Treatment of chronic hepatic porphyria (PCT)

PCT, though largely genetically determined, can nevertheless be favorably influenced by several therapeutic methods and prolonged clinical and laboratory remission can be achieved. Two effective and reliable methods exist - repeated phlebotomy therapy and prolonged low-dose chloroquine. Although the exact mechanism underlying serial phlebotomy is not yet known, this therapy seems to be the most successful at present; it does not provoke serious adverse side effects, and extended remission after only one course can be expected. Chloroquine, about as effective in PCT, can cause acute toxic reactions at the beginning of its administration. However, when this occurs treatment need not be discontinued as no permanent ill-effects result. Both kinds of therapy can be used in the same patient consecutively. Although the data are sparse, a favorable effect of hydroxychloroquine has been noted. It can rapidly normalize urinary excretion of porphyrins in relapsing cases. The results of other methods hitherto recommended (i.e., chelators, AMP, desferrioxamine, metabolic alkalinization, plasmapheresis, etc.) have not been sufficiently evaluated either with regard to effectiveness or side effects. The use of some of these has already been abandoned.