Treatment with haematin in acute hepatic porphyria.
We report our experience with intravenous haematin in the treatment of 13 attacks of acute porphyria in eight patients. Seven patients had acute intermittent porphyria and one variegate porphyria. Peripheral neuropathy was a feature of nine of the attacks and in two the neuropathy necessitated assisted ventilation. The haematin lowered the urinary excretion of porphyrins and precursors in all patients by approximately 50 per cent of the pre-haematin values. It also repressed the activity of delta-aminolaevulinic acid synthetase, the rate-controlling enzyme of haem biosynthesis, in peripheral leucocytes in seven of the nine patients in whom it was monitored. The clinical response was less consistent, with clinical improvement accompanying only half of the courses. The two patients with respiratory paralysis died. There was localized phlebitis at the injection site following five of the courses but no other side-effects were noted. Previously published reports of haematin therapy for acute porphyria are reviewed. These consist of 45 courses in 32 patients. Biochemical improvement was a consistent finding. Clinical response has been less consistent. Twenty-four of the courses were associated with sustained improvement, ten with temporary improvement--relapse occurring within two to 14 days (three fatal). In eleven there was no improvement and three died.
D000624:5-Aminolevulinate Synthetase / Q000097:blood; D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D005260:Female; D006418:Heme / Q000031:analogs & derivatives*; D006427:Hemin / Q000627:therapeutic use*; D006801:Humans; D007962:Leukocytes / Q000201:enzymology; D008107:Liver Diseases / Q000188:drug therapy* / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D011164:Porphyrias / Q000188:drug therapy* / Q000378:metabolism; D011166:Porphyrins / Q000652:urine
D011166:Porphyrins; D006418:Heme; D006427:Hemin; D000624:5-Aminolevulinate Synthetase