A drip treatment for hereditary ATTR amyloidosis nerve disease
Patisiran
A drip (infusion) treatment used for the nerve disease caused by hereditary transthyretin amyloidosis.
What is Patisiran?
Patisiran is a specialist medicine used to treat the nerve disease (polyneuropathy) caused by hereditary transthyretin (hATTR) amyloidosis, a rare inherited condition in which an abnormal protein builds up and damages the nerves. It is a type of RNA interference (siRNA) therapy that lowers production of this protein. It is given as a drip into a vein on a regular schedule, with medicines given beforehand to reduce the chance of an infusion reaction. People taking it are usually given a vitamin A supplement, as the medicine lowers vitamin A levels.
Education and reference only. This is a plain-language guide to Patisiran — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Patisiran is a medicine used to treat the nerve disease, called polyneuropathy, that develops in people with hereditary transthyretin (hATTR) amyloidosis. In this inherited condition, a protein called transthyretin is made in an abnormal form that clumps together and builds up in tissues, including the nerves, causing damage. Patisiran is a type of RNA interference (siRNA) therapy that reduces how much transthyretin the body makes. It is given as a drip (infusion) into a vein on a regular schedule by the specialist team.
How it works
In hereditary transthyretin amyloidosis, the liver makes an abnormal form of the protein transthyretin, which clumps together and is deposited in tissues such as the nerves, causing the nerve disease. Patisiran works by quietening down the instruction that tells the liver to make transthyretin, so much less of the protein is produced, both the abnormal and normal forms. With less protein being made, there is less to clump and build up, which helps slow the nerve damage. It is given by drip on a regular schedule to keep this effect going. Because transthyretin also helps carry vitamin A in the blood, lowering it can reduce vitamin A levels, which is why a supplement is given.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist drip treatment used in the UK for nerve disease caused by hereditary transthyretin (hATTR) amyloidosis.
Practical use
How to take Patisiran
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Have the drip into a vein on the regular schedule your specialist team arranges.
- Expect to be given other medicines beforehand to reduce the chance of an infusion reaction.
- Take the vitamin A supplement you are given, as the medicine lowers vitamin A levels.
- Tell your team about any eye or vision problems, as these can relate to vitamin A.
- Report any reaction during or after the infusion, such as flushing, breathlessness or a rash.
Weighing it up
Advantages & disadvantages of Patisiran
Advantages
- Slows the nerve disease caused by hereditary transthyretin amyloidosis.
- Reduces production of the abnormal protein that builds up and damages nerves.
- Given on a regular schedule under specialist care, with steps taken to reduce infusion reactions.
Disadvantages
- Given as a drip into a vein, which takes time and specialist supervision.
- Can cause infusion reactions, which is why premedication is given.
- Lowers vitamin A levels, so a supplement is needed.
Practical use
Good to know
Two practical points stand out with patisiran. First, it is given as a drip into a vein, and to reduce the chance of an infusion reaction, other medicines are usually given beforehand; the team watches you during and after the infusion. Second, because the medicine lowers transthyretin, which normally helps carry vitamin A in the blood, vitamin A levels fall, so people are usually given a vitamin A supplement and advised to report any eye or vision problems. It treats the nerve disease by slowing it rather than curing the condition, so it is a long-term treatment given on a regular schedule. The specialist team assesses how well it is working by checking nerve symptoms and overall function over time, and keeps the infusion schedule going as planned.
Who should not take it / use with caution
- People who have had a serious allergic reaction to patisiran should not use it.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be used under a specialist amyloidosis service that can manage infusions.
Monitoring
- Watching for infusion reactions during and after each drip.
- Checking nerve symptoms and overall function over time to judge how well it is working.
- Keeping an eye on vitamin A status and any eye or vision symptoms.
Side effects
- Infusion reactions such as flushing, back pain, nausea or breathlessness, which premedication helps prevent.
- Low vitamin A levels, which is why a supplement is given.
- Tummy upset or swelling of the limbs in some people.
Key interactions
- There are few well-established routine medicine interactions, but tell your team about all your medicines.
- It is given alongside a vitamin A supplement rather than relying on diet alone.
- Always tell your team about supplements and over-the-counter products you take.
Available as: A solution given as a drip (infusion) into a vein.
Answers
Patisiran: frequently asked questions
What is patisiran used for?
It is used to treat the nerve disease caused by hereditary transthyretin (hATTR) amyloidosis, by reducing production of the abnormal protein that builds up and damages nerves.
Why am I given other medicines before the drip?
Medicines are given beforehand to reduce the chance of an infusion reaction, and the team watches you during and after the infusion.
Why do I need vitamin A?
The medicine lowers transthyretin, which helps carry vitamin A in the blood, so vitamin A levels fall and a supplement is given; report any vision problems.
How is it given?
It is given as a drip (infusion) into a vein on a regular schedule arranged by your specialist amyloidosis service.
Does it cure the condition?
No. It slows the nerve disease rather than curing the condition, so it is a long-term treatment.
Authoritative sources
- BNF
- NICE CKS
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