Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease

TL;DR

Families of children with CLN2 disease reported better long-term treatment experiences with chest-sited ports compared to scalp-based devices for intracerebroventricular infusions.

Contribution

This study provides caregiver insights into the transition from scalp-based to chest-sited ports for CLN2 disease treatment, emphasizing patient-centered care.

Findings

Chest-sited ports were perceived as more durable and easier to integrate into long-term treatment routines.

Transitioning from scalp VADs to chest ports was described as well-coordinated and satisfying for caregivers.

Caregiver perspectives highlight the importance of device choice in shaping treatment burden and safety planning.

Abstract

What are the main findings?
Caregiver experience differed meaningfully between scalp-based ventricular access devices and chest-sited ports for intracerebroventricular cerliponase alfa delivery for children with CLN2 disease.Chest-sited ports were perceived favorably as access options that supported long-term treatment adherence.

Caregiver experience differed meaningfully between scalp-based ventricular access devices and chest-sited ports for intracerebroventricular cerliponase alfa delivery for children with CLN2 disease.

Chest-sited ports were perceived favorably as access options that supported long-term treatment adherence.

What are the implications of the main findings?
Device and external port selection for intraventricular therapy in CLN2 disease influences caregiver experience, perceived safety, and treatment routines.Incorporating caregiver perspectives may improve shared decision-making and patient-centered planning for long-term intracerebroventricular access for patients with CLN2 disease.

Device and external port selection for intraventricular therapy in CLN2 disease influences caregiver experience, perceived safety, and treatment routines.

Incorporating caregiver perspectives may improve shared decision-making and patient-centered planning for long-term intracerebroventricular access for patients with CLN2 disease.

Background: Cerliponase alfa is currently the only approved disease-modifying therapy for neuronal ceroid lipofuscinosis type 2 (CLN2) disease and requires lifelong intracerebroventricular (ICV) infusion, traditionally via a scalp-sited ventricular access device (VAD). Chest-sited port (chest port) for intracerebroventricular access using a tunneled central venous access device is described as an alternative, though data remain limited. Methods: We present an anonymized caregiver narrative perspective describing two pediatric patients with CLN2 disease receiving cerliponase alfa infusions via different ICV access strategies: one patient who transitioned from a scalp-based ventricular access device to a chest port and one patient who initiated therapy with a chest port. A semi-structured caregiver interview was used to capture experiential insights related to decision-making, procedural burden, safety considerations, and psychosocial adaptation. Results: The caregiver identified key advantages of chest ports for ICV infusion, including durability of the device, enhanced securement, and smooth long-term routine integration. The transition from a scalp VAD to a chest port was described as proactive, well-coordinated, and associated with high caregiver satisfaction. Noted considerations included increased visibility of the access needle to the child, proximity to oral secretions, and potential misidentification of the port by emergency medical services. Families implemented mitigation strategies through labeling, education, and coordination with the care team. Conclusions: This caregiver-centered case report highlights how access device choice meaningfully shapes treatment burden, safety planning, and daily life for families managing CLN2 disease. As chest-port methodologies become adopted, incorporating caregiver and patient perspectives is essential to developing patient-centered treatment options for long-term intracerebroventricular therapy.

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