ArticleChild's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery2025
Novel surgical approach for intraventricular cerliponase alfa enzyme replacement therapy via central venous access device (CVAD) port in neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
Article in Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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2 citing papers in PubMed.
- Conversion of a ventricular rickham reservoir to a subcutaneous chest port for repeated intraventricular enzyme replacement therapy: technical note and surgical considerations.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease.Children (Basel, Switzerland) · 2026Article
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9 authors.
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Abstract
introductionNeuronal ceroid lipofuscinosis type 2 (CLN2) is a genetic disease caused by deficiency of the enzyme tripeptidyl peptidase 1 (TPP1), resulting in seizures, vision/cognitive decline, and early death. Cerliponase alfa is an enzyme replacement therapy approved as treatment for CLN2 disease, administered by intracerebroventricular infusion via a surgically implanted device. Maintaining sterility for prolonged scalp-based infusions can be difficult in children and damage devices. We report the development of a technique connecting the intraventricular device and catheter to a chest port to mitigate some of these challenges.
methodsWe describe a seven-patient cohort who underwent implantation of our modified infusion system connecting a ventricular catheter distally to a central venous access device (CVAD) port implanted in the anterior chest.
resultsBetween March 2019 and April 2024, seven (n = 7) patients aged 2-19 years underwent the placement of this system or had an old device converted to a CVAD port system. None of our patients has experienced significant access issues, infection, or required revision since implantation.
conclusionsThe use of an intraventricular access device connected to a CVAD allows for safe and efficacious long-term infusion of cerliponase alfa therapy and provides a more stable and well-tolerated alternative to scalp-based infusions.
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