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a cytotoxic alkylating agent derived from nitrogen mustard, used as an antineoplastic agent, primarily for treatment of multiple myeloma; administered orally or intravenously.
Miller-Keane Encyclopedia and Dictionary of Medicine, Nursing, and Allied Health, Seventh Edition. © 2003 by Saunders, an imprint of Elsevier, Inc. All rights reserved.

melphalan (L-PAM, L-phenylalanine mustard, L-sarcolysin)


melphalan hydrochloride


Pharmacologic class: Alkylator

Therapeutic class: Antineoplastic

Pregnancy risk category D

FDA Box Warning

• Give under supervision of physician experienced in cancer chemotherapy, in facility with adequate diagnostic and treatment resources.

• Drug may cause severe bone marrow suppression leading to infection or bleeding. I.V. use causes greater myelosuppression than oral use, and also may lead to hypersensitivity reactions (including anaphylaxis).

• Drug may cause leukemia and is potentially mutagenic.


Forms cross-links between strands of cellular DNA, disrupting DNA and RNA transcription and causing cell death


Powder for injection (melphalan hydrochloride): 50 mg

Tablets: 2 mg

Indications and dosages

Multiple myeloma

Adults: Initially, 6 mg P.O. daily for 2 to 3 weeks, then discontinue drug for up to 4 weeks or until white blood cell (WBC) and platelet counts increase; then give maintenance dosage of 2 or 10 mg/day for 7 to 10 days, then withhold until WBC recovery followed by 2 mg/day maintenance or 0.15 mg/kg/day P.O. for 7 days or 0.25 mg/kg for 4 days, repeated q 4 to 6 weeks. For those who can't tolerate oral therapy, 16 mg/m2 by I.V. infusion over 15 to 20 minutes at 2-week intervals for four doses (usually with prednisone); I.V. dose can be repeated q 4 weeks after recovery from toxicity.

Nonresectable advanced ovarian cancer

Adults: 0.2 mg/kg/day P.O. for 5 days q 4 to 5 weeks

Dosage adjustment

• Renal impairment


• Hypersensitivity to drug

• Patients whose disease has shown previous drug resistance


Use cautiously in:

• bone marrow depression, infection, renal disease

• previous radiation therapy

• patients with childbearing potential

• pregnant or breastfeeding patients

• children (safety and efficacy not established).


• Before starting therapy, obtain CBC with white cell differential and platelet count. Repeat periodically before each course.

• For I.V. use, reconstitute by rapidly injecting 10 ml of supplied diluent into vial with lyophilized powder. Shake until solution is clear (yields a concentration of 5 mg/ml).

• Dilute desired dosage in 0.9% sodium chloride injection to a concentration no greater than 0.45 mg/ml. Administer over 15 minutes, being sure to give entire dose within 60 minutes of reconstitution.

Minimize time between reconstitution, dilution, and administration, because solution is unstable.

Adverse reactions

CV: hypotension, tachycardia, vasculitis

GI: nausea, vomiting, diarrhea, oral ulcers, stomatitis

GU: hyperuricemia, amenorrhea, gonadal suppression, infertility

Hematologic: anemia, purpura, bone marrow depression, leukopenia, thrombocytopenia

Hepatic: hepatotoxicity

Metabolic: hyperuricemia

Respiratory: dyspnea, interstitial pneumonitis, bronchospasm, fibrosis

Skin: rash, urticaria, pruritus, alopecia, sweating

Other: edema, extravasation at I.V. site, allergic reactions including anaphylaxis


Drug-drug. Carmustine: increased pulmonary toxicity

Cimetidine: decreased GI absorption of melphalan

Cisplatin: increased risk of renal dysfunction, decreased melphalan clearance

Cyclosporine: increased risk of nephrotoxicity, severe renal failure

Interferon alfa: decreased melphalan blood level

Live-virus vaccines: decreased antibody response to vaccine

Myelosuppressants: additive toxicity

Nalidixic acid: increased risk of severe hemorrhagic necrotic enterocolitis (in children)

Drug-diagnostic tests. Hemoglobin, platelets, red blood cells, WBCs: decreased values

Nitrogenous compounds: increased levels

Drug-food. Any food: decreased absorption of oral melphalan

Patient monitoring

Monitor patient for thrombocytopenia and leukopenia. If platelet count exceeds 100,000/mm3 or WBC count is below 3,000/mm3, discontinue drug until peripheral blood counts recover.

