J Neurosurg Pediatr. 2025 Oct 31:1-8. doi: 10.3171/2025.7.PEDS25222. Online ahead of print.
ABSTRACT
OBJECTIVE: Cerliponase alfa is an enzyme replacement treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), administered via biweekly intracerebroventricular infusions. Typically, infusions are delivered via a head-sited access device; however, subcutaneously tunneled chest-sited devices could offer an alternative for improved ease of access and patient comfort. The aim of this study was to evaluate the safety, efficacy, and user experience of chest-sited versus head-sited intracerebroventricular access devices in patients with CLN2.
METHODS: This single-center prospective observational study included pediatric patients with CLN2 who received cerliponase alfa via intraventricular infusions using chest-sited and/or head-sited devices. Incidents and complications, as well as access frequency, were documented over 1 year for both device types. Families and nurses were surveyed regarding sedation, distraction, access, securing, satisfaction, and device preference, and comparisons were made between chest-sited and head-sited devices regarding these factors.
RESULTS: Seventeen children (10 female, mean age 7.5 [SD 3.1] years) with CLN2 were included; 11 received chest-sited and 6 received head-sited devices. Families of 16 patients (10 with chest-sited and 6 with head-sited devices) completed the questionnaire. Sedation was required for 4 patients, 1 with a chest-sited device and 3 with head-sited devices. Distraction techniques, particularly singing and technology, were effective for 75% of patients who completed the family questionnaire. There was no significant difference in ease of access or securing between the two device types (p > 0.9999) according to the families. However, nurses reported significantly easier access (p = 0.0308) and securing (p < 0.0001) with chest-sited devices. Among patients who received both device types, 50% preferred chest-sited devices, 50% had no preference, and no patients preferred head-sited devices. Access success rates were higher for chest-sited devices (p = 0.0128), but device survival was comparable (p = 0.4492). The incident and complication rate was similar between groups, with no device-associated infections recorded.
CONCLUSIONS: Chest-sited intracerebroventricular access devices for cerliponase alfa infusions offered benefits that included reduced sedation, easier access, better device securing, higher access success rates, and greater nursing satisfaction. Both devices had similar survival rates and incident and complication rates. Chest-sited devices can be considered a safe and effective alternative in this setting.
PMID:41172362 | DOI:10.3171/2025.7.PEDS25222
