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Patisiran and Inotersen in hATTR Amyloidosis

RNA-targeted therapies that suppress TTR production in hereditary transthyretin amyloidosis.

Written by: Saygı Hospital Health Guide Editorial Board
Published:

This content is for general information; please consult your physician for diagnosis and treatment.

This content is for informational purposes only and does not constitute medical advice. You can book an appointment at our Kardiyoloji department. Book Appointment →

What is Patisiran and Inotersen in hATTR Amyloidosis?

Patisiran is a lipid-nanoparticle-encapsulated siRNA that degrades hepatic TTR mRNA and suppresses TTR production by over 80%. The APOLLO trial showed efficacy in polyneuropathy.

Inotersen is an antisense oligonucleotide that similarly reduces TTR production. The NEURO-TTR trial demonstrated slowing of neurologic progression.

Both are primarily indicated for hATTR polyneuropathy, but are also used in patients with cardiac involvement. Vutrisiran is a newer agent in the same class.

Symptoms

Peripheral neuropathy
Autonomic dysfunction
Cardiac failure
Gastrointestinal symptoms
Features of cardiomyopathy
Arrhythmia
Extreme fatigue

Risk Factors

Positive TTR variant
Family history
Early symptom onset
Multi-organ involvement
Combined cardiac and neurologic disease
Gastrointestinal involvement
Advanced stage

When to See a Doctor?

If you experience any of the following symptoms, seek medical attention promptly:

  • Proven hATTR polyneuropathy
  • Cardiac involvement with hATTR
  • Disease progression
  • Start of therapy
  • Side effect management
  • Family genetic counseling

Treatment Methods

01
Patisiran IV every 3 weeks
02
Inotersen subcutaneous weekly
03
Vutrisiran as an alternative
04
Heart failure therapy
05
Neurologic support
06
Multidisciplinary follow-up
07
Vitamin A supplementation

Which Department to Visit?

You can visit our Kardiyoloji department for these complaints. Our specialist physicians will create the most suitable treatment plan for you.

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Let us help you

You can make an appointment with our specialists or contact us for your concerns.

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Health Disclaimer: The information on this page is prepared for general informational purposes only. It does not replace medical diagnosis and treatment. Please consult your physician for your complaints. Saygı Hospital does not accept responsibility for actions taken based on the information on this page.