Treatment algorithm

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups: see disclaimer

ACUTE

mild acute attack

1st line – hemin

Back
ACUTE
|
mild acute attack
1st line – 

hemin

Intravenous hemin should be the initial treatment for most acute attacks.[15][16]​ Hemin is available in the US as lyophilized hematin and at many centers it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[27] Heme arginate is more stable, and is available in Europe and South Africa. Either product may have regulatory approval in some other countries.

Clinical improvement is rapid often within 1 to 2 days when hemin is started early in an attack. It should be continued for 3 to 4 days or until symptoms are resolving.[6]

Primary options

hemin: consult specialist for guidance on dose

Plus – supportive care

Back
ACUTE
|
mild acute attack
Plus – 

supportive care

Treatment recommended for ALL patients in selected patient group

Patients with acute attacks usually require admission to the hospital for treatment and monitoring.[6]​​

Intravenous fluid replacement may be required using 0.9% saline or 5.0% dextrose with normal saline.

Factors that may have precipitated the attack are identified and removed whenever possible.

2nd line – glucose

Back
ACUTE
|
mild acute attack
2nd line – 

glucose

May benefit some patients; however, it is considered to be less effective than hemin, so is used to treat an attack only if it is mild (i.e., mild pain [not requiring opioid analgesics]; absence of vomiting, seizures, hyponatremia, paresis, or other complications).[6]

Giving at least 300 g of dextrose (10% glucose) intravenously is recommended.

However, large volumes of intravenous glucose increase the risk of hyponatremia.

Plus – supportive care

Back
ACUTE
|
mild acute attack
Plus – 

supportive care

Treatment recommended for ALL patients in selected patient group

Patients with acute attacks usually require admission to the hospital for treatment and monitoring.[6][16]​

Intravenous fluid replacement may be required using 0.9% saline or 5.0% dextrose with normal saline.

Factors that may have precipitated the attack are identified and removed whenever possible.

severe acute attack

1st line – hemin

Back
ACUTE
|
severe acute attack
1st line – 

hemin

Intravenous hemin should be the initial treatment for most acute attacks.[15][16]​ Hemin is available in the US as lyophilized hematin and at many centers it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[27] Heme arginate is more stable, and is available in Europe and South Africa. Either product may have regulatory approval in some other countries.

Clinical improvement is rapid, often within 1 to 2 days when hemin is started early in an attack. It should be continued for 3 to 4 days or until symptoms are resolving.[6]

Primary options

hemin: consult specialist for guidance on dose

Plus – supportive care

Back
ACUTE
|
severe acute attack
Plus – 

supportive care

Treatment recommended for ALL patients in selected patient group

Patients with acute attacks usually require admission to the hospital for treatment and close monitoring for respiratory depression and other complications.[6][16]

Admission to an intensive care unit is indicated if respiration is impaired.

Intravenous fluid replacement may be required using 0.9% saline or 5.0% dextrose with normal saline.

Factors that may have precipitated the attack are identified and removed whenever possible.

Symptomatic treatment for accompanying pain (pain associated with severe attacks almost always requires opioids), nausea, vomiting, tachycardia and hypertension, depression, or seizures should be given under specialist advice.

ONGOING

recurrent noncyclic attacks

1st line – givosiran

Back
ONGOING
|
recurrent noncyclic attacks
1st line – 

givosiran

Preventive intervention is needed for the few patients who continue to have frequent attacks after known inciting factors are removed.

Givosiran is an aminolevulinate synthase 1-directed small interfering RNA approved for the treatment of acute hepatic porphyria in adults. It lowers delta-aminolevulinic acid and porphobilinogen (PBG), and reduces the frequency of recurrent attacks.[28]

Monitoring of PBG, hepatic and renal function, and blood homocysteine is recommended. Treatment should be interrupted or discontinued in patients with severe or clinically significant transaminase elevations.

Recurrent attacks are expected to become less frequent over time. Therefore, stopping a preventive treatment should be considered at some point to check if it is still needed.

Primary options

givosiran: 2.5 mg/kg subcutaneously once monthly

2nd line – hemin

Back
ONGOING
|
recurrent noncyclic attacks
2nd line – 

hemin

Hemin infusions once or twice weekly can prevent frequently recurring noncyclic attacks.[6][29] Hemin is available in the US as lyophilized hematin and at many centers it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[27] Heme arginate is more stable, and is available in Europe and South Africa. Either product may have regulatory approval in some other countries.

Serum ferritin levels should be monitored because there is some risk of iron overload. Ferritin levels should be measured as long as possible after the last dose of hemin.

Recurrent attacks are expected to become less frequent over time. Therefore, stopping a preventive treatment should be considered at some point to check if it is still needed.

Primary options

hemin: consult specialist for guidance on dose

women with frequent recurrent cyclic attacks

1st line – GnRH analog

Back
ONGOING
|
women with frequent recurrent cyclic attacks
1st line – 

GnRH analog

Attacks can be prevented by long-term administration of a gonadotropin-releasing hormone (GnRH) analog, which should be started during days 1 to 3 of the menstrual cycle.[10][30]

If a GnRH analog is effective, bone loss during long-term treatment can be prevented by estrogen add-back using a low-dose skin patch.

There is little evidence of the use of GnRH analogs such as leuprolide, nafarelin, and goserelin in AIP. Treatment should be under the guidance of a specialist.

Primary options

leuprolide: consult specialist for guidance on dose

OR

nafarelin nasal: consult specialist for guidance on dose

OR

goserelin: consult specialist for guidance on dose

nonresponsive to medical therapy

1st line – liver transplant

Back
ONGOING
|
nonresponsive to medical therapy
1st line – 

liver transplant

Liver transplant is an option for severe cases that do not respond to established medical therapies, and particularly hemin and givosiran.

Marked clinical and biochemical improvement has been reported in several severe cases after liver transplantation.[31][32]

An increased incidence of hepatic artery thrombosis has been reported in some patients with acute porphyria undergoing liver transplantation.[33][34]

back arrow

Choose a patient group to see our recommendations

Please note that formulations/routes and doses may differ between drug names and brands, drug formularies, or locations. Treatment recommendations are specific to patient groups. See disclaimer

Use of this content is subject to our disclaimer