From the Guidelines
The management of acute porphyria requires prompt intervention with intravenous hemin (Panhematin) at 3-4 mg/kg daily for 3-4 days, along with high-dose carbohydrate loading (300-500g daily) via IV dextrose or oral glucose, as recommended by the most recent guidelines 1. The initial steps in managing acute porphyria include removing triggering factors such as medications, fasting, or hormonal fluctuations.
- Pain control is essential, typically with opioids like morphine or hydromorphone, as many analgesics are porphyrinogenic.
- Nausea and vomiting should be treated with safe antiemetics such as ondansetron.
- Seizures may require gabapentin or levetiracetam, as many anticonvulsants can worsen porphyria.
- Hyponatremia should be corrected cautiously with fluid restriction or hypertonic saline in severe cases.
- Autonomic instability may require beta-blockers like propranolol. Patients need close monitoring of vital signs, neurological status, and electrolytes, particularly sodium. The rationale for hemin therapy is to suppress hepatic delta-aminolevulinic acid synthase activity, reducing production of neurotoxic porphyrin precursors, while carbohydrate loading provides an alternative mechanism to downregulate this enzyme 1. Long-term management includes education about avoiding triggers, genetic counseling, and consideration of prophylactic hemin for recurrent attacks, as outlined in recent clinical practice updates 1. It is also important to consider the chronic and long-term complications of AHP, including neuropathy, CKD, hypertension, and HCC, and the need for long-term monitoring 1. The diagnosis of AHP should be made by biochemical testing measuring ALA, PBG, porphyrins, and creatinine in a random urine sample, as recommended by recent guidelines 1. Genetic testing can confirm the diagnosis and identify the specific type of AHP, which is essential for long-term management and family planning 1. Overall, the management of acute porphyria requires a comprehensive approach that includes prompt treatment of acute attacks, long-term monitoring, and education on avoiding triggers and managing chronic complications.
From the FDA Drug Label
For mild porphyric attacks (mild pain, no vomiting, no paralysis, no hyponatremia, no seizures), a trial of glucose therapy is recommended while awaiting hemin treatment or if hemin is unavailable. For moderate to severe attacks, immediate hemin treatment is recommended Symptoms of severe attacks are severe or prolonged pain, persistent vomiting, hyponatremia, convulsion, psychosis, and neuropathy. The dose of PANHEMATIN is 1 to 4 mg/kg/day of hematin for 3 to 14 days based on the clinical signs The standard dose in clinical practice is 3 to 4 mg/kg/day.
The management approach for a patient presenting with acute porphyria includes:
- Glucose therapy for mild attacks
- Hemin treatment for moderate to severe attacks, with a dose of 1 to 4 mg/kg/day of hematin for 3 to 14 days
- Monitoring of urinary concentrations of certain compounds, such as ALA, PBG, uroporphyrin, and coproporphyrin, to assess effectiveness of treatment 2
- Elimination of triggering factors and other necessary measures, such as supportive care, to manage symptoms and prevent further attacks
- Clinical assessment to determine the severity of the attack and the need for immediate treatment 2
From the Research
Management Approach for Acute Porphyria
The management of acute porphyria involves several strategies, including:
- Treatment with heme preparations for severe or moderate acute attacks 3
- Symptomatic treatment of autonomic dysfunctions, polyneuropathy, and encephalopathy 3
- Exclusion of precipitating factors, such as certain medications or hormonal changes 3, 4
- Adequate nutrition and fluid therapy to support the patient during an acute attack 3
Diagnostic Criteria
The diagnosis of acute porphyria is based on the presence of certain symptoms, including:
- Abdominal pain without peritoneal signs 3, 5
- Acute peripheral neuropathy 3, 5
- Encephalopathy with seizures or psychosis 3, 5
- Elevated urinary porphobilinogen excretion, typically more than fivefold 3
Treatment Options
Treatment options for acute porphyria include:
- Heme therapy, which can be effective in reducing symptoms and preventing complications 3, 6
- Symptomatic treatment, such as pain management and anti-emetics 3, 5
- Prophylactic heme therapy to prevent recurrent attacks 3, 6
- Liver transplantation in patients with severe recurrent attacks 3
Long-term Complications
Patients with acute porphyria are at risk for long-term complications, including: