Patient Cases | PANHEMATIN® (hemin for injection)

Patient Cases

When to
Suspect AIP

Being aware of certain core considerations that increase the suspicion of AIP can help clinicians with diagnosis.

Introduction Female, 22: Recurrent Pain, Prior Surgeries Female, 40: Persistent Pain; Early Onset

Introduction

Key factors increasing suspicion of AIP1

Understanding certain key factors may help sharpen diagnostic reasoning when AIP is suspected. Taken together, these factors may allow for an increase or decrease in suspicion when determining what is and is not an AIP, allowing for prioritization of diagnostic testing and timely diagnosis.

  • Female patient: Symptoms most commonly develop between the 2nd and 4th decades of life.
  • Recurrent, severe abdominal pain accompanied by nausea and loss of appetite
  • Prior surgeries to relieve abdominal pain, to no effect
  • Use of pain relievers: 30% of AIP patients report daily use of pain medications.
  • Psychiatric diagnosis: Patients with frequent AIP attacks (3+/year) often report psychiatric symptoms.
  • Attack preceded by known triggers such as sulfa drugs and cigarette smoking: Attacks are usually due to the additive effects of several triggers.
  • Hyponatremia: Hyponatremia is the most common lab abnormality occurring during AIP attacks.
  • Reddish-brown urine: During attacks, 75% of AIP patients have urine that darkens to red or reddish-brown when exposed to light or air.

Female, 22: Recurrent Pain, Prior Surgeriesa,b

A portrait of a woman looking at the camera
Patient portrayal.

  • Chief Complaint: Severe abdominal pain
  • Symptom Onset & Duration: 48 hours prior to ER visit
  • Symptom Description: Aching, diffuse abdominal pain. Intensity: 9/10
  • Associated Symptoms: Nausea, decreased appetite
  • Setting: Emergency Room

Patient History

  • 2-year history of recurrent severe abdominal pain with nausea, decreased appetite
  • Prior surgeries: cholecystectomy, appendectomy, total abdominal hysterectomy (attempted to treat pain)
  • Long-term analgesic/opioid use (3 years)
  • Recent cystitis (treated with TMP-SMX)
  • MDD (diagnosed 3 years prior)
  • Smoker (½ pack daily × 7 years)

Treatment & Management

  • Hospitalized
  • Diagnosis of AIP

Triggers

  • Antibiotic use (for UTI)
  • Heavy smoking

Evaluation & Complications

  • Hyponatremia
  • Major depressive disorder
  • Possible recurrent UTI

Diagnosis

  • Hyponatremia
  • UA: possible UTI but already treated; WBC normal
  • Imaging: nonspecific abdominal/pelvic results
  • Reddish-brown urine on light exposure
  • Urinary PBG: 184 mg/L (normal: 0–4 mg/L)
  • Presumed AIP

Outcomes & Status

  • Presumed AIP diagnosis confirmed with elevated urinary PBG
  • Case details end at diagnosis (outcomes not documented)

Adapted from: Jones BJ, et al. 2014.2

This scenario may not be typical of all AIP patients.

Female, 40: Persistent Pain; Early Onsetc,d

Patient portrayal.

  • Chief Complaint: Recurrent abdominal pain related to known AIP
  • Symptom Onset & Duration: Recurrent attacks since late teens. Current pattern: temporally related to menstrual cycle
  • Symptom Description: Abdominal pain (severity varies with attacks)
  • Associated Symptoms: Confusion, sleep disturbances, discomfort during luteal phase. During acute attacks: fatigue, headache, nausea, allodynia, distal weakness
  • Setting: Outpatient clinic; prior ER and ICU admissions for severe attacks

Patient History

Early Years

  • Recurrent abdominal pain since late teens
  • Appendectomy (normal appendix)
  • Severe attack—paralysis, respiratory failure, 1-month ICU stay

Subsequent Years

  • Multiple attacks, some ICU-level
  • Permanent mild motor neuropathy + mild renal failure
  • Elevated urinary PBG at least once
  • Treated at multiple U.S. & European centers

Treatment & Management

Initial: Hemin therapy (effective, followed by rehabilitation)

Subsequent: Givosiran started ~3 years prior to clinic visit—no attacks during treatment period

Acute Attacks Post-Givosiran

  • Triggered after COVID mRNA vaccinations
  • Worsened despite carb loading
  • First attack: hemin (3 mg/kg × 3 days)—PBG normalized after 1st infusion
  • Second attack: hemin given despite normal PBG

Triggers

  • Menstrual cycle (luteal phase)
  • Prolonged fasting / low carbohydrate intake
  • COVID mRNA vaccinations

Evaluation & Complications

  • Mild chronic renal failure
  • Motor neuropathy (permanent, mild)
  • Hypertension, anemia, mild elevation of serum homocysteine
  • Episodes of confusion, sleep disturbances, abdominal discomfort (improved after nutrition counseling)

Diagnosis

  • AIP confirmed via PBG test + genetic testing
  • Severe attack led to immediate hemin therapy
  • Long-term diagnosis managed with hemin and later givosiran

Outcomes & Status

  • Continued givosiran therapy
  • Ongoing chronic complications (renal, neurologic, hematologic)
  • Occasional breakthrough attacks treated with PANHEMATIN

Adapted from: Moghe A, et al. 2023.3

This scenario may not be typical of all AIP patients.

Talk to a Recordati Rare Diseases representative to submit a case study

Because AIP is rare and often mimics more common diseases, patient case studies provide valuable insight into its identification, diagnosis, and treatment. Support other clinicians by submitting your own AIP case studies.

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References: 1. Anderson KE, Bloomer JR, Bonkovsky HL, et al. Recommendations for the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005 Mar 15;142(6):439-50. doi: 10.7326/0003-4819-142-6-200503150-00010 2. Jones BJ, Brzezinski WA, Estrada CA, Rodriguez M, Kraemer RR. A 22-year-old woman with abdominal pain. J Gen Intern Med. 2014:29(7):1074-1078. 3. Moghe A, Dickey A, Erwin A, et al. Acute hepatic porphyrias: Recommendations for diagnosis and management with real-world examples. Mol Genet Metab. 2023 Nov;140(3):107670.

INDICATION 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.

IMPORTANT SAFETY INFORMATION

PANHEMATIN is contraindicated in patients with known hypersensitivity to this drug.

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INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION 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.

IMPORTANT SAFETY INFORMATION

PANHEMATIN is contraindicated in patients with known hypersensitivity to this drug.

Risk of Phlebitis: Phlebitis is possible. Utilize a large arm vein or a central venous catheter for administration to minimize the risk of phlebitis.

Iron and Serum Ferritin: Elevated iron and serum ferritin may occur. Monitor iron and serum ferritin in patients receiving multiple administrations of PANHEMATIN.

Anticoagulant Effects: PANHEMATIN has transient and mild anticoagulant effect. Avoid concurrent anticoagulant therapy.

Renal Effects: Reversible renal shutdown has been observed with an excessive hematin dose (12.2 mg/kg in a single infusion). Strictly follow recommended dosage guidelines.

Transmissible Infectious Agents: PANHEMATIN may carry a risk of transmitting infectious agents, e.g., viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent. There is also the possibility that unknown infectious agents may be present in the product.

The most common adverse reactions (>1% of patients) are headache, pyrexia, infusion site reactions, and phlebitis.

To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Drug Interactions: 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.

PANHEMATIN® (hemin for injection), for intravenous infusion only, is available as powder for reconstitution in 350 mg vials.

Please see full Prescribing Information.