The study published in the Annals of Neurology, titled “Disease-Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy,” sheds light on the effectiveness of treatments for familial amyloid polyneuropathy (FAP).
FAP, caused by mutations in the transthyretin gene, results in the accumulation of toxic protein clumps in nerve tissues, leading to debilitating symptoms. While there is no cure, treatment options include disease-modifying drugs (DMDs) and liver transplants.
Researchers analyzed data from 201 FAP patients over three decades, observing significant differences in survival outcomes among those receiving treatment versus those untreated.
Notably, nearly all treated #patients were estimated to be alive ten years after disease onset, compared to less than half of untreated patients.
DMDs, such as Onpattro, tafamidis, and diflunisal, showed promising results in improving survival, regardless of the age at disease onset.
Even patients with late-onset FAP experienced better survival with DMDs, challenging the notion that liver transplants were the primary treatment for early-onset cases.
While liver transplants prolonged survival in early-onset FAP, their impact on late-onset cases was less significant.
The study highlights the importance of DMDs in improving long-term survival for FAP patients, regardless of disease onset age or symptom type.
However, the analysis is limited to a single center, and further studies are needed to validate these findings across different populations and medical settings.
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