Treatment of porphyria cutanea tarda with oral thalidomide

Eight male patients with overt clinical and biochemical features of porphyr ia cutanea tarda (PCT) were orally treated with 300 mg/day thalidomide for 1 week and with 200 mg/day for 3 more weeks. Already after the first week o f treatment no new vesicles and/or bullae could be observed. Spontaneous bl isters completely disappeared, increased skin fragility subsided and skin h yperpigmentation receded about 2 months after completion of therapy, wherea s hypertrichosis persisted. There was a rapid decrease in the urinary total porphyrin excretion which reached normal levels in all patients by the end of the fourth week of therapy, whereas the post-treament chromatographic p attern of urinary porphyrins revealed a slight reduction of higher carboxyl ated porphyrin metabolites and an increase in the amount of the excreted co proporphyrin, as compared to the pretreatment period. Somnolence, intermitt ent constipation and dry mouth occurred in all patients, 2 patients additio nally experienced dizziness. No evidence of peripheral neuropathy could be detected and laboratory investigations revealed no abnormalities, as compar ed to the pretreatment period. During the 16- to 28-month follow-up of the patients, no clinical or biochemical relapse was observed. In view of the e ncouraging results of the present investigation, further studies are now wa rranted in order to definitely answer the question whether oral thalidomide may be regarded as an effective alternative approach to the treatment of P CT.