Name of the Procedure:

Injection, Patisiran, 0.1 mg (J0222)

Summary

Patisiran is a medication administered via injection to treat a specific type of genetic disorder known as hereditary transthyretin-mediated (hATTR) amyloidosis. This treatment helps to reduce the buildup of abnormal protein deposits in the body.

Purpose

Patisiran is used to slow the progression of hATTR amyloidosis, which can cause nerve damage and affect various organs. The goal is to reduce symptoms, improve quality of life, and prevent further complications of the disorder.

Indications

• Patients diagnosed with hATTR amyloidosis.
• Symptoms such as peripheral neuropathy (numbness, tingling, pain in the hands and feet) and autonomic neuropathy (digestive issues, heart problems).

Preparation

• Patients may need to undergo blood tests and a complete medical evaluation before starting treatment.
• Instructions on medication adjustments (e.g., stopping specific drugs beforehand) may be given.
• Hydration is often recommended before the procedure.

Procedure Description

1. The patient arrives at the healthcare facility.
2. A healthcare professional prepares the patisiran injection.
3. The injection is administered intravenously over approximately 80 minutes.

4. Vital signs are monitored throughout the procedure.

   Tools and Equipment:

   • IV administration set
   • Patisiran solution in appropriate dosage

   Anesthesia or Sedation:

   • Not typically required for this procedure.

Duration

The injection itself typically takes about 80 minutes, but the entire visit, including preparation and observation, may last around 2 to 4 hours.

Setting

The procedure is generally performed in a hospital, outpatient clinic, or infusion center.

Personnel

• Trained nurses or medical assistants administer the injection.
• A physician or specialist oversees the treatment process.
• A pharmacist may prepare the medication.

Risks and Complications

Common risks:

• Infusion-related reactions (itching, flushing, fever)

Rare risks:

• Severe allergic reactions (anaphylaxis)
• Injection site infections

Complications management includes the administration of antihistamines or corticosteroids and monitoring for severe reactions.

Benefits

• Slowed disease progression.
• Reduction in symptoms like pain and numbness.
• Improved quality of life can be noticed within months of regular treatment.

Recovery

• Patients are monitored for a short period post-injection for any adverse reactions.
• Normal activities can typically be resumed immediately.
• Follow-up appointments every three weeks for additional injections.

Alternatives

• Other pharmaceutical treatments for hATTR amyloidosis (e.g., inotersen).
• Liver transplant in severe cases. Pros and cons:
• Alternative medications may have different efficacy and side effect profiles.
• Liver transplant is invasive and comes with significant risks and a long recovery period.

Patient Experience

During the procedure, the patient may feel a slight pinch at the IV insertion site and should expect to remain relatively still. Post-procedure, some may experience mild reactions but can usually continue their day with minimal disruption. Pain management and comfort measures include pre- and post-medication to minimize infusion-related reactions.