Watch closely for indications of bone marrow depression, including infection, anemia, and bleeding.

After multiple courses, watch for acute hypersensitivity reaction. If it occurs, discontinue drug and administer volume expanders, corticosteroids, or antihistamines, as prescribed.

• Watch for signs and symptoms of GI or pulmonary toxicity.

• Evaluate renal and hepatic function.

Patient teaching

• Tell patient to take oral tablets without food, because food may decrease drug absorption.

• Instruct patient to take entire daily oral dose at one time on empty stomach.

Advise patient to immediately report unusual bleeding or bruising, fever, chills, sore throat, shortness of breath, yellowing of skin or eyes, persistent cough, flank or stomach pain, joint pain, black tarry stools, rash, or unusual lumps or masses.

• Tell patient to consult prescriber before using over-the-counter medications.

• Advise patient to use reliable contraception.

• Caution patient to avoid breastfeeding.

• As appropriate, review all other significant and life-threatening adverse reactions and interactions, especially those related to the drugs, tests, and foods mentioned above.

McGraw-Hill Nurse's Drug Handbook, 7th Ed. Copyright © 2013 by The McGraw-Hill Companies, Inc. All rights reserved


An alkylating nitrogen mustard used to treat ovarian cancer, myeloma, and occasionally melanoma.

Adverse effects
Nausea, vomiting, oral ulcers, myelosuppression, mutagenesis, allergic reactions.
Segen's Medical Dictionary. © 2012 Farlex, Inc. All rights reserved.


Alkeran® Oncology An oral alkylating anticancer nitrogen mustard agent used to treat  ovarian CA and myeloproliferative disease Adverse effects Myelosuppression, mutagenesis
McGraw-Hill Concise Dictionary of Modern Medicine. © 2002 by The McGraw-Hill Companies, Inc.


A drug used in the treatment of POLYCYTHAEMIA VERA, chronic LEUKAEMIA and MYELOMATOSIS. A brand name is Alkeran.
Collins Dictionary of Medicine © Robert M. Youngson 2004, 2005
References in periodicals archive ?
This study's aims are to explore the efficacy of intravitreal and subconjunctival melphalan in controlling intraocular retinoblastoma and its mechanism of action.
Dose-intensive melphalan with blood stem-cell support for the treatment ofAL (amyloid lightchain) amyloidosis: survival and responses in 25 patients.
Combination chemotherapy versus melphalan plus prednisone as treatment for multiple myeloma: an overview of 6,633 patients from 27 randomized trials.
Notably, renal failure has been viewed as a poor prognostic factor for survival following high-dose melphalan, so ASCT protocols often exclude patients with renal impairment.
Twenty-four hours after the melphalan dose the autologous stem cells are reinfused.
Thal/Dex was associated with an increased response rate before, but not after, therapy with high-dose melphalan and ASCT.
Patients conditioned with melphalan generally achieved neutrophil engraftment by day +11 (range day +10-13) and platelet engraftment by day +20 (range 15-25).
Mrs Doswell, who had bone cancer, visited her consultant, Dr Aboobucker Abdul-Cader at St Cross Hospital in Rugby on June 26, 1997 and was prescribed a course of Melphalan.
Treatment: Ninety-one patients were allocated melphalan and-prednisone (MP) (melphalan 9 mg/[m.sup.2] and prednisone 60 mg/[m.sup.2] orally from days 1 to 4).
and its affiliates for which CASI has exclusive rights to the greater China market, consisting of EVOMELA (Melphalan for Injection), ZEVALIN (Ibritumomab Tiuxetan) and MARQIBO (Vincristine Sulfate Liposome Injection) and (iv) a portfolio of FDA-approved and pending abbreviated new drug applications (ANDAs), including entecavir and tenofovir disoproxil fumarate (TDF) indicated for the treatment of hepatitis B virus.
Melphalan hydrochloride for injection (EVOMELA) received market approval by the China National Medical Products Administration for use as high-dose conditioning treatment prior to hematopoietic progenitor cell transplant in patients with multiple myeloma, and as a palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.