Patisiran is prescribed to treat a rare and progressive polyneuropathy in patients with hereditary transthyretin-mediated (hATTR) amyloidosis which affects 50,000 people globally.
Polyneuropathy is a disease that causes damage to the peripheral nerves resulting in burning pain, numbness, and weakness that affects the same areas on both sides of the body.
Patisiran is a gene silencing drug and works by delivering small interfering RNAs (siRNA) to the liver and causing degradation of the mutant type transthyretin (TTR) protein. This leads to further reduction of this abnormal TTR protein deposits in tissues.
Patisiran should not be used in patients who are -
• Allergic to patisiran
• Children below 18 years of age (due to lack of safety and effectiveness data in these age groups)
Patisiran injections should not be given to patients with severe liver or kidney disease as patisiran has not been tested in the concerned patients.
The dose of patisiran should be calculated based on the body weight of the individual patient.
For patients weighing below 100 kg:
• The recommended dose of patisiran is 0.3 mg/kg given once every three weeks.
For patients weighing more than 100 kg:
• The recommended dose of patisiran is 30 mg once every three weeks.
Patients must be premedicated at least 1 hour before giving patisiran injection to minimize infusion-related reactions (IRRs).
The premedication involves the following medicines-
• Patisiran injection should be given by intravenous (IV) injection directly into the vein only by the healthcare personnel.
• Calculate the required dose of patisiran and withdraw the necessary contents from the vial. Filter patisiran injections by using a sterile filter and dilute with 0.9% sodium chloride to make a total volume of 200 ml into an infusion bag.
• The diluted patisiran solution should be used immediately after preparation. If it is not used immediately, store the infusion bag at room temperature (below 25°C) but use within 16 hours from the preparation time.
• The infusion should be given intravenously or deep into the vein very slowly over a period of 80 minutes under the supervision of a medical specialist.
• The time of infusion can be extended in patients who show infusion-related side effects.
• Use a separate IV set for administering patisiran infusion; the set should be mainly free from DHEP (Di (2-ethylhexyl) phthalate) to avoid serious side effects.
• Flush the IV set with 0.9% sodium chloride solution once the infusion of patisiran is complete.
• Patients must be monitored carefully while administering patisiran infusion for symptoms and signs of infusion-related side effects such as a headache, stomach pain, breathing difficulty, and reddening of the skin.
• The infusion can be stopped temporarily if the side effects occur and can be started again very slowly after the side effects are resolved.
• Permanently discontinue the infusion in case of life-threatening side effects and treat the patient appropriately.
• Vitamin A supplementation should be given to patients under treatment with patisiran as it may decrease vitamin A levels in the body.
• Patients should get an eye checkup with an eye specialist if any symptoms of vitamin A deficiency such as night blindness are reported.
• Caution should be taken while giving the patisiran infusion to pregnant women as patisiran may reduce vitamin A levels. However, loading patients with high levels of vitamin A supplementation to combat the loss can also affect the fetus. Hence maintaining an optimum level is necessary.
• Infusion-related reactions: Skin redness, pain, and swelling at the injection site, stomach pain, headache, fluctuations in blood pressure, nausea, swelling of the face and lips
• Respiratory: Pharyngitis, sinusitis, rhinitis, nasal congestion, bronchitis, lung infections, breathing difficulty
• Musculoskeletal: Muscle pain, back pain, pain in the joints
• Others: Indigestion, vertigo, vision disturbances, dry eye
• Inspect the patisiran vial for the presence of any visible particles or discoloration before diluting and administering to the patient. Discard the vial or the diluted mixture if such conditions exist.
• The vial should be removed from the freezer and allowed to cool down to room temperature before diluting it into an infusion bag.
• Do not shake but gently invert the infusion bag to facilitate mixing.
• No evidence of drug interactions was reported.
• However, the doctor should be kept informed if the patient is consuming or planning to take any prescription, Over-The-Counter medications, or herbal supplements to avoid any serious drug-related side effects.
• Store patisiran vials in a refrigerator at 2°C to 8°C. Do not freeze or shake the vial.
• The patisiran vials can also be stored at room temperature below 25°C for a maximum of 2 weeks if refrigeration or cold storage is not available.
• Store the infusion bag containing diluted mixture at room temperature (below 25°C) but use within 16 hours from the preparation time.
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