Diagnosis and Treatment of Angiography Positive Medium to Large Vessel Childhood Primary Angiitis of Central Nervous System (p-cPACNS): An International Survey

Abstract

Childhood Primary Angiitis of Central Nervous System (cPACNS) is rare, but can cause significant damage and result in disability or even death. Because of its rarity, the sometimes acute and variable presentation, limited awareness, and the absence of widely accepted diagnostic and therapeutic standards, cPACNS is a diagnostic and therapeutic challenge. Three subcategories of cPACNS exist, including angiography-positive non-progressive p-cPACNS, angiography-positive progressive p-cPACNS which both affects the medium to large vessels, and angiography-negative small vessel sv-cPACNS. Diagnosis and treatment of cPACNS relies on personal experience, expert opinion and case reports/case series. To collect information on diagnostic and therapeutic approaches to transient and progressive cPACNS, a survey was shared among international clinicians (German Society for Pediatric Rheumatology, the Pediatric Rheumatology European Society, the German speaking “Network Pediatric Stroke,” and members of the American College of Rheumatology/CARRA Pediatric Rheumatology list server). Results from this survey will be used to define statements toward a consensus process allowing harmonization of diagnostic and therapeutic approaches and the generation of evidence in a rare condition.

Diagnosis and Treatment of Small Vessel Childhood Primary Angiitis of the Central Nervous System (sv-cPACNS): An International Survey

“…Thus, some authors use more aggressive treatment regimens for sv-PACNS, including the cytotoxic agent cyclophosphamide (22). Due to the lack of widely accepted and prospectively evaluated diagnostic criteria, treatment recommendations and clinical trials, clinical practise varies significantly between centres and geographical regions (17).…”

“…A survey was designed to collect information on diagnostic and therapeutic approaches in sv-cPACNS by experts in the field and was closely related to a previously published survey focusing on p-cPACNS (17). The survey was based on real-life cases treated at the Department of Paediatrics, Faculty of Medicine Carl Gustav Carus, University of Technology, Dresden, Germany.…”

“…The objective of this survey was to collect information from international experts on diagnostic and therapeutic approaches in sv-cPACNS. This study is closely related to a previous survey on p-cPACNS (17), and collected information on standard of care approaches to diagnosis and treatment in sv-cPACNS to, in a next step, generate statements that can then be used towards an expert agreement for CTP development (18,19).…”

Arterial ischemic stroke in childhood and adolescence

“…Die Therapieentscheidung sollte interdisziplinär v. a. zusammen mit den Kolleg:innen der pädiatrischen Neurologie, Rheumatologie und Immunologie getroffen werden. Das therapeutische Vorgehen ist national und international uneinheitlich; evidenzbasierte Leitlinien fehlen [ 60 , 61 ]. Nach einer internationalen, interdisziplinären Erhebung des Status quo zu Diagnostik und Therapie der primären ZNS-Vaskulitiden im Kindesalter soll nun ein Konsensus erarbeitet werden.…”

Vertebrobasilar circulation hemorrhages in childhood primary angiitis of the central nervous system

“…The resulting clinical presentation varies in terms of; i) affected anatomical territory, which can be focal or diffuse; ii) the type of lesion, which in a majority of cases is an ischemic or a mass lesion; 2 iii) pathological progression, which can be benign or progressive, and iv) disease pattern, which can be constant or relapsing/ remitting. 3 It is diagnostically challenging because; i) It is a relatively rare disease with no ethnic, gender, or age group preponderance, 4,5 ii) There is no distinct clinical picture, and the cases present with a variety of neurological syndromes, [6][7][8] iii) There is no universal diagnostic criterion so far, 9 iv) There is no specific diagnostic test and confirmation relies on histology, v) Brain and spinal cord are relatively unsafe to biopsy, and vi) It is a poorly understood disease with diverse immune effectors and no precise immunopathological description (for example granulomatous or lymphocytic vasculitis). 6 Consequently, there are very limited therapeutic options, and it is at best only controllable with immunosuppression and antiplatelet therapy.…”