Important Safety Information
Prescribing Information
Indications and Usage
Support Icon

Contact a Recordati Rare
Diseases Representative

For Healthcare Professionals For Patients

Recognizing Acute
Intermittent Porphyria (AIP)

Acute intermittent porphyria (AIP) is a rare inherited disease caused by a partial deficiency of the enzyme porphobilinogen (PBG) deaminase in the heme biosynthetic pathway. PBG deficiency disrupts normal heme production, leading to overproduction of porphyrin precursors that cause diverse pathologic changes.1 The most common symptom is severe abdominal pain.2

Symptoms

- +

Recognize the signs and symptoms of AIP

There is wide variability in the clinical features of an acute AIP attack.3 Gastrointestinal, urinary, neurologic, and cardiovascular symptoms commonly occur.2

ABDOMINAL PAIN, which is neuropathic in origin, is the most common AIP symptom.2

An accurate, timely diagnosis of AIP is critical

Delayed treatment can result in serious consequences, including neurologic 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.2

Review diagnostic testing guidance

Incidence of Common AIP Symptoms*2

Incidence of Common AIP Symptoms* Table 2 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. *Based on several series of patients with symptomatic AIP. Dark or reddish urine is also a common symptom of AIP.2 See important safety information: https://www.panhematin.com/#important-safety-information

*Based on several series of patients with symptomatic AIP.
Dark or reddish urine is also a common symptom of AIP.2

Precipitating Factors

- +

Patient characteristics and precipitating factors

In differentiating AIP from other conditions with these common symptoms, certain patient characteristics and possible precipitating factors should heighten the suspicion of AIP.

AIP Patient Profile: Women of reproductive age

Acute AIP attacks are most common in women of reproductive age (most commonly in their 30s) during the luteal phase of their menstrual cycles.2-4

Exacerbating factors in AIP attacks

The enzyme deficiency of AIP predisposes patients to certain triggering factors that may lead to abnormally high accumulations of neurotoxic porphyrin precursors, which can precipitate an acute neurovisceral attack.7 Attacks are usually due to the additive effects of several precipitating factors,2 including:

  1. Endogenous steroid hormones, particularly estrogen and progesterone4
  2. Use of certain prescription drugs, especially barbiturates, hydantoins, rifampin, and sulfonamides,4 and often newly prescribed drugs5
  3. Use of alcohol or illicit drugs such as amphetamines, cocaine, and derivatives3
  1. Fasting4 or crash dieting2
  2. Cigarette smoking2,3
  3. Metabolic stress induced by infections or surgery2
  4. Psychological stress2

Family History

- +

Family history of AIP

An acute attack of AIP should be suspected in individuals experiencing symptoms consistent with AIP who also have a family history of the disorder. However, one third of patients have no family history of AIP.1,8

AIP is inherited in an autosomal dominant manner. The estimated prevalence of acute attacks and the estimated frequency of clinical pathogenic variants are low; as a result, AIP penetrance is approximately 1% of all heterozygotes.9

Review genetic testing information

Some individuals may have only one or a few attacks in their lifetimes. Others may have recurrent attacks throughout their lives.2,7,8

Potential Consequences

- +

Possible serious consequences of not recognizing AIP

Individuals with undiagnosed or inappropriately treated AIP attacks may suffer serious, potentially fatal, consequences, including:2

  • Peripheral neuropathy
  • Paresis
  • Respiratory and bulbar paralysis
  • Hepatocellular carcinoma
  • Chronic neuropathic pain
  • Renal impairment
  • Death resulting from cardiac arrhythmia or paralysis during an attack

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.2


Diagnosing and Managing AIP Review diagnostic testing guidance
About PANHEMATIN Review efficacy and safety data
Patient website - Acute Intermittent Porphyria Learn more about acute intermittent porphyria

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

+ See More

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. Gonzalez-Arriaza HL, Bostwick JM. Acute porphyrias: a case report and review. Am J Psych. 2003;160(3);450-458.
  2. 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.
  3. Thadani H, Deacon A, Peters T. Diagnosis and management of porphyria. BMJ. 2000;320(7250);1647-1651.
  4. Pischik E, Kauppinen R. An update of clinical management of acute intermittent porphyria. Dove Press Journal: The Application of Clinical Genetics. 2015; 204
  5. 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.
  6. Ventura P, Cappellini MD, Rocchi E. The acute porphyrias: a diagnostic and therapeutic challenge in internal and emergency medicine. Intern Emerg Med. 2009;4:297-308.
  7. 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.
  8. Whatley SD, Badminton MN. Acute Intermittent Porphyria. 2005 Sep 27 [Updated 2013 Feb 7]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019.
  9. Chen B, Solis-Villa C, Hakenberg J, Qiao W, Srinivasan RR, Yasuda M, Balwani M, Doheny D, Peter I, Chen R, Desnick RJ. Acute intermittent porphyria: Predicted pathogenicity of HMBS variants indicates extremely low penetrance of the autosomal dominant disease. Hum Mutat. 2016;37(11):1215-1222.

Important Safety Information

SIGN UP

Download the FREE Family Discussion Guide and opt in to receive future communications from Recordati Rare Diseases. Fill out the form below.

Please see Important Safety Information on this website

You are about to leave the PANHEMATIN.com website and enter a website operated by an independent third party. The links to third-party websites contained on PANHEMATIN.com are provided solely for your convenience. Recordati Rare Diseases does not control the opinions, claims or comments contained on any third-party website linked to PANHEMATIN.com, and your activities at those websites will be governed by the policies and practices of those third parties.

OK to proceed to third-party website CANCEL to return to PANHEMATIN.com