Researchers have developed treatments that reduce amyloidosis symptoms and protect the body from damage. These treatments can help some people with amyloidosis live disease-free for many years. In some cases, amyloidosis may never come back. There are currently no treatments that can guarantee an amyloidosis cure, however.

Understanding Cure vs. Remission

Sometimes, a particular disease is said to have been “cured,” while other times a doctor will say that a disease is “in remission.” Both of these terms mean the signs and symptoms of a disease have disappeared. However, there is a big difference between cure and remission.

When a disease is cured, this means:

• The underlying causes of the disease are gone.
• The disease will not progress (get worse).
• The disease will not relapse (come back).
• You don’t need any more doctor’s visits to look for disease signs.
• You can stop using treatment.

When a disease is in remission, this means:

• The causes of the disease are still present, but under control.
• The disease may get worse over time.
• The disease may relapse, and symptoms may return.
• You need to have regular follow-up visits to make sure the disease is effectively controlled.
• You often need to continue certain treatments.

Chronic (long-term) diseases like amyloidosis often can’t be cured. However, remission may be possible. Occasionally, treatments may lead to a cure for some people with amyloidosis — meaning their body no longer makes amyloid proteins, and signs of amyloidosis disappear and don’t return. This curative effect is rare, however.

If you have amyloidosis, treatment may help you go into remission, which can sometimes last for many years. Remission could be a partial remission, in which some but not all disease signs go away. Alternatively, it could be a complete remission, in which doctors can’t find any remaining signs of disease.

Unfortunately, remission often doesn’t last forever. At some point, the amyloidosis usually comes back (relapses). When you relapse, your doctor may treat you with the same drugs that you used before, or they may recommend a different treatment plan.

Amyloidosis Remission and Prognosis

The likelihood of going into remission, and how long the remission lasts, depends on what type of amyloidosis you have and which treatments you receive. Certain characteristics called prognostic factors can tell you whether you are likely to have a better or worse outcome.

AL Amyloidosis

If treatments can effectively get AL amyloidosis — also called amyloid light-chain amyloidosis or primary amyloidosis — under control within the first six months after diagnosis, then the outlook is usually good. Many people can have close to a normal life span.

Effectiveness of AL Amyloidosis Treatments

Many medications can treat AL amyloidosis. Often, drugs used to treat multiple myeloma are used to get AL amyloidosis under control because both conditions are caused by abnormal plasma cells. Medications used to treat AL amyloidosis include:

• Alkeran (melphalan)
• Cytoxan (cyclophosphamide)
• Velcade (bortezomib)
• Darzalex (daratumumab)
• Thalomid (thalidomide)
• Revlimid (lenalidomide)

These drugs are not a cure. However, they can slow down the disease and help someone live longer. Some of these drugs can help people reach long-term remission. For example, one study of 46 people with AL amyloidosis found that 1 out of 3 people who took both melphalan and dexamethasone achieved complete remission. Another study found that thalidomide could bring about remission that lasted more than a decade for some people.

Researchers are studying whether stem cell transplantation can help treat AL amyloidosis. One study found about 1 out of 3 people who got a stem cell transplant went into complete remission. However, stem cell transplants don’t cure AL amyloidosis — the disease may temporarily improve, but it will come back.

AL Amyloidosis Prognostic Factors

Researchers have developed a scoring system that helps predict a person’s outcome after they develop AL amyloidosis. This system takes into account three different proteins: amyloid light-chain proteins, cardiac troponin T (cTnT), and N-terminal pro-brain natriuretic peptide (NT-proBNP). People with AL amyloidosis are assigned a stage based on their levels of these proteins. People with a lower stage have a better prognosis.

• Stage 1 — People live an average of 9.8 years after diagnosis.
• Stage 2 — People live an average of 6.3 years after diagnosis.
• Stage 3 — People live an average of 5.3 years after diagnosis.
• Stage 4 — People live an average of 2.3 years after diagnosis.

Other factors also play a role in a person’s outlook. Having more severe heart problems can increase your chances of having a bad outcome. Additionally, many people with AL amyloidosis have abnormal levels of certain blood cells. If these levels don’t return to normal after treatment, they may have a worse prognosis.

About 50 percent of people with AL amyloidosis have a gene change called t(11;14). This gene may increase or decrease a person’s chances of having a good outcome, depending on which treatment they receive. People with t(11;14) are more likely to go into remission after having a transplant. However, t(11;14) can lead to worse outcomes with bortezomib treatment.

If you want to learn more about your own prognosis, talk to your health care team. Your doctor can help you understand how your risk factors may affect your outcome.

AA Amyloidosis

People with AA amyloidosis can have a good outcome with the proper treatments. People who have this condition live for an average of 11 years after being diagnosed.

AA Amyloidosis Treatments and Remission

AA amyloidosis treatment often involves treating the underlying condition that causes inflammation. Many new drugs can keep inflammation under control, leading to improved outcomes for people with AA amyloidosis.

