II. Indications

  1. Leishmaniasis
  2. Ameba Infection (off-label)
    1. Naegleria fowleri
    2. Balamuthia mandrillaris
    3. Acanthamoeba

III. Contraindications

  1. Pregnancy
  2. Lactation
  3. Age under 12 years old
  4. Sjogren-Larsson Syndrome

IV. Mechanism

  1. Alkyl-phosphocholine affects cellular membranes, interferes with cell Mitosis and acts as an immunomodulator

V. Medications

  1. Capsules 50 mg in Blister packs

VI. Dosing: Adults and Children (age >12 years)

  1. General
    1. Take with food
  2. Leishmaniasis (Visceral, cutaneous or mucosal)
    1. Weight 30 to 44 kg: Miltefosine 50 mg orally twice daily for 28 days
    2. Weight >=45 kg: Miltefosine 50 mg orally three times daily for 28 days
  3. Ameba infections (Naegleria fowleri, Balamuthia mandrillaris, Acanthamoeba)
    1. See CDC and other references for dosing

VII. Adverse Effects

  1. Serum Creatinine Increase
    1. Monitor Serum Creatinine weekly for 4 weeks
  2. Increased Liver Function Tests
    1. Monitor transaminases and Serum Bilirubin
  3. Decreased Platelet Count (in Visceral Leishmaniasis)
    1. Monitor Platelet Count
  4. Common
    1. Vomiting and Diarrhea

VIII. Safety

  1. Avoid in Lactation for 5 months after last use
  2. Pregnancy Category D (avoid)
    1. Use reliable Contraception while taking Miltefosine and for 5 months after use

IX. Drug Interactions

  1. Oral Contraceptives
    1. Decreased absorption if Vomiting and Diarrhea occur

XI. References

  1. Hamilton (2020) Tarascon Pocket Pharmacopoeia

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Related Studies

Ontology: miltefosine (C0068006)

Definition (NCI) An orally- and topically-active alkyl-phosphocholine compound with potential antineoplastic activity. Miltefosine targets cellular membranes, modulating cell membrane permeability, membrane lipid composition, phospholipid metabolism, and mitogenic signal transduction, resulting in cell differentiation and inhibition of cell growth. This agent also inhibits the anti-apoptotic mitogen-activated protein kinase (MAPK) pathway and modulates the balance between the MAPK and pro-apoptotic stress-activated protein kinase (SAPK/JNK) pathways, thereby inducing apoptosis. As an immunomodulator, miltefosine stimulates T-cells, macrophages and the expression of interleukin 3 (IL-3), granulocyte-macrophage colony stimulating factor (GM-CSF), and interferon gamma (INF-gamma). (NCI04)
Definition (PDQ) An orally- and topically-active alkyl-phosphocholine compound with potential antineoplastic activity. Miltefosine targets cellular membranes, modulating cell membrane permeability, membrane lipid composition, phospholipid metabolism, and mitogenic signal transduction, resulting in cell differentiation and inhibition of cell growth. This agent also inhibits the anti-apoptotic mitogen-activated protein kinase (MAPK) pathway and modulates the balance between the MAPK and pro-apoptotic stress-activated protein kinase (SAPK/JNK) pathways, thereby inducing apoptosis. As an immunomodulator, miltefosine stimulates T-cells, macrophages and the expression of interleukin 3 (IL-3), granulocyte-macrophage colony stimulating factor (GM-CSF), and interferon gamma (INF-gamma). Check for "http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?id=41212&idtype=1" active clinical trials or "http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?id=41212&idtype=1&closed=1" closed clinical trials using this agent. ("http://nciterms.nci.nih.gov:80/NCIBrowser/ConceptReport.jsp?dictionary=NCI_Thesaurus&code=C1170" NCI Thesaurus)
Concepts Pharmacologic Substance (T121) , Organophosphorus Compound (T115)
MSH C039128
English HDPC, hexadecylphosphocholine, miltefosine, n-hexadecylphosphorylcholine, Ethanaminium, 2-(((hexadecyloxy)hydroxyphosphinyl)oxy)-N,N,N-trimethyl-, hydroxide, innner salt, 2-[[(Hexadecycloxy)hydroxyphosphinyl]oxy]-N,N,N-trimethylethanaminium Inner Salt, Hexadecyl 2-(N,N,N-trimethylamino)ethyl Phosphate, n-Hexadecylphosphorylcholine, Miltefosinum, Choline Phosphate Hexadecyl Ester, Hydroxide, Inner Salt, Miltefosina, Miltefosin, MILTEFOSINE, Hexadecylphosphocholine, Miltefosine