II. Indications

  1. FDA Approved
    1. Multiple Myeloma
    2. Epithelial Ovarian Cancer (non-resectable)
  2. Off-Label Use
    1. Amyloidosis (light chain)
    2. Ewing Sarcoma (children, for Stem Cell Transplant)
    3. Neuroblastoma (children, for Stem Cell Transplant)
    4. Non-Hodgkin Lymphoma (for Stem Cell Transplant)
    5. Hodgkin Lymphoma (refractory)
    6. Testicular Cancer

III. Mechanism

  1. See Alkylating Agent
  2. Antineoplastic, Phenylalanine derivative of nitrogen mustard
    1. As with other Alkylating Agents (and nitrogen mustards), cross-links DNA
    2. Melphalan alkylates DNA at the N7 position of guanine
    3. Melphalan was first synthesized in 1953, and has been used in Multiple Myeloma since that time

IV. Medications

  1. Alkeran IV: 50 mg/vial
  2. Alkeran Tabs: 2 mg
  3. Evomela Lypholized IV: 50 mg/vial (lypholized powder for reconstitution)
    1. Indicated for high dose regimens in Stem Cell Transplant

V. Dosing

  1. See other references for dosing protocols specific to indications

VI. Adverse Effects

  1. Myelosuppression (severe)
    1. Intravenous forms result in greater Bone Marrow suppression
    2. Monitor Complete Blood Count
  2. Hypersensitivity Reactions (2% with IV preparation)
  3. Impaired future fertility
  4. Alopecia
  5. Carcinogenic
    1. Associated with secondary Leukemia risk

VII. Safety

  1. Avoid in Lactation
  2. Avoid in Pregnancy (all trimesters)
    1. Use reliable Contraception

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Ontology: Melphalan (C0025241)

Definition (NCI_NCI-GLOSS) A drug that is used to treat multiple myeloma and ovarian epithelial cancer and is being studied in the treatment of other types of cancer. It belongs to the family of drugs called alkylating agents.
Definition (NCI) A phenylalanine derivative of nitrogen mustard with antineoplastic activity. Melphalan alkylates DNA at the N7 position of guanine and induces DNA inter-strand cross-linkages, resulting in the inhibition of DNA and RNA synthesis and cytotoxicity against both dividing and non-dividing tumor cells.
Definition (MSH) An alkylating nitrogen mustard that is used as an antineoplastic in the form of the levo isomer - MELPHALAN, the racemic mixture - MERPHALAN, and the dextro isomer - MEDPHALAN; toxic to bone marrow, but little vesicant action; potential carcinogen.
Definition (CSP) 4-bis-(2-chloroethyl)amino-L-phenylalanine; a bifunctional alkylating agent used as a DNA strand crosslinker and antineoplastic.
Definition (PDQ) An orally available phenylalanine derivative of nitrogen mustard with antineoplastic activity. Melphalan alkylates DNA at the N7 position of guanine and induces DNA inter-strand cross-linkages, resulting in the inhibition of DNA and RNA synthesis and cytotoxicity against both dividing and non-dividing tumor cells. Check for "http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?id=42973&idtype=1" active clinical trials or "http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?id=42973&idtype=1&closed=1" closed clinical trials using this agent. ("http://nciterms.nci.nih.gov:80/NCIBrowser/ConceptReport.jsp?dictionary=NCI_Thesaurus&code=C633" NCI Thesaurus)
Concepts Pharmacologic Substance (T121) , Amino Acid, Peptide, or Protein (T116)
MSH D008558
SnomedCT 387297002, 544002
English L-PAM, Mustard, Phenylalanine, Phenylalanine Mustard, melphalan, 4-(Bis(2-chloroethyl)amino)phenylalanine, L-Phenylalanine, 4-(bis(2-chloroethyl)amino)-, 4-[bis(2-chloroethyl)amino]-L-phenylalanine, p-di(chloroethyl)amino-L-phenylalanine, Phenylalanine Nitrogen Mustard, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, alanine nitrogen mustard, Melfalan, sarcolysin, Melphalan, MELPHALAN, Melphalan [Chemical/Ingredient], l-phenylalanine mustard, melfalan, l-pam, Phenylalanine nitrogen mustard, Alanine Nitrogen Mustard, L-Phenylalanine mustard, Phenylalanine mustard, L-sarcolysin, Melphalan (product), Melphalan (substance), L-Sarcolysin, L-phenylalanine mustard, Sarcolysin, L-Phenylalanine Mustard, phenylalanine nitrogen mustard
Swedish Melfalan
Czech melfalan, melphalan
Finnish Melfalaani
Russian FENILANIN-IPRIT, SARKOLIZIN, MELFALAN, MERFALAN, MEDFALAN, МЕДФАЛАН, МЕЛФАЛАН, МЕРФАЛАН, САРКОЛИЗИН, ФЕНИЛАНИН-ИПРИТ
Japanese メルファラン, メドファラン, サルコリシン, フェニルアラニンマスタード
French 4-[Bis(2-chloroéthyl)amino]-L-phénylalanine, Melphalan
Croatian MELFALAN
Spanish melfalano, melfalán (producto), melfalano (producto), L-sarcolisina, melfalano (sustancia), melfalán (sustancia), melfalán, mostaza de fenilalanina, Mostaza de Fenilalanina, Melfalán
Polish Melfalan, Alkeran, Sarkolizyna
German Melphalan, Phenylalanin-Stickstoff-Lost
Italian Melfalan
Portuguese Fenilalanina Mostarda, Melfalan