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Melphalan Side Effects

For the Consumer

Applies to melphalan: oral tablet

Along with their needed effects, medicines like melphalan can sometimes cause unwanted effects such as blood problems and other side effects. These and others are described below. Also, because of the way these medicines act on the body, there is a chance that they might cause other unwanted effects that may not occur until months or years after the medicine is used. These delayed effects may include certain types of cancer, such as leukemia. Discuss these possible effects with your doctor.

Along with its needed effects, melphalan may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking melphalan:

Less common
  • Black, tarry stools
  • blood in urine or stools
  • cough or hoarseness, accompanied by fever or chills
  • fast or irregular heart beat
  • fever or chills
  • lower back or side pain, accompanied by fever or chills
  • painful or difficult urination, accompanied by fever or chills
  • pinpoint red spots on skin
  • shortness of breath
  • skin rash or itching (sudden)
  • troubled breathing
  • unusual bleeding or bruising
  • Diarrhea
  • difficulty swallowing
  • joint pain
  • redness and/or soreness in arm or leg
  • sores in the mouth and on the lips
  • swelling of the feet or lower legs

Some side effects of melphalan may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

Less common

For Healthcare Professionals

Applies to melphalan: intravenous powder for injection, oral tablet


Hematologic side effects including bone marrow suppression and anemia have been reported. Bone marrow suppression is the most common and the most significant toxicity associated with melphalan in most patients. Hemolytic anemia has also been reported.[Ref]

Bone marrow suppression is usually reversible if melphalan is withdrawn early enough.[Ref]


The risk of leukemogenesis increases with chronicity of treatment and with cumulative dose.[Ref]

Oncologic side effects including secondary malignancies such as acute nonlymphocytic leukemia, myeloproliferative syndrome, and carcinoma have been reported. Melphalan has been reported to cause chromatid or chromosome damage in humans.[Ref]


Genitourinary side effects including the suppression of ovarian function in premenopausal women and both reversible and irreversible testicular suppression have been reported.[Ref]

The suppression of ovarian function in premenopausal women has been reported to result in amenorrhea in a significant number of patients.[Ref]


Fatal outcomes associated with pulmonary fibrosis have been reported.[Ref]

Respiratory side effects including pulmonary fibrosis and interstitial pneumonitis have been reported.[Ref]


Dermatologic side effects including , maculopapular rashes, skin rashes, alopecia, and skin hypersensitivity have been reported.[Ref]


Cardiovascular side effects including cardiac arrest and vasculitis have been reported.[Ref]


Gastrointestinal side effects including nausea and vomiting, diarrhea, and oral ulceration have been reported infrequently.[Ref]


Hepatic side effects including toxicity have been reported rarely.[Ref]


Hypersensitivity side effects including anaphylaxis have been reported rarely.[Ref]

Hypersensitivity reactions have been reported after multiple courses of treatment and have recurred in patients who previously experienced a hypersensitivity reaction.[Ref]


Endocrine side effects including two cases of syndrome of inappropriate antidiuretic hormone secretion have been reported following high-dose intravenous melphalan.[Ref]


Other side effects including a sensation of warmth and/or tingling have been reported.


Renal side effects including temporary significant elevation of the blood urea have been seen in the early stages of therapy in patients with renal damage.


1. "Product Information. Alkeran Tablets (melphalan)." Glaxo Wellcome, Research Triangle Pk, NC.

2. Greene MH, Harris EL, Gershenson DM, Malkasian GD Jr, Melton LJ 3d, Dembo AJ, Bennett JM, Moloney WC, Boice JD Jr, Melton LJ "Melphalan may be a more potent leukemogen than cyclophosphamide." Ann Intern Med 105 (1986): 360-7

3. Curtis RE, Boice JD Jr, Stovall M, Bernstein L, Greenberg RS, Flannery JT, Schwartz AG, Weyer P, Moloney WC, Hoover RN "Risk of leukemia after chemotherapy and radiation treatment for breast cancer." N Engl J Med 326 (1992): 1745-51

4. Kaldor JM, Day NE, Pettersson F, Clarke EA, Pedersen D, Mehnert W, Bell J, Host H, Prior P, Karjalainen S, et al, Hst H "Leukemia following chemotherapy for ovarian cancer." N Engl J Med 322 (1990): 1-6

5. Akasheh MS, Freytes CO, Vesole DH "Melphalan-associated pulmonary toxicity following high-dose therapy with autologous hematopoietic stem cell transplantation." Bone Marrow Transplant 26 (2000): 1107-9

6. Greenbaum-Lefkoe B, Rosenstock JG, Belasco JB, Rohrbaugh TM, Meadows AT "Syndrome of inappropriate antidiuretic hormone secretion. A complication of high-dose intravenous melphalan." Cancer 55 (1985): 44-6

Some side effects of melphalan may not be reported. Always consult your doctor or healthcare specialist for medical advice. You may also report side effects to the FDA.

Disclaimer: Every effort has been made to ensure that the information provided is accurate, up-to-date and complete, but no guarantee is made to that effect. In addition, the drug information contained herein may be time sensitive and should not be utilized as a reference resource beyond the date hereof. This material does not endorse drugs, diagnose patients, or recommend therapy. This information is a reference resource designed as supplement to, and not a substitute for, the expertise, skill , knowledge, and judgement of healthcare practitioners in patient care. The absence of a warning for a given drug or combination thereof in no way should be construed to indicate safety, effectiveness, or appropriateness for any given patient. does not assume any responsibility for any aspect of healthcare administered with the aid of materials provided. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the substances you are taking, check with your doctor, nurse, or pharmacist.