Controlling the underlying condition can help someone with AA amyloidosis go into remission. Treatments can slow down or even stop the body from making new amyloid proteins. In some cases, the body may even be able to remove old amyloid deposits, causing the disease to reverse.

Many people with AA amyloidosis have kidney disease. Treating the underlying disease may help kidney disease go into remission. However, not all damage can be reversed. Some kidney damage is permanent.

Prognostic Factors for AA Amyloidosis

A few factors can lead to worse outcomes in people with AA amyloidosis. People with very high levels of amyloid protein have a shorter life expectancy. If treatments are able to lower amyloid levels successfully, a person’s prognosis may be better.

Other factors that may indicate a worse disease outcome include:

• Having end-stage renal disease (advanced kidney disease)
• Needing dialysis to treat the kidneys
• Older age
• Having low levels of serum albumin protein in the blood
• Having high levels of C-reactive protein (CRP) or immunoglobulin G (IgG) in the blood

If AA amyloidosis is caught early, a person has a better prognosis. Additionally, new treatments are currently being developed that may help people with AA amyloidosis and could lead to better outcomes in the future.

Hereditary Amyloidosis

Many different gene mutations lead to hereditary amyloidosis. Each mutation may have a different impact on a person’s outcome. People with mutations in the transthyretin (TTR) gene can develop hereditary ATTR (hATTR) amyloidosis. On average, people with hATTR amyloidosis live for seven to 12 years after being diagnosed.

When mutations in other genes cause amyloidosis, the condition is known as non-TTR amyloidosis. The outlook for non-TTR amyloidosis depends on which gene mutation a person has. People with mutations in the fibrinogen gene often live for 15 years or more. Amyloidosis caused by mutations in the gelsolin gene may not have any impact on a person’s life span.

Effectiveness of Hereditary Amyloidosis Treatments

Researchers have recently developed several new medications to treat hATTR amyloidosis. Because these treatments are fairly new, doctors don’t yet know how they may affect a person’s long-term outlook. Studies may find that these treatments lead to a better prognosis for people with hATTR amyloidosis.

Hereditary amyloidosis is sometimes treated with a liver transplant because the liver is responsible for making amyloid proteins. A liver transplant may provide a cure for some people with hereditary amyloidosis by fixing the underlying cause of the disease. However, many people with amyloidosis are not good candidates for a liver transplant.

Prognostic Factors for Hereditary Amyloidosis

Catching hereditary amyloidosis early gives people the best chance of a good outlook. During early stages of the disease, levels of amyloid proteins are still low and haven’t caused as much damage.

There are over 100 different gene mutations that can cause hATTR amyloidosis. Some of them lead to a better outlook, while others tend to cause worse outcomes. One gene mutation is called S50R. Only 21 percent of people with this mutation are alive 10 years after being diagnosed. On the other hand, 85 percent of people with the V71A mutation are alive after 10 years.

A specific genetic mutation can also provide a clue as to which treatments may work best. People with the V30M mutation often have very good outcomes after having a liver transplant, but people without this mutation usually see a smaller benefit.

Wild-Type ATTR Amyloidosis

Wild-type ATTR amyloidosis often affects the heart. People with this condition may have a good outlook. One study found that 85 percent of people survived to their one-year follow-up.

Treatments and Wild-Type ATTR Amyloidosis Outlook

Drugs like tafamidis may help slow wild-type ATTR amyloidosis temporarily. A heart transplant can also help people with wild-type ATTR amyloidosis. In one study, 100 percent of people were alive three years after having a heart transplant. Unfortunately, many people are older when they are diagnosed with wild-type ATTR amyloidosis and are not good candidates for a heart transplant.

Wild-Type ATTR Amyloidosis Prognostic Factors

People with wild-type ATTR amyloidosis are likely to have a worse outcome if they have more severe heart problems. If you have a pacemaker, or if you have higher levels of cTnT protein in your blood, you may have a poor prognosis.

Research for a Cure

Researchers are currently running clinical trials to find better amyloidosis treatments. Some of these therapies may help produce remission or even a cure. Some examples of treatments under investigation include:

• Vibramycin (doxycycline), an antibiotic that may be able to break apart amyloidosis fibrils
• PRX-004, an antibody that can possibly lower levels of amyloid proteins
• AG10, a small molecule that may be able to prevent amyloid formation

One exciting new treatment uses CRISPR (gene-editing) technology. One of the first human clinical trials using a CRISPR drug is currently underway to study a possible treatment for hATTR amyloidosis. The drug, NTLA-2001, is designed to delete the TTR gene in the liver. Because this treatment could remove the root cause of amyloidosis, it could be a potential cure.

Talk With Others Who Understand

MyAmyloidosisTeam is the social network for people with amyloidosis and their loved ones. On MyAmyloidosisTeam, members come together to ask questions, give advice, and share their stories with others who understand life with amyloidosis.

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