II. Indications

  1. Topical: Unwanted facial hair
    1. See Eflornithine Cream
  2. Systemic: Cancer
    1. Neuroblastoma
  3. Systemic: Protozoa
    1. Severe Stage 2 West African Trypanosomiasis (T. gambiense) with CSF Leukocytes >100 wbc/uL (any age)
      1. Used in a combination known as NECT (with Nifurtimox)

III. Mechanism

  1. Difluoromethylated Ornithine with antineoplastic and Antiprotozoal activity
  2. Eflornithine irreversibly inhibits Ornithine decarboxylase, a key enzyme in polyamine biosynthesis
    1. Inhibiting polyamine synthesis suppresses tumor cell formation and proliferation
    2. Also suppresses Protozoal growth and replication

IV. Dosing: West African Trypanosomiasis

  1. Eflornithine renal dose adjustment needed for eGFR <30 ml/min
  2. NECT (Eflornithine/Nifurtimox)
    1. Indications
      1. Severe Stage 2 West African Trypanosomiasis with CSF Leukocytes >100 wbc/uL (any age)
    2. Dosing
      1. Eflornithine 400 mg/kg/day IV divided every 12 hours for 7 days AND
      2. Nifurtimox 15 mg/kg/day divided every 8 hours for 10 days

V. Adverse Effects

  1. Gastrointestinal
    1. Vomiting
    2. Diarrhea
    3. Abdominal Pain
    4. Hepatotoxicity
  2. Hematologic (Bone Marrow suppression)
    1. Anemia
    2. Leukopenia
    3. Thrombocytopenia
  3. Neurologic
    1. Seizures
    2. Headache
  4. Miscellaneous
    1. Alopecia
    2. Hearing Impairment

VI. Safety

  1. Monitoring
    1. Complete Blood Count with Platelet Count
      1. Baseline before starting therapy
      2. Repeat twice weekly during therapy
      3. Repeat weekly after stopping therapy until normalizes
  2. Lactation
    1. May continue Lactation while taking Eflornithine
  3. Pregnancy
    1. Avoid if possible during pregnancy
    2. However, may be used for rescue therapy in West African Trypanosomiasis if alternatives are unavailable

VII. References

  1. Freedman (2025) Sanford Guide, accessed on IOS, 7/12/2025
  2. Priotto (2008) BMJ 336(7646):705-8 +PMID: 18321960 [PubMed]

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