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Diagnosing and Managing AIP

With acute intermittent porphyria (AIP), an accurate, timely diagnosis is critical. Delayed treatment can result in neurological damage or even death. AIP should be considered in any patient with symptoms that are prominent for AIP, particularly abdominal pain, when initial clinical evaluation does not support another cause.1

Diagnostic testing for AIP

Initial testing

If AIP is suspected, the diagnosis should be confirmed with a porphobilinogen (PBG) urine test.1 Prompt treatment of symptoms and disease-specific therapy should follow.

Urinary PBG levels are substantially increased (20–200 mg/L) in patients with acute attacks of AIP and other acute porphyrias. Testing for urinary PBG that occurs at or near the time of symptoms will confirm the initial diagnosis of acute porphyria, except in patients who are already receiving hemin.1

Second-line testing

If a patient's PBG level is increased, second-line testing will determine the precise acute porphyria. These tests may include PBG, ALA, and porphyrin testing on the same urine sample, as well as testing for plasma and fecal porphyrins and erythrocyte PBG deaminase. Treatment should not be delayed pending these results.1

Laboratory findings that differentiate AIP from other acute porphyrias include:1

Urine porphyrin levels
(mostly uroporphyrin) markedly increased

Fecal porphyrin levels
normal or slightly increased

Plasma porphyrin levels
normal or slightly increased




Erythrocyte PBG
deaminase levels decreased by ~50%

Diagnostic tests are available through major clinical laboratory testing companies.

Genetic testing for family members

AIP is an inherited disease. These tests can help to identify the patient's family members who may be at risk:1

  • Erythrocyte PBG deaminase levels: This test may be useful, but note that normal activity does not exclude AIP.
  • DNA studies: These tests can identify the disease-causing mutation(s) in the defective gene.

Knowledge of genetic status enables family members to make informed decisions about lifestyle and to understand the potential risks of the use of certain drugs, preferably before the development of acute illness. Download a guide to help your patients discuss AIP with their families.

Managing AIP symptoms and precipitating factors

Acute AIP attacks require prompt treatment of symptoms and disease-specific therapy to restore heme homeostasis in order to prevent an attack from reaching the critical stage of neuronal degeneration.2 It is also critical to identify and eliminate any potential precipitating factors.

Treatment considerations for patients with AIP

These steps will help keep your patients safe and on the road to recovery:

  1. Take patients off of all unsafe medications.
    Many drugs, including some that are prescribed to treat AIP symptoms, can increase the demand for hepatic heme and induce ALA synthase. This may precipitate or worsen AIP attacks and should be avoided.4 Lists of unsafe, possibly unsafe, and safe drugs for patients with AIP are continuously updated. Review the Porphyria Foundation's AIP Drug Database
  2. Consider hospitalization as needed to control acute symptoms.1
  3. Provide nutritional support, generally intravenously.
    Carbohydrate loading may provide nutritional replacement and may have some repressive effect on hepatic ALA synthase.1
  1. Monitor for potentially life-threatening developments.
    These include electrolyte imbalances, acute psychiatric manifestations, muscle weakness, bladder distention, and ileus.1
  2. Talk to your patients about potential precipitating factors that they may be able to modify.
    These include cigarette smoking, alcohol or other drug misuse, and crash dieting. Refer patients to resources or counseling as appropriate.
  3. Encourage patients to wear a medical alert bracelet or carry a wallet card.
    This will help to alert medical personnel of the patient’s AIP and ensure that unsafe drugs are not given.1

Review clinical information for PANHEMATIN® (hemin for injection) as a treatment option for AIP



About PANHEMATIN Review efficacy and safety data
Dosing & Administration Get step-by-step instructions

Indications And Usage

PANHEMATIN® is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women, after initial carbohydrate therapy is known or suspected to be inadequate.

Limitations of Use

  • Before administering PANHEMATIN, consider an appropriate period of carbohydrate loading (i.e., 400 g glucose/day for 1 to 2 days).
  • Attacks of porphyria may progress to a point where irreversible neuronal damage has occurred. PANHEMATIN therapy is intended to prevent an attack from reaching the critical stage of neuronal degeneration. PANHEMATIN is not effective in repairing neuronal damage.

Indications and Important
Safety Information

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Indications And Usage

PANHEMATIN® is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women, after initial carbohydrate therapy is known or suspected to be inadequate.

Important Safety Information

  • Do not use in patients with known hypersensitivity to PANHEMATIN.
  • Phlebitis is possible. Utilize a large arm vein or a central venous catheter for administration to minimize the risk of phlebitis.
  • Elevated iron and serum ferritin may occur. Monitor iron and serum ferritin in patients receiving multiple administrations of PANHEMATIN.
  • PANHEMATIN has transient and mild anticoagulant effect. Avoid concurrent anticoagulant therapy.
  • Reversible renal shutdown has been observed with an excessive hematin dose (12.2 mg/kg in a single infusion). Strictly follow recommended dosage guidelines.
  • PANHEMATIN may carry a risk of transmitting infectious agents, e.g., viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
  • Most common adverse reactions in >1% of patients are headache, pyrexia, infusion site reactions, and phlebitis.
  • To report SUSPECTED ADVERSE EFFECTS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1800-FDA-1088 or www.fda.gov/medwatch.
  • Avoid CYP inducing drugs such as estrogens, barbituric acid derivatives and steroid metabolites which induce δ‑aminolevulinic acid synthetase 1 (ALAS1) through a feedback mechanism.
For more information, please see PANHEMATIN Full Prescribing Information at www.recordatirarediseases.com.

References

  1. Anderson KE, Bloomer JR, Bonkovsky HL, Kushner JP, Pierach CA, Pimstone NR, Desnick RJ. Recommendations for the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005;142:439-450.
  2. PANHEMATIN® package insert. Recordati Rare Diseases Inc., Lebanon, NJ 08833 USA; 2017
  3. Stein P, Badminton M, Barth J, Rees D, Stewart MF. Best practices guidelines on clinical management of acute attacks of porphyria and their complications. Ann Clin Biochem. 2013;50:217-223.
  4. Balwani M, Wang B, Anderson KE, Bloomer JR, Bissell DM, Bonkovsky HL, Phillips JD, Desnick RJ; for the Porphyrias Consortium of the Rare Diseases Clinical Research Network. Acute hepatic porphyrias: Recommendations for evaluation and long-term management. Hepatol. 2017;66(4):1314-1322.

Important Safety Information

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