Why is medication adherence important in treating FAP?

FAP can cause symptoms like peripheral neuropathy and heart problems

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by Ezekiel Lim |

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Medication adherence is crucial to managing familial amyloid polyneuropathy (FAP). Left untreated, FAP can have life-threatening consequences.

As an article published in U.S. Pharmacist notes, according to the World Health Organization (WHO), medication adherence can have a more direct impact on patient outcomes than even the specific treatments themselves. A 2018 article in The Permanente Journal notes that medication nonadherence may contribute to over 100,000 preventable deaths and $100 billion in preventable medical costs each year.

Ensuring that patients adhere to prescribed treatments is crucial. According to an article in the Oman Medical Journal, the risks of medication nonadherence include a worsening of the disease, increased treatment costs, and even possible death.

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The benefits of medication adherence for FAP patients

As FAP News Today explains, FAP is “caused by mutations in the TTR gene. These mutations lead to the production of misfolded transthyretin, a protein produced mainly in the liver. Transthyretin normally transports vitamin A and the thyroid hormone thyroxine in the blood and cerebrospinal fluid. Abnormal transthyretin can lead to deposits of amyloid fibrils — or protein clusters — inside nerve cells, and in the heart and gastrointestinal tract, causing the symptoms of this disease.”

FAP can cause symptoms such as peripheral neuropathy and heart and gastrointestinal problems. Medications such as Amvuttra (vutrisiran) and Onpattro (patisiran) may help to reduce the production of abnormal transthyretin protein. And medication adherence can help prevent the development of severe organ damage, reducing the need for serious invasive treatments such as organ transplants. Adherence to prescribed treatments can enable FAP patients to spend more time with loved ones and engage in meaningful life activities.

Always talk to your healthcare team before starting or stopping a particular medication. Follow your doctor’s advice about treatment options and protocols.

Living with a chronic, progressive condition like FAP can be emotionally challenging. During the three-year hiatus I took from writing this column, I witnessed my mother-in-law improve her physical well-being simply by adhering to her treatments. One marked improvement I noticed was in her mobility, which allowed her to hold her new granddaughter without experiencing peripheral neuropathy pain.

Healthcare providers, caregivers, and patients must work together to help maximize the potential benefits for FAP patients of intentional adherence to prescribed treatments.


Note: FAP News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of FAP News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.

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