Tafamidis: A New Treatment Option for ATTR-cm Patients
- Tafamidis is a new drug that was recently approved by the FDA in 2019 to improve the quality of life and survivorship for ATTR-cm patients as well as slow the progress of the disease and alleviate symptoms.
- Tafamidis works by targeting the mutated transthyretin gene (TTR gene) responsible for producing the abnormal transthyretin protein and preventing it from misfolding and depositing on organs.
- Although it doesn't reverse or cure ATTR-cm, it can slow the disease from progressing and stop severe complications. Thus, the success rate of this medication primarily relies on how early an ATTR-cm patient gets diagnosed the earlier, the better
In this article, we'll discuss the impact of tafamidis (Vyndaqel or Vyndamax) on treating transthyretin amyloid cardiomyopathy patients and how it significantly improved their quality of life and outlook. But first, let's learn more about the ATTR-cm. The only difference between Vyndaqel and Vyndamax is its composition quantity, thus, the dosage will differ.
Transthyretin Amyloid Cardiomyopathy (ATTR-cm) A Quick Guide
Read MoreCardiomyopathy. A term that signifies a heart muscle disease, which compromises its normal function.
There are two types of transthyretin amyloid cardiomyopathy, these are:
Hereditary transthyretin amyloid cardiomyopathy (hATTR-cm) This type of ATTR-cm generally runs in families, where they carry a mutated variant of the TTR gene which produces the abnormal transthyretin protein leading to its deposition. Symptoms for patients with hATTR-cm may appear anytime between 20-80 years old.
Wild-type transthyretin amyloid cardiomyopathy (wATTR-cm) This type of ATTR-cm appears sporadically and is closely associated with old age. Thus, symptoms generally appear in patients older than 65 years old.
You should be clear with your doctor about all the signs and symptoms you're experiencing to help them make an early diagnosis.
Below is a list of the most common symptoms of transthyretin amyloid cardiomyopathy (ATTR-cm):
- Shortness of breath (especially during inactivity or lying down)
- Irregular heartbeats (arrhythmias especially atrial fibrillation)
- Edema (swelling in the legs due to fluid retention)
- Tiredness and extreme fatigue
- Dizziness
- Fainting
- Bloated stomach
- Coughing and wheezing
- Confusion
- Disorientation
"You could have lightheadedness," says Dr. Philip Weintraub, a cardiologist at NYU Langone. "You could have fatigue, but a lot of that is based upon the fact that without the necessary oxygen that needs to be in your bloodstream, it'll compromise the body's ability to function properly. The heart works best when it's enriched with oxygen and nutrients, and to deprive the heart of that leads to a compromised lifestyle."
Nevertheless, not every patient will experience the same symptoms. Some may experience these symptoms in severe forms while others may have milder symptoms or no symptoms at all, this all depends on the following criteria:
- Type of ATTR-cm (hereditary or wild-type)
- Age of the patient
- The overalll health of the patient
- Other underlying medical conditions
Tafamidis A New Promising Treatment Option for ATTR-cm Patients
As stated by Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiovascular and Renal Drugs in the FDA's Center for Drug Evaluation and Research "Transthyretin-mediated amyloidosis is a rare, debilitating, and often fatal disease, The treatments we're approving today are an important advancement in the treatment of the cardiomyopathy caused by transthyretin-mediated amyloidosis."
Despite having no cure for ATTR-cm, the closest scientists have come to improve the quality of life and survivorship for ATTR-cm patients is to slow the progress of the disease and alleviate the symptoms through Tafamidis. But how does it work?
Tafamidis works by targeting the mutated transthyretin gene (TTR gene) responsible for producing the abnormal transthyretin protein and preventing it from misfolding and depositing on organs.
The FDA approved this drug on the 3rd of May 2019 after the success of a clinical trial of 441 patients with ATTR-cm who were categorized into two groups: the first group receiving the drug 'tafamidis' and the other group receiving a placebo for the duration of 30 months. Results revealed that patients who did receive tafamidis experienced a higher survival rate than those who received the placebo.
This clinical study was published in The New England Journal of Medicine in 2018, and the authors concluded that tafamidis is an effective treatment for patients with ATTR-cm as it provides the following functions:
- Reducing mortality and hospitalizations
- Improving the quality of life for patients with ATTR-cm
- Slowing down disease progress
Another review study published in the American Journal of Cardiovascular Drugs: Drugs, Devices and Other Interventions in 2021, reiterated the benefit of tafamidis for ATTR-cm patients. The author says: "Given that treatment for this condition has in the past been largely limited to symptom management, tafamidis constitutes a valuable disease-modifying therapy for patients with ATTR-CM."
Long-term effects of tafamidis have also been investigated in a published study in Circulation: Heart Failure as cited by the American Heart Association in 2022, patients were categorized into two groups: those who were still on tafamidis and others who took a placebo. The results revealed that for the duration of 58.5 months, those in the first group who took tafamidis should a much better survival rate than those who only took a placebo proving the superior effect tafamidis has on ATTR-cm patients.
The authors state "patients initially treated with tafamidis in ATTR-ACT had substantially better survival than those first treated with placebo, highlighting the importance of early diagnosis and treatment in transthyretin amyloid cardiomyopathy."
Are There Any Side Effects for Tafamidis?
Until now, there are no known side effects for Tafamidis, but it's recommended to consult your doctor first so that they can assess its safety for you and whether you're a suitable candidate.
That being said, it's absolutely prohibited to take Tafamidis if you're pregnant or breastfeeding as it may harm the baby.
What Are Other Treatment Options Available for ATTR-cm Besides Tafamidis?
Below is a list of other treatment options available to manage transthyretin amyloid cardiomyopathy symptoms:
- Inotersen or Tegsedi Drugs to slow the production of amyloid protein by the liver
- Medications to relieve symptoms caused by other comorbidities like heart failure or carpal tunnel syndrome (anticoagulants, antiarrhythmics, and diuretics)
- Organ transplantation surgery (Liver, kidney, or heart transplant) depending on the affected organ
Dr. Aeshita Dwivedi, a cardiologist at Lenox Hill Hospital, says trusts like Tegsedi "have been known as silencers of the protein, meaning they instruct the liver to stop producing the abnormal protein and are helpful in patients neuropathy or nerve disease related to TTR amyloid."
Additionally, you can ask your doctor about the possibility of enrolling in a clinical trial for transthyretin amyloid cardiomyopathy (ATTR-cm).
The Bottom Line
With the rising rates of transthyretin amyloid cardiomyopathy (ATTR-cm) cases, several researchers are trying to come up with new treatments that can improve the outlook for patients with minimal risk. FDA-approved Tafamidis meglumine (Vyndaqel) and Tafamidis (Vyndamax) are an example of such efforts.
Keep in mind that this medication doesn't reverse or cure ATTR-cm, it can only slow the disease from progressing and stop severe complications. Thus, the success rate of this medication primarily relies on how early an ATTR-cm patient gets diagnosed the earlier, the better. Moreover, A lot of clinical trials are underway for a potential new treatment that can cure transthyretin amyloid cardiomyopathy (ATTR-cm) with a good safety and efficacy profile.
Questions to Ask Your Doctor
- Which type of transthyretin amyloid cardiomyopathy (ATTR-cm) do I have?
- What are the symptoms associated with this disease?
- What are the pros and cons of taking tafamidis?
- What are other treatment options available for ATTR-cm?
- What are the disease complications and how can I avoid them?
- How can I improve my life expectancy?
- Are there any clinical trials suitable for me?
- Are there any support groups for ATTR-cm patients?
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