006493:Heparin / Q000008:administration & dosage; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D018810:Magnetic Resonance Angiography; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage*; D010975:Platelet Aggregation Inhibitors / Q000008:administration & dosage; D013988:Ticlopidine / Q000008:administration & dosage / Q000031:analogs & derivatives; D013997:Time Factors; D016896:Treatment Outcome; D020293:Vasculitis, Central Nervous System / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy*
chemical_list: D000305:Adrenal Cortex Hormones; D000925:Anticoagulants; D007166:Immunosuppressive Agents; D010975:Platelet Aggregation Inhibitors; D006493:Heparin; D000077144:Clopidogrel; D009173:Mycophenolic Acid; D013988:Ticlopidine; D001241:Aspirin