What is the treatment for acute porphyria?

From the Guidelines

The treatment for acute porphyria focuses on managing attacks and preventing complications, with intravenous hemin being the primary treatment for acute attacks, as recommended by the Porphyrias Consortium of the NIH’s Rare Diseases Clinical Research Network 1. During an acute attack, patients should receive intravenous hemin (Panhematin or Normosang) at 3-4 mg/kg daily for 3-4 days. This helps suppress heme production by replenishing the body's heme pool. Some key points to consider in the management of acute porphyria include:

• Glucose loading with IV dextrose (300-500g daily) can be used for mild attacks or while waiting for hemin.
• Pain management typically requires opioid analgesics like morphine.
• Nausea and vomiting are treated with antiemetics such as ondansetron.
• Seizures may require gabapentin or levetiracetam, as many anticonvulsants can worsen porphyria.
• Hyponatremia should be corrected with appropriate fluid management. Long-term management includes avoiding trigger factors like certain medications (barbiturates, sulfonamides, estrogens), alcohol, fasting, and stress, as identified and counseled by the patient's healthcare provider 1. For patients with recurrent attacks, prophylactic hemin infusions or GnRH analogs in women with menstrual-related attacks may be beneficial. Givosiran, an RNA interference therapeutic that reduces aminolevulinic acid synthase 1, is approved for preventing attacks in adults with acute hepatic porphyria. Liver transplantation may be considered in severe cases with frequent, life-threatening attacks.

From the FDA Drug Label

PANHEMATIN therapy for the acute porphyrias is not curative. After discontinuation of PANHEMATIN treatment, symptoms generally return although in some cases remission is prolonged. The effectiveness of PANHEMATIN for the amelioration of recurrent attacks of acute intermittent porphyria was evaluated in five open-label studies, one compassionate-use study, case reports, and an observational study investigating patient reported outcomes in patients with acute porphyrias In the initial 5 open-label studies,1-5 99 patients with acute porphyrias (72 with AIP) were treated with 3-4 mg/kg/day of hemin once or twice daily.

The treatment for acute porphyria is PANHEMATIN (hemin for injection), which is administered intravenously. The recommended dose is 3-4 mg/kg/day of hemin, given once or twice daily. Key points about the treatment include:

• Symptomatic improvement: PANHEMATIN therapy can produce symptomatic improvement in patients with acute episodes of the hepatic porphyrias.
• Non-curative: PANHEMATIN therapy is not curative, and symptoms generally return after discontinuation of treatment.
• Remission: In some cases, remission may be prolonged after treatment.
• Dosing: The dose of PANHEMATIN is typically 3-4 mg/kg/day, given once or twice daily 2.

From the Research

Treatment for Acute Porphyria

• The primary treatment for acute porphyria is heme therapy, which has been shown to be effective in reducing the duration and severity of attacks 3, 4, 5.
• Heme arginate is the preferred form of heme therapy due to its better stability, fewer side effects, and better documentation of its benefits 5.
• Treatment with heme preparations should be started immediately if an acute attack is severe or moderate 6.
• Symptomatic treatment of autonomic dysfunctions, polyneuropathy, and encephalopathy is also important during an acute attack 6.
• Exclusion of precipitating factors, adequate nutrition, and fluid therapy are also crucial in managing acute porphyria 6.
• Heme arginate treatment has been shown to be effective in reducing the duration of abdominal pain and hospitalization time 3, 4.
• Early initiation of heme therapy is critical in achieving a favorable response 3, 4, 7.

Heme Therapy Administration

• Heme arginate can be administered in a dose of 250 mg or 3 mg/kg, with four daily infusions given during an acute attack 3.
• The treatment should be started within 24 hours of admission, and ideally within 4 days of the onset of symptoms 3, 4.

Importance of Early Diagnosis and Treatment

• Early diagnosis of acute porphyria is essential to prevent serious neurological complications, which can be precipitated by porphyrinogenic medications 7.
• Early treatment with heme arginate can help reduce the risk of long-term complications, such as chronic hypertension, chronic kidney insufficiency, and hepatocellular carcinoma 6.