Abstract
Background Diflunisal is a well known FDA-registered commonly used NSAID therapy in the USA since the 1970’s. In Europe, the drug has been seldom authorised on a national basis with the next safety update report scheduled for 2025 (http://www.ema.europa.eu/ema/). Spain is one such country where commercial use has not been authorised, likely because of concerns on liver hypersensitivity and availability of other NSAIDs. Interestingly, recent advances have shown a potential beneficial effect in transthyretin (TTR) hereditary amyloidosis, as evidenced by encouraging data from the diflunisal trial consortium (Berk et al. JAMA. 2013;310(24):2658-2667) where quality of life, neuropathy impairment scores and nutritional status showed significant, though modest, better results in patients randomised to receive diflunisal instead of placebo.
Highlights
Diflunisal is a well known FDA-registered commonly used NSAID therapy in the USA since the 1970’s
We aimed at describing the first off-label use of diflunisal in a small cohort of 10 patients affected by variable degrees of transthyretin familial amyloidotic polyneuropathy (TTR-FAP) in our centre
Inclusion criteriae consisted of any symptomatic hereditary TTR-amyloidosis patient with progression of FAP either (i) unfit or unwilling to receive either liver transplantation (LT)/Vyndaqel® as per on-label indication or to enter an ongoing clinical trial (e.g. RNA silencing), (ii) already under on-label treatments (Vyndaqel®, liver transplantation (LT)) or (iii) any Domino-LT (DLT) recipient with de novo signs/symptoms of polyneuropathy with biopsy proven culprit ATTR deposits
Summary
Diflunisal is a well known FDA-registered commonly used NSAID therapy in the USA since the 1970’s. In Europe, the drug has been seldom authorised on a national basis with the safety update report scheduled for 2025 (http://www.ema.europa.eu/ema/). Spain is one such country where commercial use has not been authorised, likely because of concerns on liver hypersensitivity and availability of other NSAIDs. Interestingly, recent advances have shown a potential beneficial effect in transthyretin (TTR) hereditary amyloidosis, as evidenced by encouraging data from the diflunisal trial consortium 2013;310(24):2658-2667) where quality of life, neuropathy impairment scores and nutritional status showed significant, though modest, better results in patients randomised to receive diflunisal instead of placebo Recent advances have shown a potential beneficial effect in transthyretin (TTR) hereditary amyloidosis, as evidenced by encouraging data from the diflunisal trial consortium (Berk et al JAMA. 2013;310(24):2658-2667) where quality of life, neuropathy impairment scores and nutritional status showed significant, though modest, better results in patients randomised to receive diflunisal instead of placebo
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