How is acute intermittent porphyria (AIP) treated?

Updated: Jul 08, 2020
  • Author: Thomas G DeLoughery, MD; Chief Editor: Emmanuel C Besa, MD  more...
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The treatment goal for acute attacks of porphyria is to decrease heme synthesis and reduce the production of porphyrin precursors. High doses of glucose can inhibit heme synthesis and are useful for treatment of mild attacks. United Kingdom guidelines recommend administering 5% glucose in 0.9% sodium chloride solution, infused intravenously at a rate of 2 L/24 h. Intravenous glucose in water solutions (eg, dextrose 5% or 10% [D5W, D10W]), should be avoided as they may aggravate hyponatremia. [13]

Patients experiencing severe attacks, especially those with severe neurologic symptoms, should be treated with hematin in a dose of 4 mg/kg/d for 4 days. Once hematin is initiated, glucose therapy no longer has a role. [13]

Pain can be remarkably severe, and pain control is best achieved with narcotics. Laxatives and stool softeners should be administered with the narcotics to avert exacerbating existing constipation.

Symptomatic treatment also includes the use of beta-blockers to control tachycardia and prevent arrhythmia; beta- blockers, clonidine, or other recommended antihypertensives can also be used to treat hypertensive crisis. Nausea and vomiting can be controlled with olanzapine, lorazepam, or prochlorperazine. [14]

Treat seizures with gabapentin. Most classic antiseizure medicines are contraindicated, as they can lead to acute porphyria attacks.

A minority of patients with acute intermittent porphyria (AIP) experience recurrent attacks. In addition to avoidance of precipitating factors, treatment options that may be considered in those cases include gonadotrophin-releasing hormone analogues (for women with attacks related to their menstrual cycles) and prophylactic hematin infusions. [13]

Givosiran (Givlaari) was approved by the US Food and Drug Administration (FDA) in 2019 for adults with acute hepatic porphyrias, in which attacks are caused induction of the enzyme 5-aminolevulinic acid synthase 1 (ALAS1). Givosiran is a small-interfering RNA that causes degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. It is given monthly by subcutaneous injection. [15]  

Rarely, liver transplantation may be indicated for patients with intractable recurrent attacks that are life-threatening or severely affect quality of life. Liver transplantation cures AIP. [13]

A comprehensive rehabilitation program, overseen by a physiatrist, can help patients regain functional independence after attacks of AIP. [16]

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