Long-term treatment of anemia with recombinant human erythropoietin in familial amyloidosis TTR V30M.

Familial amyloidosis or familial amyloid polyneuropathy (FAP) TTR V30M is a hereditary disease presented, in most cases, as a sensorimotor and autonomic neuropathy. Normocytic and normochromic anaemia was found in 24.8% of symptomatic FAP patients associated to lower serum erythropoietin (Epo) levels. Erythropoietin has been reported as efficient in anaemia correction in this disease. To evaluate the tolerance and efficacy of this treatment, a retrospective longitudinal study with 24 patients was undertaken. Patients were followed for at least 6 months. Haemoglobin, hematocrit, iron status, serum creatinine and urea and r-HuEPO doses were monitored, at 0, 3 months, 6 months and at the end of the follow-up. Long-term use of r-HuEPO proved to be efficient in the treatment of anaemia in familial amyloidosis TTR V30M and, despite the disease progression, no resistance cases to this treatment were observed. Positive side effects, like improvement on orthostatic hypotension symptoms and well-being sensation, contributing to confirm erythropoietin as a drug of choice to treat anaemia in amyloidosis TTR V30M.