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Table 5 Interventions and Treatments for CLN2 disease, statements and consensus data

From: Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients

Statement Responders Evidence level Consensus
Initiation of long-term ERT with cerliponase alfa at 300 mg (or age-appropriate) dose every other week through intraventricular infusion is suggested in non-classical TPP1 deficiency patients after confirmed diagnosis and agreement between parents and provider, as long as no contraindications to therapy exist. Initiation of long-term ERT with cerliponase alfa at 300 mg (or age-appropriate) dose every other week through intraventricular infusion is recommended in classical CLN2 patients with the potential to benefit from this therapy 37 C 84
Disease-modifying treatment with a licensed therapy ideally should be delivered by a team experienced in the management of CLN2 disease and use of any required devices. For current ERT treatment for CLN2 disease, this includes brain intraventricular devices 39 C 93
There is no evidence currently that HSCT benefits patients with CLN2 and at this time is not recommended or approved as a treatment 34 C 93
Intraventricular devices should be placed under general anaesthesia by a very experienced paediatric neurosurgeon 36 C 92
Intraventricular device should only be accessed by a trained individual to limit/ minimise complications 39 C 95