Strategies for treatment of childhood primary angiitis of the central nervous system

Beelen, Jocelyne; Benseler, Susanne M.; Dropol, Anastasia; Ghali, Brianna; Twilt, Marinka

doi:10.1212/nxi.0000000000000567

Cited by 28 publications

(27 citation statements)

References 21 publications

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“…The paucity of evidence for treatment is in part due to small patient numbers and therefore reduced interest by industry and funding bodies, and the fact that "standard-ofcare" approaches are considered effective and the introduction of "placebo controls" would be unethical. Currently, diagnostic approaches and classification of cPACNS relies on suggested, but nor prospectively evaluated criteria, and treatment is based on mostly retrospective and relatively small case series and/or expert opinion (10). In such situations, establishing consensus on diagnostic and treatment plans to harmonize approaches and prospectively collect meaningful clinical datasets in relation to consensus treatment plans can be a helpful tool to generate evidence and allow improvement of outcomes in patients with rare diseases (11,(15)(16)(17).…”

Section: Discussionmentioning

confidence: 99%

“…While patients with transient pPACNS can exhibit mild clinical symptoms indicative of focal neurological deficits, patients with progressive pPACNS usually present with symptoms of both focal and diffuse neurological deficits, including cognitive impairment, headaches and in some cases seizures ( 10 , 22 , 23 ). As 80.8% of participants agreed with the aforementioned approach to stratify patients by likely risk for disease progression and classified case 2 as transient p-cPACNS (81.4%), it appears surprising that 69.9% also stated that case 1 with posterior arteries affected may have transient disease.…”

Section: Discussionmentioning

confidence: 99%

“…Transient disease can be discriminated from progressive p-cPACNS that can affect either short or longer segments of one or more medium to large sized cerebral arteriae, including posterior vessels ( 1 ). In progressive p-cPACNS, the absence of sufficient treatment results in progressive narrowing of affected vessels on angiography after 3 or more months ( 10 ). Treatment of p-cPACNS is largely empiric and based on preliminary evidence ( 10 ), small case series and expert opinion ( 4 ).…”

Section: Introductionmentioning

confidence: 99%

“…In progressive p-cPACNS, the absence of sufficient treatment results in progressive narrowing of affected vessels on angiography after 3 or more months ( 10 ). Treatment of p-cPACNS is largely empiric and based on preliminary evidence ( 10 ), small case series and expert opinion ( 4 ).…”

Section: Introductionmentioning

confidence: 99%

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

et al. 2021

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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.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

et al. 2021

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“…No management guidelines were available for cPACNS, but a review by Beelen et al . [ 21 ] suggested a treatment strategy depending on phenotype, consisting of induction pulse corticosteroid therapy followed by maintenance for 6 months, along with early initiation of monthly cyclophosphamide and IVIg, which we believe provided a favorable overall outcome.…”

Section: Discussionmentioning

confidence: 99%

Elevated intracranial pressure requiring decompressive craniectomy in a child with progressive primary angiitis of the central nervous system: a case report

et al. 2021

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Background Elevated intracranial pressure is a potentially catastrophic complication of neurologic injury in children. Successful management of elevated intracranial pressure requires prompt recognition and therapy directed at both reducing intracranial pressure and reversing its underlying cause. A rare condition that causes elevated intracranial pressure is childhood primary angiitis of the central nervous system, which is a rare inflammatory central nervous system disease that poses diagnostic and therapeutic challenges. To our knowledge, this is the first reported case of angiography-positive progressive childhood primary angiitis of the central nervous system requiring decompressive hemicraniectomy for refractory elevated intracranial pressure in children. Case presentation We report the case of a 5-year-old Saudi girl who presented to the pediatric emergency department with fever and new-onset status epilepticus. She had elevated inflammatory markers with radiological and histopathological evidence of angiography-positive progressive childhood primary angiitis of the central nervous system, complicated by elevated intracranial pressure. Despite medical management for both childhood primary angiitis of the central nervous system and elevated intracranial pressure, her neurological status continued to deteriorate and the elevated intracranial pressure became refractory. She developed right uncal, right subfalcine, and tonsillar herniation requiring decompressive hemicraniectomy with a favorable neurological outcome. Conclusion Decompressive craniectomy might be considered in cases of angiography-positive progressive childhood primary angiitis of the central nervous system with elevated intracranial pressure refractory to medication. A multidisciplinary approach for the decision of decompressive craniectomy is advised to ensure patient safety and avoid possible morbidities and mortality.

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Mimics of Pediatric Small Vessel Primary Angiitis of the Central Nervous System

Stredny

Blessing

et al. 2022

Annals of Neurology

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Objective: Small vessel primary angiitis of the central nervous system is a rare and often severe disease characterized by central nervous system-restricted inflammatory vasculitis on histopathology. Diagnosis requires brain biopsy for confirmation and is suggested prior to starting immunotherapy when feasible. However, emerging evidence suggests that other neuroinflammatory conditions may have a clinical and radiographic phenotype that mimics small vessel primary angiitis, at times with overlapping pathologic features as well. Such diagnoses, including myelin oligodendrocyte glycoprotein antibody-associated disease and central nervous system-restricted hemophagocytic lymphohistiocytosis, can be non-invasively diagnosed with serum antibody or genetic testing that would prompt different monitoring and treatment paradigms. To determine the ultimate diagnosis of patients who were suspected prior to biopsy to have small vessel primary angiitis, we reviewed the clinical, radiographic, and pathological features of a cohort of patients at a single center undergoing brain biopsy for non-oncologic indications. Methods: Clinical data were retrospectively extracted from the medical record. Pathology and neuroimaging review was conducted. Results: We identified 21 patients over a 19-year time-period, of whom 14 (66.7%) were ultimately diagnosed with entities other than small vessel primary angiitis that would have obviated the need for brain biopsy. Diagnoses included anti-myelin oligodendrocyte glycoprotein antibody associated disease (n = 9), central nervous system-restricted hemophagocytic lymphohistiocytosis (n = 3), anti-GABA A receptor encephalitis (n = 1), and Aicardi-Goutières syndrome (n = 1). Interpretation: This study highlights the importance of pursuing now readily available non-invasive testing for mimicking diagnoses before performing a brain biopsy for suspected small vessel primary angiitis of the central nervous system.

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Strategies for treatment of childhood primary angiitis of the central nervous system

Cited by 28 publications

References 21 publications

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

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

Elevated intracranial pressure requiring decompressive craniectomy in a child with progressive primary angiitis of the central nervous system: a case report

Mimics of Pediatric Small Vessel Primary Angiitis of the Central Nervous System

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