Cerliponase alfa is a recombinant human enzyme, used to treat late infantile neuronal ceroid lipofuscinosis type 2 (CLN2).
Cerliponase alfa is recombinant human tripeptidyl peptidase-1 enzyme (TPP1) which is required for normal brain development.
It is used to slow down the loss of walking ability in symptomatic pediatric patients from the age group of 3 years and above who suffer from late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease.
These patients lack the tripeptidyl peptidase-1 enzyme (TPP1) in their central nervous system (CNS).
Cerliponase alfa is infused directly into the patient’s brain bypassing the blood brain barrier through a device called an intraventricular device.
Cerliponase alfa is contraindicated on the following conditions:
a) Allergy to the drug
b) The presence of intraventricular access device related complications which include:
• Failure of the device
• Signs of device-related infection such as swelling, scalp redness, extravasation of fluid, bulging of scalp around or above intraventricular access device.
c) Patients with ventriculoperitoneal shunts (catheter that connects the brain to the abdomen to reduce the fluid content in the brain) due to the risk of the following:
• Bleeding or blood clot in brain
• Cerebrospinal fluid leakage under the skin in the shunt area
• Brain swelling and damage
• Infection occurring in the shunt, brain or abdomen
• The recommended dosage of cerliponase alfa for patients 3 years of age and older is 300mg administered using intraventricular infusion every other week.
• The drug infusion should be followed by intraventricular electrolytes at a rate of 2.5ml per hour. The total time of infusion for both should be approximately 4 and a half hours.
• Cerliponase alfa comes in a concentration of 150mg/5ml in two single dose vials along with intraventricular electrolyte injection of 5ml in a single dose vial.
• It is administered into the cerebral ventricle (central cavity of the brain) through a surgically implanted reservoir connected to the catheter of an intraventricular access device.
• The whole process should be done under aseptic technique and the first dose of infusion should be carried out at least 5 to 7 days after implantation of the device.
• Since allergic reactions can take place, the patients should be pretreated with antihistamines with or without antipyretics or corticosteroids 30-60 minutes before starting the infusion.
• The vital signs should be monitored before, during and after the infusion administration.
• Routine CSF samples should be tested regularly to detect the presence of device related infections.
• General: Allergic reaction, fever, vomiting, hematoma (blood clot), infection
• Central Nervous System: Headache, irritability, feeling jittery, decrease or increase of cerebrospinal fluid protein, increased WBC count in the cerebrospinal fluid, seizures
• Cardio vascular system: ECG abnormalities including bradycardia (reduced heart rate), low blood pressure
The intraventricular access device should considered for replacement after around 105 perforations or roughly after 4.3 years of regular use.
• Cerliponase alfa injection and the intraventricular electrolytes should be stored upright in a freezer at a temperature of -25 °C to -15 °C. It should be protected from light.
• Administration sets should be kept in the original carton and should not to be frozen.
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