Complete information including dosage, side effects, interactions and pregnancy & breast feeding warnings for patient and professional use.

Overview and uses

Melphalan is an alkylating agent used for the treatment of cancer.

Indication (uses)

  • Tablets
    • For the palliative treatment of multiple myeloma.
    • For the palliation of non-resectable epithelial carcinoma of the ovary.
  • Injection (vial for reconstitution)
    • Melphalan injection is used as a high dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma.
    • For the palliative treatment of patients with multiple myeloma for whom oral therapy is not recommended.

Pharmacological classification

Alkylating agent used for the treatment of cancer.

Brand names

Alkeran, Evomela

Dosage form

  • Supplied as white, film coated, biconvex tablets containing 2 mg and 5 mg Melphalan in amber glass bottles with child resistant closures.
  • Supplied as vial for reconstitution containing 50 mg Melphalan.

Mechanism of action

Alkylating agents produce highly reactive carbonium ion intermediates which transfer alkyl groups to cellular macromolecules by forming covalent bonds. These intermediates may react with carboxyl, hydroxyl, amino, sulfhydryl and phosphate groups of bio macromolecules. Alkylation results in cross linking/abnormal base pairing/scission of DNA strand. Cross linking of nucleic acids with proteins can also take place.


  • Dosage (tablets)
    • Multiple myeloma
      • 6 mg (3 tablets) once a day.
      • The drug should be discontinued for up to 4 weeks after 2 – 3 weeks of treatment. During this time, the blood count should be carefully followed.
      • When the white blood cell and platelet counts are rising, a maintenance dose of 2 mg daily may be instituted.
      • Because of the patient to patient variation in melphalan plasma levels following oral administration of the drug, several investigators have recommended that the dosage of melphalan be cautiously escalated until some myelosuppression is observed in order to assure that potentially therapeutic levels of the drug have been reached.
    • Epithelial ovarian cancer
      • 2 mg/kg daily for 5 days as a single course.
      • Courses are repeated every four to five weeks depending upon hematologic tolerance.
    • Dosage (injection – vial for reconstitution)
      • For conditioning treatment
        • 100 mg/m2/day administered over 30 minutes by intravenous infusion for 2 consecutive days prior to autologous stem cell transplantation.
        • For patients who weigh more than 130% of their ideal body weight, body surface area should be calculated based on adjusted ideal body weight.
      • For palliative treatment
        • 16 mg/m2 administered as a single intravenous infusion over 15-20 minutes at 2-week intervals for 4 doses.
        • The next dose should be administered after adequate recovery from toxicity, at 4-week intervals.

Hepatic dose adjustment

  • Data not available.

Renal dose adjustments

  • No changes in the dose are required for the conditioning treatment of patients with renal impairment.
  • For patients on palliative treatment, consider a 50% reduction in IV dose of Melphalan in patients with BUN levels greater than or equal to 30 mg/dL.

Pediatric patients

  • Data not available.

Geriatric dose adjustments

  • Caution should be exercised while administering Melphalan in elderly patients since an increase in age is related to reduced hepatic, renal and/or cardiac functions as well as presence of concomitant disease or treatment of concomitant disease.
  • Dose selection for elderly patient should be cautious, starting from lower end of the dose range and gradually increasing the dose based on therapeutic response and side effects.


  • Symptoms of over dose
    • Severe nausea and vomiting
    • Decreased consciousness
    • Convulsions
    • Muscular paralysis
    • Cholinomimetic effects
    • Stomatitis
    • Severe mucositis
    • Colitis
    • Diarrhea
    • Hemorrhage of gastro intestinal tract
    • Elevation in liver enzyme
    • Veno-occlusive disease
    • Hyponatremia
    • Nephrotoxicity
    • Adult respiratory distress syndrome
  • Treatment
    • General supportive measures
    • Appropriate blood transfusion
    • Appropriate antibiotics
    • Melphalan is not be removed by hemodialysis.

