Melphalan

What this treatment is and how it works

An alkylating chemotherapy medicine that damages cancer-cell DNA (the genetic instructions inside cells).

When it may be used

Melphalan may be used to treat myeloma, including high-dose treatment before a planned stem-cell transplant. In myeloma, the aim is usually to achieve a deeper response and keep the disease controlled for longer. Melphalan is also part of some specialist transplant-conditioning regimens for other cancers; the treatment goal depends on the diagnosis and complete transplant plan.

A listed use is not a claim that every brand is licensed for that use in every country. Your oncologist will explain why this treatment is appropriate for you.

Before treatment

Before starting, tell the team about previous cancer treatment, allergies, infection symptoms, heart, lung, kidney or liver disease, hearing or nerve problems, diabetes and all medicines you take. Discuss pregnancy, contraception and fertility. Ask for a written treatment calendar, supportive-medicine instructions and a 24-hour oncology contact number. Baseline blood tests usually include a full blood count and kidney and liver function; additional tests depend on the medicine and your health. Before chemotherapy that reaches the whole body, the team should check for current or past hepatitis B infection. If a test is positive, extra monitoring or antiviral treatment may be needed to prevent the virus becoming active again. The team will coordinate testing and treatment; do not delay urgent cancer treatment yourself.

How treatment is given and the schedule

As tablets or into a vein. High-dose intravenous melphalan may be used before a stem-cell transplant; this is a separate intensive treatment setting.

Oral courses may be given on several days with longer rest periods. Transplant conditioning uses a defined high-dose course followed by planned stem-cell support.

The dose is calculated for your treatment and health. Rest periods, delays or dose changes may be needed. Do not change doses or treatment dates yourself, except to follow specific instructions to stop a medicine and seek help for warning symptoms. The team will give you a revised plan when needed.

During an infusion, tell the nurse immediately about pain, burning, swelling or leaking around the cannula or line. Do not massage the area or apply heat or ice unless the team instructs you.

Tests and monitoring during treatment

Blood counts, kidney function, mouth and bowel symptoms and infection risk are checked. High-dose treatment requires specialist monitoring, transfusion support and a transplant care plan.

At each review, describe symptoms since the previous dose, including problems that have already settled. Blood tests can detect effects before you feel them. Your team may delay, reduce, omit or stop a medicine if results or symptoms make this necessary. The decision depends on the exact protocol; a normal result does not make new symptoms safe to ignore.

Side effects: what to expect

The frequency and severity of side effects differ greatly between conventional oral courses and high-dose transplant treatment. The table lists reported effects with practical care; it does not assign one frequency to both settings. Mouth injury, diarrhoea and hair loss can be especially prominent with high-dose treatment.

You are unlikely to have all of these effects. This is not an exhaustive list. Tell the team about any new or troublesome symptom, including one not mentioned here.

Reported side effects and practical care

Other reported side effects (frequency varies)

These effects may be common in some treatment settings, less common in others, or have no reliable frequency estimate. Their position here does not mean they are necessarily less frequent or less serious.

Allergic reactions and changes in kidney or liver tests can occur. High-dose melphalan can cause sudden kidney injury, especially if kidney function is already reduced. Report much less urine, new swelling or persistent vomiting urgently. Mouth soreness is particularly important with high-dose treatment; rates from transplant treatment should not be applied to a low-dose oral course.

Report these at review, or sooner if worsening. Use the urgent guidance for symptoms that affect breathing, drinking, urination, walking, consciousness or bleeding.

Important serious side effects, including rare effects

Serious does not always mean rare. Where reliable incidence is unavailable, no numerical frequency is assigned. The action to take matters even when the likelihood is uncertain.

Serious infection or sepsis

Low white cells can allow an infection to become life-threatening quickly. Fever, shaking chills, rapid breathing, confusion or sudden deterioration needs immediate help. Infection is possible even with a normal temperature.

Severe infusion or allergic reaction

Itching, flushing, wheeze, dizziness, throat tightness or facial swelling can start during an infusion or later. Tell the nurse immediately. Severe breathing difficulty, collapse or sudden face/throat swelling is an emergency.

