Canadian Association for Porphyria

Porphyria Treatments

Is porphyria treatable?

Simply put, the answer is yes. Prevention of the acute attacks in both known sufferers of the disease and suspected latent carriers is the most important approach. In a known porphyric patient it is essential to identify the factors that can precipitate the acute symptoms. The avoidance of porphyrogenic medications, bright sunlight, or alcohol is often all that is necessary to avoid these attacks. In women with repeated premenstrual relapses, the inhibition of ovulation by the use of pituitary hormones or the LHRH analogues such as leuprolide may be effective in reducing their frequency. Avoidance of starvation (excessive dieting, missing meals) is helpful.

During the acute attacks, supportive therapy is required including narcotic analgesics, tranquilizers such as chlorpromazine, antinauseants, rehydration, sodium and magnesium replacement, high carbohydrate diets, and intravenous therapy with high concentrations of glucose. In acute attacks especially those requiring hospitalization, early intravenous treatment with heme arginate, which is essentially the final product of the heme biosynthetic pathway, should be started in order to limit the attack and prevent neurological damage,

Heme arginate decreases the activity of the enzyme ALA synthase, the first step in the heme biosynthetic cascade. The reduced activity of this enzyme slows down the entire metabolic pathway and stops the overproduction of ALA and PBG. However this drug must be used with caution, as it can be associated with side effects.

During severe attacks the patient may require hospitalization. Seizures are often a difficult problem to control since many of the drugs used to control epilepsy such as phenytoin (Dilantin®) may precipitate or worsen attacks of porphyria. Anticonvulsants, such as gabapentin and clonazepam, have been shown to be effective and safe. Each type of porphyria has its own specific therapeutic program and it is important to try to identify the specific enzyme defect if possible. In cases of iron overload or lead toxicity the removal of the offending heavy metal excess may be all that is required. There appears to be some evidence that the treatment of hepatitis C may also be effective.

There are several drugs and medications used to treat other diseases that can precipitate an acute attack, but there are many more that are safe. It is preferable to take only the medicines that are absolutely essential. Before the patient with porphyria takes any drug they must ensure its safety. Lists of safe and unsafe drugs are now readily available on the Internet.

Source: This information originally appeared in a booklet written for the Canadian Porphyria Foundation: A Guide to Porphyria (1991) by Dr. Barry A. Tobe, MD, Ph.D, FRCP(C), Toronto, Ontario, Canada. The content has been updated by Dr. Brian M. Gilfix, MDCM, PhD, FRCPC, DABCC, FACB on 22/03/2015. 

Last Updated: 22/March/2015 

Medical Disclaimer

Care has been taken to ensure that the information on this website is accurate at the time of publication. This information is, however, intended for general guidance only and is not meant to substitute consultation from a recognized health professional. The Canadian Association for Porphyria/Association Canadienne de Porphyrie disclaims for itself and for the author of this information, all responsibility for any mis-statements or for consequences of actions taken by any person while acting on information contained herein. Physicians and patients must make their own decisions about therapy according to the individual circumstances of each case.

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