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 – haemin

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ACUTE
|
mild acute attack
1st line – 

haemin

Intravenous haemin should be the initial treatment for most acute attacks.[15][16]​ Haemin is available in the US as lyophilised haematin and at many centres it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[24] Haem 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 haemin is started early in an attack. It should be continued for 3 to 4 days or until symptoms are resolving.[6]

Primary options

haem arginate: consult specialist for guidance on doses

Plus – supportive care

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ACUTE
|
mild acute attack
Plus – 

supportive care

Treatment recommended for ALL patients in selected patient group

Patients with acute attacks usually require admission to 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

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ACUTE
|
mild acute attack
2nd line – 

glucose

May benefit some patients; however, it is considered to be less effective than haemin, so is used to treat an attack only if it is mild (i.e., mild pain [not requiring opioid analgesics]; absence of vomiting, seizures, hyponatraemia, 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 hyponatraemia.

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 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 – haemin

Back
ACUTE
|
severe acute attack
1st line – 

haemin

Intravenous haemin should be the initial treatment for most acute attacks.[15][16]​ Haemin is available in the US as lyophilised haematin and at many centres it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[24] Haem 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 haemin is started early in an attack. It should be continued for 3 to 4 days or until symptoms are resolving.[6]

Primary options

haem arginate: 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 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 non-cyclic attacks

1st line – givosiran

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ONGOING
|
recurrent non-cyclic 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.[25]

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 – haemin

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ONGOING
|
recurrent non-cyclic attacks
2nd line – 

haemin

Haemin infusions once or twice weekly can prevent frequently recurring non-cyclic attacks.[6][26] Haemin is available in the US as lyophilised haematin and at many centres it is reconstituted with human albumin, rather than sterile water, in order to enhance stability.[24] Haem arginate is more stable, and is available in Europe and South Africa.

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 haemin.

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

haem arginate: consult specialist for guidance on dose

women with frequent recurrent cyclic attacks

1st line – GnRH analogue

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

GnRH analogue

Attacks can be prevented by long-term administration of a gonadotrophin-releasing hormone (GnRH) analogue, which should be started during days 1 to 3 of the menstrual cycle.[10][27]

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

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

Primary options

leuprorelin: consult specialist for guidance on dose

OR

gonadorelin: consult specialist for guidance on dose

OR

nafarelin: consult specialist for guidance on dose

OR

goserelin: consult specialist for guidance on dose

non-responsive to medical therapy

1st line – liver transplant

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ONGOING
|
non-responsive 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 haemin and givosiran.

Marked clinical and biochemical improvement has been reported in several severe cases after liver transplantation.[28][29]

An increased incidence of hepatic artery thrombosis has been reported in some patients with acute porphyria undergoing liver transplantation.[30][31]

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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

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