Table of ContentsView AllTable of ContentsDrug TreatmentSurgeries and Organ TransplantOTC MedicationMental HealthTreatment BreakthroughsSpecialists and HospitalsNext in hATTR Amyloidosis GuideLearning to Live With hATTR Amyloidosis

Table of ContentsView All

View All

Table of Contents

Drug Treatment

Surgeries and Organ Transplant

OTC Medication

Mental Health

Treatment Breakthroughs

Specialists and Hospitals

Next in hATTR Amyloidosis Guide

Hereditary transthyretinamyloidosis, or hATTR, is a rare disease that causes proteins to build up in the body, forming amyloid deposits. This causes serious, wide-rangingsymptoms, including heart disease, kidney failure, and nerve damage.

Amyloidosis treatment involves addressing the symptoms, including those ofATTR cardiac amyloidosis, while also stopping the protein production that makes symptoms worse. Promising treatments have been approved and more are undergoing clinical trials, including ATTR-CM treatments.

Continue reading to learn more about hATTR amyloidosis treatments that can slow the progression of the disease and improve your quality of life.

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Person receiving prescription from pharmacist

hATTR Amyloidosis Drug Treatment

Drugs used to treat amyloidosis work in the following two ways:

Here’s an overview of the type of amyloidosis medications in each category.

TTR Stabilizers

TTR stabilizers are drugs that stop the TTR protein from creating amyloid deposits. These deposits are what cause many of the symptoms of hATTR. The two types of TTR stabilizers are:

Another type of TTR stabilizer, acoramidis, is currently being studied in clinical trials.

Diagnosis and Genetic Testing for hATTR Amyloidosis

TTR Silencers

Supportive Therapies

In addition to treating hATTR, your healthcare provider may recommend medications to address its symptoms, including neuropathy and heart disease. These are called supportive therapies, because they support you in living a healthier, more comfortable lifestyle.

The supportive therapies for hATTR often include:

Always tell your healthcare provider about any other medications or supplements you’re taking before starting a new medication.

Learning to Live With hATTR Amyloidosis

hATTR Amyloidosis Surgeries and Organ Transplant

Before 2018, the only treatment available for hATTR was aliver transplant.With the development of new medications, people with hATTR have been able to control their symptoms without a liver transplant.

However, a liver transplant is still an option for patients who don’t respond to treatments. Because the TTR protein is produced in the liver, a transplant may help stop production. Yet, some patients continue to have irregular TTR production even with a new liver.

Cardiac disease is one of the main symptoms of hATTR, some patients with the disease will need aheart transplant. Heart disease sometimes continues after surgery for patients with hATTR, so it’s very important to talk with your healthcare provider about the benefits and risks when considering this major surgery.

Over-the-Counter Medications to Manage Symptoms

Because hATTR causes many symptoms, some symptoms experienced with the condition hATTR may not seem related. It’s tempting to reach for over-the-counter (OTC) treatments like laxatives or diarrhea medicine without knowing the root cause.

Before you do that, talk with your healthcare provider. They can help you understand whether OTC medications could interact with prescription medication, and how they might work for you given the unique circumstances that hATTR presents.

Your healthcare provider can also suggest lifestyle changes, such as provider-approved exercise, that might help manage symptoms. Working with a physical therapist or dietitian familiar with hATTR and your symptoms might also help.

Complementary and Alternative Medicine (CAM) Therapies

Many people with chronic diseases find relief through CAM treatments, like acupuncture, massage, and holistic healing.Integrated medicinecan be impactful, especially in addition to traditional medical treatment.

Be sure that any alternative providers you see understand hATTR, and talk with your medical healthcare provider about which alternative treatments might be helpful and which you should avoid.

Mental Health and hATTR Amyloidosis

Living with a life-threatening condition like hATTR can be very stressful, and can take a toll on your mental health. Consider seeing a counselor, or joining a support group for people with amyloidosis.Self-carecan play an important role too.

hATTR Amyloidosis Treatment Breakthroughs

Since 2019, there have significant breakthroughs in the treatment of hATTR, and even more are on the horizon.

Participating in clinical trialscan open up new treatment options for patients with hATTR. Talk with your healthcare provider about the benefits and risks of participating in clinical trials.

Specialists and hATTR Amyloidosis Hospitals

You’ll need a group of healthcare providers to treat hATTR, including a hematologist (blood doctor), cardiologist (heart doctor), and neurologist (nerve doctor). The Amyloidosis Research Consortium has atoolthat can help you find healthcare providers in your area that are familiar with the disease.

Many major university medical schools, including John Hopkins University, Boston University, and the University of Chicago, have amyloidosis treatment programs.

Summary

Hereditary transthyretinamyloidosiscan be fatal, but there have been marked improvements in treatment in recent years. Medications and injections can stop or slow protein buildup, potentially sparing patients from more serious symptoms like heart disease and organ failure.

Working with a healthcare provider who is familiar with hATTR is crucial, since your treatment will likely involve many different medications and lifestyle adjustments.

6 SourcesVerywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M.Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: Current perspectives on improving patient care. Ther Clin Risk Manag. 2020. doi: 10.2147/TCRM.S219979Amyloidosis Research Consortium.Hereditary transthyretin amyloidosis.Amyloidosis Research Consortium.Onpattro™ (Patisiran) factsheet.Amyloidosis Research Consortium.Inotersen (Tegsedi) factsheet.Amyloidosis Research Consortium.Eplontersen (Wainua) factsheet.Amyloidosis Foundation.Hereditary amyloidosis.

6 Sources

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M.Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: Current perspectives on improving patient care. Ther Clin Risk Manag. 2020. doi: 10.2147/TCRM.S219979Amyloidosis Research Consortium.Hereditary transthyretin amyloidosis.Amyloidosis Research Consortium.Onpattro™ (Patisiran) factsheet.Amyloidosis Research Consortium.Inotersen (Tegsedi) factsheet.Amyloidosis Research Consortium.Eplontersen (Wainua) factsheet.Amyloidosis Foundation.Hereditary amyloidosis.

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.

Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M.Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: Current perspectives on improving patient care. Ther Clin Risk Manag. 2020. doi: 10.2147/TCRM.S219979Amyloidosis Research Consortium.Hereditary transthyretin amyloidosis.Amyloidosis Research Consortium.Onpattro™ (Patisiran) factsheet.Amyloidosis Research Consortium.Inotersen (Tegsedi) factsheet.Amyloidosis Research Consortium.Eplontersen (Wainua) factsheet.Amyloidosis Foundation.Hereditary amyloidosis.

Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M.Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: Current perspectives on improving patient care. Ther Clin Risk Manag. 2020. doi: 10.2147/TCRM.S219979

Amyloidosis Research Consortium.Hereditary transthyretin amyloidosis.

Amyloidosis Research Consortium.Onpattro™ (Patisiran) factsheet.

Amyloidosis Research Consortium.Inotersen (Tegsedi) factsheet.

Amyloidosis Research Consortium.Eplontersen (Wainua) factsheet.

Amyloidosis Foundation.Hereditary amyloidosis.

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