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

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