II. Indications
- Leishmaniasis
- Ameba Infection (off-label)
- Naegleria fowleri
- Balamuthia mandrillaris
- Acanthamoeba
III. Contraindications
- Pregnancy
- Lactation
- Age under 12 years old
- Sjogren-Larsson Syndrome
IV. Mechanism
- Alkyl-phosphocholine affects cellular membranes, interferes with cell Mitosis and acts as an immunomodulator
V. Medications
- Capsules 50 mg in Blister packs
VI. Dosing: Adults and Children (age >12 years)
-
General
- Take with food
-
Leishmaniasis (Visceral, cutaneous or mucosal)
- Weight 30 to 44 kg: Miltefosine 50 mg orally twice daily for 28 days
- Weight >=45 kg: Miltefosine 50 mg orally three times daily for 28 days
- Ameba infections (Naegleria fowleri, Balamuthia mandrillaris, Acanthamoeba)
- See CDC and other references for dosing
VII. Adverse Effects
-
Serum Creatinine Increase
- Monitor Serum Creatinine weekly for 4 weeks
- Increased Liver Function Tests
- Monitor transaminases and Serum Bilirubin
- Decreased Platelet Count (in Visceral Leishmaniasis)
- Monitor Platelet Count
- Common
VIII. Safety
- Avoid in Lactation for 5 months after last use
- Pregnancy Category D (avoid)
- Use reliable Contraception while taking Miltefosine and for 5 months after use
IX. Drug Interactions
X. Resources
XI. References
- Hamilton (2020) Tarascon Pocket Pharmacopoeia
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Related Studies
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 |