Note: This document contains side effect information about melphalan. Some dosage forms listed on this page may not apply to the brand name Hepzato.

Applies to melphalan: oral tablet. Other dosage forms:

• powder for solution

Along with their needed effects, medicines like melphalan (the active ingredient contained in Hepzato) 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.

Serious side effects of Hepzato

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

Rare

• 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

Other side effects of Hepzato

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

• Nausea and vomiting

For Healthcare Professionals

Applies to melphalan: intravenous powder for injection, oral tablet.

Hematologic

thrombocytopenia, anemia)

Rare (less than 0.1%): Hemolytic anemia^[Ref]

Oncologic

Frequency not reported: Secondary malignancies (e.g., acute nonlymphocytic leukemia, myeloproliferative syndrome, carcinoma^[Ref]

Respiratory

Rare (less than 0.1%): Interstitial pneumonitis, pulmonary fibrosis

Frequency not reported: Bronchospasm, dyspnea^[Ref]

Dermatologic

Very common (10% or more): Alopecia (at high dose)

Common (1% to 10%): Alopecia (at conventional dose)

Rare (less than 0.1%): Maculopapular rash, pruritus

Frequency not reported: Skin hypersensitivity, skin necrosis (rarely requiring skin grafting)^[Ref]

Cardiovascular

Frequency not reported: Tachycardia, hypotension, vasculitis^[Ref]

Gastrointestinal

Very common (10% or more): Nausea, vomiting, diarrhea, stomatitis (at high dose)

Rare (less than 0.1%): Stomatitis (at conventional dose)^[Ref]

Hepatic

Rare (less than 0.1%): Abnormal liver function tests to clinical manifestations such as hepatitis and jaundice; veno-occlusive disease following high dose treatment^[Ref]

Hypersensitivity

Uncommon (0.1% to 1%): Allergic reactions (e.g., urticaria, edema, skin rashes, anaphylactic shock)^[Ref]

Other

Very common (10% or more): Warmth, tingling

Frequency not reported: Chromosome damage^[Ref]

Renal

Common (1% to 10%): Temporary significant elevation of the blood urea has been seen in the early stages of therapy with this drug in myeloma patients with renal damage^[Ref]

Local

Frequency not reported: Skin ulceration at injection site^[Ref]

Musculoskeletal

Very common (10% or more): Muscle atrophy, muscle fibrosis, myalgia, blood creatine phosphokinase increased

Common (1% to 10%): Compartment syndrome

Frequency not reported: Muscle necrosis, rhabdomyolysis^[Ref]