AL (Primary) amyloidosis

AL (primary) amyloidosis is the most common form of the disease. It is a blood disorder that results from an unregulated production of lambda and kappa light chains from an abnormal clone of plasma cells. These circulating light chains form amyloid fibrils which are toxic to tissues and deposit in various organs to produce the clinical manifestations of the disease. .

Although AL amyloidosis can occur at any age, it predominantly affects individuals in their 5th to 7th decades of life and is slightly more common in males. It is a multisystemic disease that could affect the nervous systems, liver, gastrointestinal tract, soft tissues and blood vessels, but the kidney is the most common organ affected followed by the heart.

Life expectancy depends on the predominant organ affected and the degree of organ involvement. Cardiac involvement is associated with poor prognosis. In untreated cases, the median survival from the time of diagnosis in cardiac amyloidosis is 6 months, making early diagnosis critical for good outcomes in cardiac disease.

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AA (Secondary) Amyloidosis

AA (secondary) amyloidosis is associated through a protein called "serum amyloid A." This protein is produced by the body as a response to inflammation or infection.

While high levels of the protein do not cause amyloid deposits over the short term, it can lead to amyloid deposits over a long period of time. As a result, diseases which lead to chronic states of inflammation like uncontrolled rheumatoid arthritis or to chronic infection like chronic tuberculosis) can result in AA amyloidosis deposits over several years.

AA (secondary) amyloidosis commonly affects the kidneys, liver or spleen.:

The heart’s involvement in AA (secondary) amyloidosis is extremely rare.

Treatment targets the cause of the inflammation or infection – for example, with immunosuppressants, or appropriate antibiotics.

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Familial ATTR Amyloidosis

In Familial ATTR amyloidosis the body makes a mutant form of a protein called "transthyretin." Transthyretin is abbreviated "TTR" and is the reason this disease is called familial TTR amyloidosis. While everyone makes transthyretin, the mutated form is more likely to develop into amyloid fibrils which are toxic to tissues. While many different mutations have been identified, one of the most common is called "V-122-I" and is particularly common in individuals of African descent.

This disease usually affects individuals at 40 years of age and above with slightly greater predominance in males than females. The median survival after diagnosis in untreated cases is 25 months. Familial TTR amyloidosis usually affects the heart and nerves. The predominant system affected varies according to the type of mutation such that some patients may present with predominantly cardiac or neurological manifestations.

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Wild-Type (Senile) ATTR Amyloidosis

Wild-type (senile) TTR amyloidosis is similar to familial TTR amyloidosis, except the protein that is deposited is the normal transthyretin protein. The normal transthyretin protein is less likely to form amyloid deposits than the mutated form seen in familial TTR.

As a result, patients only develop the disease as they get older, usually at 65 years of age or above. Wild type TTR amyloidosis commonly affects males more than females and a prior history of carpal tunnel syndrome is reported 5-10 years before cardiac manifestations. Since amyloid deposits accumulate slowly in this form of the disease, the prognosis is generally better than AL (primary) amyloidosis and familial TTR amyloidosis with a median survival of 43 months from the time of diagnosis in untreated cases.

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Type of Medication

Name of Medication

How this Medication Works?

Approved for Use

How this medication is given?

ATTR Silencers

Patisiran (Onpattro®)


These medications work in the liver to reduce the creation of TTR.


FDA approved for patients with hereditary ATTR who also have neuropathy.

Infusion given every 3 weeks

Inotersen (Tegsedi®)

Injection given once a week and requires weekly lab work.


ATTR stabilizers

Tafamidis (Vyndamax®, Vyndaqel®)

These medications make the TTR protein more stable, preventing it from breaking apart and forming the abnormal amyloid fibrils

FDA approved for patients with hereditary or wild-type ATTR that has affected their heart.

Vyndamax/Vyndaqel: Capsule that is taken once daily.



Commonly used off-label. This is a nonsteroidal anti-inflammatory drug (NSAID) that has been shown to stabilize the TTR protein.


Tablet that is usually taken twice a day.



Currently being tested in clinical trials.



Fibril Disruptors

Doxycycline (antibiotic)/ TUDCA*

These medications may help break up and clear ATTR amyloid fibrils.

Commonly used off-label.

Only tested in small studies and there is limited evidence that these medications would be helpful.



Green Tea Extract




Over the Counter Supplement