A recent study has shed light on a potential treatment for Triglyceride Deposit Cardiomyovasculopathy (TGCV), a rare cardiovascular disorder that affects the body's ability to process triglycerides as an energy source. This disorder can lead to severe heart failure and coronary artery disease. The research, conducted on a Japanese population, marks a significant step towards understanding and potentially treating this obscure condition.
TGCV is characterized by the accumulation of triglycerides within heart and blood vessel cells, causing dysfunction that can result in serious health complications. The study involved 22 participants who received tricaprin, a medium-chain triglyceride, compared to 190 control participants who did not receive the supplement. All participants had confirmed diagnoses of TGCV, which they received at or after the age of twenty.
The collaboration between the Tochino Foundation and Osaka University played a crucial role in providing tricaprin to participants through the Patient Association. This partnership enabled ongoing communication with participants, ensuring their well-being throughout the study.
A notable finding from the study was that tricaprin not only improved heart failure outcomes among those participants who also suffered from heart failure but also demonstrated enhanced lipid metabolism. Scott C. Feitell, DO, FACC, FHFSA, highlighted the study's design and its outcomes, stating,
“This was a very well-designed trial with an excellent analysis done for a small patient population with a rare disease. Based on prior research, the team in this trial extended their proposed treatment model to a larger patient population, compared it to an untreated control arm, and followed patients for a longer period of time. They were able to not only demonstrate marked improvement in ejection fraction by echocardiography once patients received treatment, but they also used a novel radiotracer (I-123 BMIPP) to demonstrate enhanced lipid metabolism in patients on treatment. Most importantly patients felt better and had improved functional class.”
In examining the survival rates of participants, those receiving tricaprin exhibited a remarkable 100% three-year and five-year survival rate when considering only those with heart failure. In contrast, the control group had survival rates of 76.8% at three years and 64.8% at five years. However, it is important to note that one participant receiving tricaprin died 5.3 years after starting treatment.
While the findings are promising, the study's methodology included certain limitations. The timing of diagnosis in relation to tricaprin administration varied among participants, which could introduce bias regarding overall survival outcomes. Additionally, only three women in the intervention group had heart failure, a factor that may have influenced the results.
Cheng-Han Chen, MD, emphasized the complexity of TGCV in his remarks on the disorder:
“Triglyceride deposit cardiomyovasculopathy (TGCV) is a rare cardiovascular disorder in which the body’s heart and blood vessel cells are not able to properly process triglycerides as an energy source. This then causes heart muscle and coronary artery dysfunction, resulting in severe heart failure and coronary artery disease. The condition is not entirely understood, and effective treatment options have been lacking.”
The research opens avenues for future studies exploring whether tricaprin could offer similar cardiovascular benefits to patients with elevated triglyceride levels who do not have TGCV. Dr. Feitell noted:
“The potential clinical effects of this data is to design future studies to determine if tricaprin has similar cardiovascular effects (TG [triglyceride] regression) in patients with elevated triglyceride levels, but do not have TGCV. If the results show improvement as well, then tricaprin may be an additional treatment option for patients with elevated triglycerides.”
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