L-THREO-DOPS TREATMENT OF ORTHOSTATIC HYPOTENSION IN SWEDISH PATIENTSWITH FAMILIAL AMYLOIDOTIC POLYNEUROPATHY (TTR-MET(30))

L-threo-DOPS is a synthetic precursor to norepinephrine (NE). In order to study its efficacy and dosage, we carried out an open pilot study in ten patients with orthostatic hypotension and a confirmed diagnosis of familial amyloidotic polyneuropathy (FAP). The initial dosage was 200 mg of L-threo-DOPS daily, with a weekly increment of 100 mg, until a maintenance dose was reached. Ten patients were treated for 12 week s and five of them for 24 weeks. The blood pressure (BP) and pulse rat e were monitored weekly. Before treatment, after four and 12 weeks, th e patients underwent clinical and laboratory examinations. Nine patien ts completed the study. There was a rise in mean BP values in all meas urements. After 12 weeks of treatment the rise in mean systolic BP val ues were statistically significant (p < 0,01) in all measurements in u pright position. When treatment was withdrawn BP fell to almost the sa me levels as before and when treatment was restarted (five patients), there was again a rise in mean BP values. Before treatment, all patien ts suffered from mild to severe symptoms of orthostatic hypotension. A fter 12 weeks of treatment, eight out of nine patients were free of sy mptoms. Most frequent side effects were tachycardia and nausea, but no severe side effects were seen.