Inflammation or injury in the lungs

A new cough, breathlessness, chest discomfort or falling exercise tolerance needs urgent oncology contact. Severe or rapidly worsening breathing difficulty is an emergency. Infection and other causes must also be considered.

A later second cancer

Some chemotherapy medicines can increase the risk of another cancer, including a blood cancer, years later. This is considered when balancing treatment benefits and risks. Keep follow-up appointments and report persistent unexplained symptoms.

Serious liver injury

Yellow eyes or skin, dark urine, severe upper abdominal pain, rapid abdominal swelling or unexplained weight gain needs urgent review. Some treatments can injure the small blood vessels in the liver.

Practical advice for this treatment

For high-dose treatment, the transplant unit may offer supervised oral cooling to reduce mouth injury. Follow its protocol. For tablets, check food timing and whether refrigeration is needed; do not crush or split them without pharmacy advice.

Taking cancer medicines at home

Keep cancer tablets or capsules in their labelled packaging, away from children and pets. Do not share them or put them in a household pill organiser unless the oncology pharmacist agrees. Do not crush, open, split or dissolve them without product-specific pharmacy instructions. A caregiver who handles the medicine should follow the pharmacy’s precautions. Return unused medicine to the pharmacy. If you miss a dose or vomit after one, do not take an extra dose; contact the team and follow the medicine-specific plan. If you accidentally take an extra dose or the wrong strength, contact the oncology team urgently even if you feel well. Do not try to make yourself vomit or work out a compensating dose yourself.

Medicines, interactions and vaccinations

Give the oncology pharmacist a complete list of prescription medicines, over-the-counter products, vitamins, herbal and Ayurvedic remedies and supplements. Ask before starting anything new. Do not stop prescribed medicines, including anticoagulants or infection-prevention medicines, without a coordinated plan. Live vaccines are generally unsuitable while immunity is suppressed; discuss vaccine type and timing with the team. Non-live vaccines may be offered, although protection can be reduced. Follow any specific stop-and-call instructions for the cancer medicine on this page or in your written treatment plan.

Other immune-suppressing or kidney-affecting medicines need review. Oral absorption and storage depend on the product, so follow pharmacy instructions.

Pregnancy, fertility and breastfeeding

Chemotherapy can harm a developing baby and may affect fertility temporarily or permanently. Discuss sperm, egg or embryo storage before treatment when feasible. If you could become pregnant, or your partner could become pregnant, use effective contraception during treatment and for the drug-specific period afterwards. Ask for a written end date based on every medicine in your plan: the required period differs by product and can extend for months. Periods stopping does not guarantee infertility. Tell the team immediately about a suspected pregnancy. Do not breastfeed during treatment; restart only after the team confirms the interval for the exact medicine and formulation.

Everyday life

Gentle activity, regular meals and rest can help you cope. Prepare food hygienically and drink according to your individual fluid plan. Avoid smoking; ask whether alcohol is safe with your exact treatment and other medicines. Plan work, travel and driving around symptoms and monitoring visits. Do not drive if dizzy, sleepy, weak, visually affected or impaired by neuropathy. You can usually be close to family and friends: chemotherapy does not make you radioactive. Follow the unit’s instructions for handling urine, vomit, laundry and sexual precautions for the period it specifies.

When to contact the oncology team

Contact the oncology team the same day for persistent vomiting or diarrhoea, difficulty drinking, reduced urine, mouth pain affecting eating, new cough, jaundice, a painful or spreading rash, unusual bruising, worsening constipation or new nerve symptoms. Use the more urgent instructions for the particular symptom when given above. If the team cannot be reached and you are unwell, seek urgent medical care.

Write down your hospital’s daytime and out-of-hours numbers before treatment. CTI’s general contact details do not replace the emergency number provided by your treating hospital.

After treatment

Keep the planned blood tests and follow-up appointments after the last dose. Blood counts and immunity may take time to recover, and some effects can start or persist later. Contact the team about symptoms rather than waiting for a routine visit. Keep a treatment summary, including drug names, dates and any serious reactions, for future doctors. Response may be assessed with examination, scans or blood or marrow tests, depending on the cancer.

Frequently asked questions