Scavenger treatment of free radical injury in familial amyloidotic polyneuropathy: a study on Swedish transplanted and non-transplanted patients

Since oxidative stress has been implicated in amyloid diseases, a study of scavenger treatment of hereditary transthyretin amyloidosis was undertaken on 23 familial amyloidotic polyneuropathy (FAP) patients. Nine patients had undergone a liver transplantation for the disease. Twenty patients complet ed the 6-month study period of scavenger treatment (vitamin C, 1 g, three t imes daily, vitamin E, 0.1 g, three times daily and acetylcysteine, 0.2 g t hree times daily). They were evaluated clinically and by immunohistochemica l measurement of hydroxynonenal (HNE), a product of lipid peroxidation, in biopsy specimens. For non-transplanted patients, no improvement was found f or HNE in relation to the amyloid content in biopsy specimens, whereas a te ndency to a decreased amount was noted for transplanted patients. Clinicall y, no differences were found for non-transplanted patients, but an increase d nutritional status, measured by a modified body mass index (mBMI) was not ed for transplanted patients. In summary, scavenger treatment with the drug s and doses used in the present study appears to be unable to decrease lipi d peroxidation in amyloid-rich tissue in non-transplanted FAP patients. For transplanted patients, lipid peroxidation tended to decrease, and the nutr itional status measured by mBMI improved, even though the findings may be e xplained by liver transplantation alone, scavenger treatment may facilitate recovery after transplantation.