Treatment of Porphyria cutanea tarda with Oral Thalidomide

Abstract

Eight male patients with overt clinical and biochemical features of porphyria 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 of treatment no new vesicles and/or bullae could be observed. Spontaneous blisters completely disappeared, increased skin fragility subsided and skin hyperpigmentation receded about 2 months after completion of therapy, whereas 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 posttreament chromatographic pattern of urinary porphyrins revealed a slight reduction of higher carboxylated porphyrin metabolites and an increase in the amount of the excreted coproporphyrin, as compared to the pretreatment period. Somnolence, intermittent constipation and dry mouth occurred in all patients, 2 patients additionally experienced dizziness. No evidence of peripheral neuropathy could be detected and laboratory investigations revealed no abnormalities, as compared 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 encouraging results of the present investigation, further studies are now warranted in order to definitely answer the question whether oral thalidomide may be regarded as an effective alternative approach to the treatment of PCT.

Porfiria cutânea tardia

“…137 Other possible treatments are the slow administration of subcutaneous deferoxamine (iron chelating agent), 138 cholestiramine 139 and oral thalidomide. 140 In the follow-up, the coexisting hepatic disease is supervised and, in order to prevent relapses, urinary porphyrins are measured, because porphyrinuria precedes dermatological manifestations. …”

Dermatologic and Nondermatologic Uses of Thalidomide

“…91 A prompt decrease in the urinary porphyrin excretion was also detected. No relapses were recorded in any patient who completed a 16-month follow-up.…”

Cutaneous porphyrias part II: treatment strategies

“…Thalidomide, which inhibits the porphyrinogenic activity of hexachlorobenzene and increases hepatic P450 content in rats, was used in eight patients with PCT and is reported to have achieved good clinical improvement . All patients had overt clinical and biochemical features of PCT and were given the medication at a dose of 300 mg/d for one week, followed by 200 mg/d for three weeks.…”

“…Side effects experienced by all patients included somnolence, intermittent constipation, and dry mouth. During the 16‐ to 28‐month follow‐up of these patients, no clinical or biochemical relapses were observed . Although thalidomide may represent an alternative therapy in PCT, its use in PCT patients has not been studied further.…”