Side effects or adverse effects

  • Local side effects
    • Thrombophlebitis and thrombosis at the site of injection.
  • Hypersensitivity reactions
    • Anaphylaxis has been reported with Mechlorethamine use.
    • Pain in chest or abdomen, skin rash, difficulty in breathing, rapid breathing, shortness of breath, dizziness, fainting, low blood pressure are some of the symptoms.
    • Consult your doctor as soon as possible.
  • Other systemic side effects
    • Jaundice
    • Vertigo
    • Tinnitus
    • Diminished hearing
    • Occurrence/precipitation of hemolytic anemia
  • Bone marrow depression
    • Bone marrow depression is the most serious side effect/toxicity. It is also a limiting factor for the dose. Infections and bleeding are the common complications.
    • Bone marrow depression results in granulocytopenia, agranulocytosis, thrombocytopenia and aplastic anemia.
    • Consult your doctor as soon as possible if you observe any of the below mentioned symptoms:
      • Sore throat
      • Chills
      • Sudden fever
      • Weakness in limbs
      • Mouth ulcers
      • Bleeding gums
      • Sore mouth
      • Fast heart rate
      • Low blood pressure
      • Sore gums
      • Skin abscesses
      • Rapid breathing
      • Rapid progression of infection of any organ
      • Rapid progression of Septicemia
    • Gastrointestinal side effects
      • Nausea and vomiting are commonly observed with anti-cancer medicines.
      • Mucositis – painful inflammation and ulceration of mucus membrane lining the digestive tract.
      • Diarrhea, shredding of mucosa and haemorrhage due to decreased rate of renewal of gastrointestinal mucosa lining.
    • Skin
      • Alopecia or hair loss.
      • Dermatitis
      • Pigmentation of the skin and changes in the nail colour.
    • Oral side effects
      • Breaches during chewing.
      • Depression of immunity due to reduction in number of WBCs largely increases the risk of oral infections.
      • Bleeding gums
      • Rapid progression of dental caries.
    • Gonadal side effects
      • Infertility due to Oligozoospermia in males.
      • Impotence in males.
      • Loss or reduces sex drive.
      • Inhibition of ovulation in females
      • Amenorrhea in females.
      • Damage to germinal cells may lead to mutagenesis.
    • Lymphoreticular tissue
      • Lymphocytopenia (reduced number of lymphocytes)
      • Inhibition of lymphocyte function.
      • Suppression of immunity due to bone marrow depression and reduced lymphocyte function highly increases the risk of opportunistic infections like:
        • Candida and other infections causing deep mycosis.
        • Cytomegalo virus
        • Herpes zoster
        • Pneumocystis jiroveci
        • Toxoplasma
      • Pulmonary toxicity
        • Pneumonitis
        • Pulmonary fibrosis
        • Pulmonary veno-occlusive disease
      • Impairment of wound healing
        • Cyclophosphamide may interfere with normal wound healing.
      • Hyponatremia
        • Hyponatremia associated with increased total body water, acute water intoxication and a syndrome resembling SIADH.
      • Secondary malignancies
        • Urinary tract cancer
        • Myelodysplasia
        • Acute leukemias
        • Lymphomas
        • Thyroid cancer
        • Sarcomas
      • Cardiac side effects
        • Congestive heart failure
        • Myocarditis
        • Myopericarditis
        • Pericardial effusion including cardiac tamponade
        • Supraventricular arrhythmias
        • Ventricular arrythmias
      • Fetal side effects
        • Use of anti-cancer medicines in pregnant women may cause fetal damage leading to:
          • Fetal death
          • Abortion
          • Teratogenesis
        • Renal side effects
          • Renal failure, Gout and Urate stones may develop in urinary tract.
          • Hemorrhagic cystitis, pyelitis, ureteritis and hematuria.
        • Hyperuricemia
          • An elevated level of uric acid is common in leukemia and bulky lymphomas.

Please note: The side effects mentioned above are from the reported cases. However, side effects are not limited to above mentioned conditions and symptoms. Consult your doctor as soon as possible if you feel or observe any symptoms that you relate to your treatment.

Warnings & Precautions

  • Contra-indicated in patients with known hypersensitivity to Melphalan.

Pregnancy & breast feeding warnings

  • Pregnancy
  • Category D, which means there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use of the drug in pregnant women despite potential risks.
  • Nursing:
  • It is not known whether Melphalan is excreted in human milk.
  • Based on the potential for serious side effects, a decision should be made whether to continue breast feeding or to continue the treatment with Melphalan.
  • Melphalan should not be given to nursing mothers.


Drug – drug interactions

  • The development of severe renal impairment has been reported in patients treated with a single dose of intravenous Melphalan 140-250 mg/m2 followed by standard oral doses of cyclosporine.
  • When nalidixic acid and IV Melphalan are given simultaneously, the incidence of severe hemorrhagic necrotic enterocolitis has been reported to increase in pediatric patients.
  • Concomitant use of Melphalan with following drugs/medicines should be strictly avoided.
    • ACE inhibitors can cause leukopenia.
    • Thiazide diuretics
    • Zidovudine
    • Paclitaxel causes increased hematotoxicity.
    • Leflunomide as it increases the risk of infections.
    • Adalimumab as it increases the risk of infections.
    • Teriflunomide as it increases the risk of infections.
    • Certolizumab as it increases the risk of infections.
    • Clozapine as it may lower the WBC count.
    • Deferiprone as it may lower the WBC count.
    • Etanercept as it increases the risk of infections.
    • Fingolimod as it increases the risk of infections.
    • Golimumab as it increases the risk of infections.
    • Infliximab as it increases the risk of infections.
    • Talimogene as it increases the risk of herpes infections.
    • Nalidixic acid as the combination may cause unusual bruising or bleeding, nausea, stomach pain, unusual weakness, low fever and loss of appetite.
    • Natalizumab as it increases the risk of infections.
    • Tofacitinib as it increases the risk of infections.

Click here to go to Melphalan forums.


  • “Product information. Alkeran (Melphalan).” GlaxoSmithKline, NC 27709.
  • “Product information. Evomela (Melphalan).” Spectrum pharmaceuticals, Irvine, CA.
  • NIOSH Alert: Preventing Occupational Exposures to Antineoplastic and Other Hazardous Drugs in Healthcare Settings. U.S. Department of Health and Human Services, Public Health Service. Centers for Disease Control and Prevention, National Institute for Occupational Safety and Health, DHHS (NIOSH) Publication No. 2004-165.
  • OSHA Technical Manual, TED 1-0.15A, Section VI: Chapter 2. Controlling Occupational Exposure to Hazardous Drugs. OSHA, 1999.
  • American Society of Health-System Pharmacists. (2006) ASHP Guidelines on Handling Hazardous Drugs. Am J Health-Syst Pharm. 2006;63:1172-1193.
  • Polovich M, White JM, Kelleher LO (eds.) 2005. Chemotherapy and Biotherapy Guidelines and Recommendations for Practice. (2nd ed.) Pittsburgh, PA: Oncology Nursing Society.

melphalan wikipedia page

By |2018-12-29T07:00:46+00:00May 2nd, 2017|medicines|Comments Off on melphalan

About the Author:

B. Pharm (K.L.E. society's S.V.V. Patil College of Pharmacy, Bengaluru) M. Pharm (Maharishi Arvind Institute of Pharmacy, Jaipur)


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