A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients exhibited cardiac and neurological symptoms.
According to a report in FAP News Today, researchers are now calling for teams of doctors whose expertise includes various specialties. It is called multidisciplinary management of disease.
ATTR amyloidosis is an umbrella term. It is associated with diseases characterized by a buildup of abnormal fibrous deposits of unstable transthyretin protein in the body’s organs and tissues.
This includes familial amyloid polyneuropathy or FAP, a form of hereditary ATTR amyloidosis that causes external nerve damage in and around the spinal cord and brain.
Amyloid’s fibrous protein deposits may accumulate in the brain, heart, kidneys, spleen or other body parts.
About the Survey
The THAOS survey is a global observational survey with a minimum ten-year duration. It is open to patients with transthyretin amyloidosis and is dependent upon a vast number of participants to understand the history of these diseases.
As anticipated, the findings of the researchers highlighted the various characteristics of these diseases. They found that the symptoms were mixed, and they also uncovered multiple bodily systems. The researchers were satisfied that they improved their knowledge of ATTR amyloidosis.
The data that has been collected will raise awareness for purposes of diagnosis. It will provide doctors with a more thorough understanding of the best methods to care for patients. The team is also anticipating the development of newer treatment guidelines.
The ATTR amyloidosis disorder is known to be difficult to diagnose due to a low disease awareness. The team notes that the study may lead to earlier intervention and therefore improved outcomes.
The study, which was funded by Pfizer, consists of a 15-year consolidated analysis of over 6,000 patients garnered from the THAOS trial.
Whether or not the patient has neurological and cardiac symptoms (or both) depends upon the patient’s particular type of hereditary ATTR amyloidosis. Pain tends to be high in FAP cases with 40% neurological and 30% cardiac symptoms or both.
The Fifteen Year Survey
The researchers ran the study from 2007 through 2022 reviewing data from 6,368 patients in 23 countries. Results showed 4,428 patients having symptoms while 1,707 patients had no symptoms. It is noteworthy that 24.5% of participants had both types together while 4.9% did not exhibit predominant symptoms.
The Val30Met mutation is a common mutation that causes FAP. Patients in Europe, South America, and Asia carried a high percent of Val30Met and were diagnosed with early-onset disease.
On the contrary, most of the patients in North America had wild-type amyloidosis. The mean age of patients in this group (56.6) was great in wild type.
The researchers noted the strengths of THAOS in that it represents a large geographical and diverse group of countries worldwide. Thus, in their opinion the survey presents an improved global picture of the status of ATTR amyloidosis.
