Treatment of symptomatic children ≥3 years of age with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) (or tripeptidyl peptidase 1 [TPP1] deficiency).
Proenzyme is activated in the lysosomes and then cleaves tripeptides from a broad range of proteins. This leads to the breakdown of lysosomal storage materials that otherwise accumulate in patients with late infantile neuronal CLN2 and lead to progressive decline in motor function.
Slowed decline in ambulation.
Distribution: Distributed into CSF following intraventricular administration.
Metabolism and Excretion: Degraded through peptide hydrolysis.
Half-life: 6.2–7.7 hr.
TIME/ACTION PROFILE (CSF levels)
- Acute infection around device insertion site or CNS infection
- Acute intraventricular access device-related complication
- Ventriculoperitoneal shunts.
Use Cautiously in:
- Bradycardia, conduction disorder, or structural heart disease (↑ risk of conduction disorder or heart disease)
- Children <3 yr (safety and effectiveness not established).
Adverse Reactions/Side Effects
CNS: SEIZURES, MENINGITIS, headache, irritability, pleocytosis, jitteriness
CV: ECG abnormalities, bradycardia, hypotension
Misc: HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS), fever, device-related complication (including device leakage, bulging of scalp around device, device failure), device-related infection
* CAPITALS indicate life-threatening.
Underline indicate most frequent.
Intraventricular: Children 300 mg every other wk. Follow each infusion with infusion of Intraventricular Electrolytes.
Solution for intraventricular infusion: 30 mg/mL (with 5–mL vial of Intraventricular Electrolyte solution for intraventricular infusion)
- Obtain a sample of CSF for cell count and culture prior to each infusion and as clinically indicated.
- Monitor vital signs (blood pressure, heart rate) before infusion starts, periodically during infusion, and post-infusion in a health care setting. Upon completion of infusion, clinically assess patient. Continue observation if clinically indicated.
- Monitor ECG during infusion in patients with history of bradycardia, conduction disorder, or with structural heart disease. Monitor regular 12-lead ECG every 6 months in patients without cardiac abnormalities.
- Assess scalp for signs of intraventricular access device leakage or failure (extravasation of CSF fluid, bulging of the scalp around or above intraventricular access device), or potential infection (erythema, tenderness, or discharge, suspected or confirmed CNS infection) before each infusion. If signs occur, discontinue infusion.
- Monitor for signs and symptoms of hypersensitivity reactions (pyrexia, vomiting, pleocytosis, irritability) during and after infusion. Treatment depends on reaction severity; may include temporarily interrupting infusion, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If a severe reaction occurs, immediately discontinue infusion and treat as needed.
- Activity intolerance
- Pre-treat patients with antihistamines with or without antipyretics or corticosteroids 30 to 60 minutes prior to the start of infusion.
- Cerliponase alfa is infused into the cerebrospinal fluid CSF via a surgically implanted reservoir and catheter. First infusion is usually 5–7 days after device implantation. Follow aseptic technique during preparation and administration.
- Cerliponase alfa is administered by, or under direction of, a health care professional experienced in intraventricular administration.
- Thaw cerliponase alfa and Intraventricular Electrolytes vials at room temperature for about 60 min. Do not thaw or warm any other way; do not shake vials. Condensation will occur during thawing period. Do not re-freeze vials or freeze syringes containing cerliponase alfa or Intraventricular Electrolytes. Solution is clear to slightly opalescent and colorless to pale yellow; do not infuse solutions that are discolored, cloudy, or contain particulate matter. May occasionally contain thin translucent fibers or opaque particles removed by 0.2 micron filter. Infuse with the B Braun Perfusor® Space Infusion Pump System. Solution can be stored in syringes in refrigerator for up to 4 hr before infusion and vials can be stored in refrigerator for up to 24 hrs. Use compatible 20 mL syringes provided in the Administration Kit.
- Rate:Infuse at rate of 2.5 mL/hr with delivery accuracy of +/- 1 mL through a 0.2 micron in-line filter. Set occlusion alarm setting to ≤281 mm Hg. Following cerliponase alfa infusion, administer Intraventricular Electrolytes using the provided Administration Kit. Solution is clear to colorless; may contain particles, which appear during thaw period and dissolve when solution reaches room temperature. Both infusions require a total of 4.5 hrs. Intraventricular Electrolytes are used to flush infusion line, port needle, and intraventricular access device, to fully administer medication and maintain patency of intraventricular access device.
- Explain purpose of cerliponase alfa infusion to patient and parents.
- Instruct patient or parent to notify health care professional immediately if sign and symptoms of infection, hypotension, bradycardia, and hypersensitivity reactions occur.
- Advise female patient to notify health care professional of pregnancy is planned or suspected or if breastfeeding.
Slowed decline in ambulation.
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