Friday, March 31, 2017

Effective Management of Acute Intermittent Porphyria: Part 3

Effective Management of Acute Intermittent Porphyria:
Patients Deserve Access to Appropriate Care

What is Acute Intermittent Porphyria (AIP)?
AIP is one of a group of rare, inherited disorders called porphyrias that all involve the
overproduction and buildup of chemicals called porphyrins or porphyrin precursors. AIP is

Patients Diagnosed with AIP Deserve Access to Treatment
For people living with Acute Intermittent Porphyria (AIP), there are treatments available, but many
still encounter barriers to receiving accessible care at their preferred hospitals.

The American Porphyria Foundation (APF) urges all healthcare institutions to provide timely access
to medically necessary care. We encourage everyone involved in the care of patients with AIP to
demand timely access to FDA-approved treatments. We believe:

Regulators should advocate for patient access to approved treatments
Providers need to understand AIP and provide prompt, appropriate treatment Patients deserve access
and coverage for available, life-saving treatment  options
deaminase) and is characterized by acute attacks, often triggered by environmental factors or
hormone changes. In some cases, the cause

Signs and Symptoms


Living with AIP

Because the symptoms of AIP mimic other common conditions, the diagnostic process can be long
suffering for years before receiving a proper diagnosis.
• Severe, intense abdominal pain
• Nausea and vomiting
• Constipation
• Diarrhea
• Pain in the extremities, back, chest, neck,or head
•  Muscle weakness
• Convulsions
• Mental symptoms
• Respiratory paralysis
• Fast heart beat
• High blood pressure
•Dark reddish or purple urine

This educational material is provided by the American Porphyria Foundation.
For more information,visit

Managing AIP
AIP is a genetic disease and there is no cure. However, there are strategies that can be used to
prevent the onset or severity of attacks:

•  Avoiding dieting or fasting, even for short periods
•  Avoiding certain medications that can trigger an attack
•  Limiting physical and emotional stress
•  Avoiding alcohol

People with frequent attacks can also consider wearing a medical alert bracelet.

1.  Anderson KE, Bloomer JR, Bonkovsky HL, et al. Recommendations for
the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005;142:439-450.

2.   Bonkovsky HL, et al. Acute porphyrias in the USA: features of 108 subjects from porphyrias
consortium. The Amer. Jrnl of Med. 2014;127: 1233-1241.

3.   Crimlisk HL. The little imitator-porphyria: a neuropsychiatric disorder. J Neurol Neurosurg
Psychiatry. 1997;62:319-328.

Prompt Treatment is Critical
Even with proper diet and control of environmental factors, some patients still have recurring, and
often severe attacks. Acute attacks typically require hospitalization and delays in treatment can
be life-threatening and result in serious complications such as irreversible nerve damage.

Treatments can include:
•   Medication to manage pain and other symptoms
• Glucose and carbohydrates given orally or intravenously
•  Intravenous heme if the patient’s symptoms fail to improve within 36 hours of glucose treatment

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