Misdiagnosis and underdiagnoses of a disease considerably reduces the benefits various therapeutic interventions and therapies bring to patients. This may prove fatal if the disease is life-threatening. Transthyretin amyloid cardiomyopathy (ATTR-CM), a rare but more common disease than clinicians believe, confers critical morbidities in patients as it rapidly progresses to advanced heart failures causing death. Given the paucity of noninvasive imaging for diagnosing the disease until recently, the management has been increasingly difficult for clinicians. However, with advancements in diagnostics, detection of the conditions is easy with a minimally-invasive imaging test. Nonetheless, it is still a challenge to rein in over the mortality rate of ATTR-CM. A study at phase III stage has sought to find a way out.
Drug promising for reducing Cardiovascular-related Hospitalizations
Researchers at Columbia University Medical Center in the clinical trial has confirmed that the drug tafamidis has been effective in substantially reducing cardiovascular-related hospitalizations and death in patients. The drug if approved by the FDA will be the first medical therapy for this progressive form of heart cancer and will bring improvement in the quality of the life.
The findings of the clinical trial are published recently in the New England Journal of Medicine.
The researchers in the clinical trial spanning two and a half year found that tafamidis in 441 residents reduced the hospitalizations by as much as 32% compared to a placebo. The drug also slowed down the decline in the heart functions of the patients reducing the effect of the disease on the quality of life. The researchers also stated that the drug has no noticeable side-effects.
Patients after having been diagnosed with ATTR-CM live only for 2-5 Years
ATTR-CM is one of the conditions leading to amyloid-related heart failure. The disease caused by dysregulation in the protein transthyretin is prevalent in elderly men over 60 and is associated with substantial mortality; the patients after the diagnosis have life span on only three to five years.
The researchers are hoping that the FDA will soon consider the approval of the drug, paving the way for treating transthyretin amyloid cardiomyopathy. However, challenges still remain, believe one of the researchers, since patients are diagnosed with ATTR-CM only in advanced stages.