Alkeran (Melphalan)
Alkylating chemotherapy agent for multiple myeloma and ovarian cancer
Alkeran (melphalan) is a cytotoxic alkylating chemotherapy drug prescribed for the treatment of multiple myeloma and advanced ovarian cancer. It works by forming cross-links in the DNA of cancer cells, preventing them from dividing and growing. Alkeran is available as a 2 mg film-coated tablet and requires a prescription. Treatment must be supervised by a specialist oncologist, with regular blood monitoring to manage potential bone marrow suppression.
Quick Facts
Key Takeaways
- Alkeran (melphalan) is a chemotherapy drug used primarily for multiple myeloma and advanced ovarian cancer, given in cyclical treatment courses.
- Bone marrow suppression is the most significant side effect, requiring regular blood count monitoring before and during each treatment cycle.
- Tablets must be stored in a refrigerator at 2–8°C and swallowed whole—never crushed, split, or chewed.
- Alkeran must not be used during pregnancy or breastfeeding; both female and male patients must use effective contraception during and after treatment.
- Long-term use may increase the risk of developing secondary cancers, including acute leukemia; your oncologist will weigh benefits against risks.
What Is Alkeran and What Is It Used For?
Melphalan, the active ingredient in Alkeran, was first synthesized in the 1950s and has been a cornerstone of cancer treatment for decades. It is classified as a nitrogen mustard derivative—a type of alkylating agent that works by forming chemical cross-links between and within the two strands of DNA in cancer cells. These cross-links prevent the DNA from being properly replicated during cell division, ultimately leading to programmed cell death (apoptosis). Melphalan is considered cell cycle non-specific, meaning it can damage cancer cells regardless of what phase of the cell cycle they are in, making it effective against both actively dividing and resting cancer cells.
In the treatment of multiple myeloma, Alkeran has been used for over 60 years and remains an important part of many treatment regimens. Multiple myeloma is a cancer of plasma cells—a type of white blood cell that normally produces antibodies to help fight infections. When these plasma cells become malignant, they multiply uncontrollably in the bone marrow, producing abnormal proteins and crowding out healthy blood cells. Alkeran is often used in combination with prednisone (the MP regimen) or with other agents in various combination protocols. While newer therapies such as proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies have expanded treatment options, melphalan-based regimens continue to play a significant role, particularly for patients who are not candidates for stem cell transplantation.
For advanced ovarian cancer (specifically epithelial ovarian carcinoma), Alkeran may be used as a single-agent treatment, particularly in patients who have relapsed or who are not suitable for platinum-based chemotherapy. Ovarian cancer is often diagnosed at an advanced stage because early symptoms are vague and non-specific. Alkylating agents like melphalan have demonstrated activity against ovarian cancer cells and can provide clinical benefit in certain patient populations.
Melphalan is also used in high-dose conditioning regimens prior to autologous stem cell transplantation (ASCT), though this application typically uses the intravenous formulation rather than the oral tablets described here. The drug is manufactured by Aspen Pharma Trading Limited and is available internationally under the brand name Alkeran.
Alkeran should only be prescribed and supervised by physicians experienced in cancer chemotherapy. The dosage is carefully individualized based on body weight, blood counts, and the specific type and stage of cancer being treated. Never start, stop, or change the dose of this medication without your oncologist's guidance.
What Should You Know Before Taking Alkeran?
Before your oncologist prescribes Alkeran, a thorough medical evaluation is necessary. This includes a complete blood count, kidney function tests, and a comprehensive review of your medical history and all medications you are currently taking. Open communication with your healthcare team is essential for safe and effective treatment.
Contraindications
There are specific situations in which Alkeran must not be used. Understanding these contraindications is critical for patient safety:
- Hypersensitivity: Do not take Alkeran if you are allergic to melphalan or any of the inactive ingredients in the tablet formulation, including microcrystalline cellulose, crospovidone, colloidal anhydrous silica, magnesium stearate, hypromellose, macrogol, or titanium dioxide (E 171).
- Breastfeeding: Alkeran is contraindicated during breastfeeding. Cytotoxic drugs can pass into breast milk and harm the nursing infant.
Warnings and Precautions
Several important precautions must be considered before and during treatment with Alkeran. Discuss the following situations with your oncologist:
- Recent radiotherapy or chemotherapy: If you have recently received radiation treatment or other chemotherapy drugs, your bone marrow may already be suppressed. Your oncologist will need to assess your blood counts carefully before starting Alkeran, as the additive effect on bone marrow suppression can be significant.
- Impaired kidney function: Patients with reduced kidney (renal) function may experience slower clearance of melphalan from the body, leading to increased drug levels and a higher risk of toxic effects. Dose adjustments are often necessary in patients with renal impairment. Your oncologist will monitor kidney function tests and adjust your dose accordingly.
- Vaccination status: Certain live vaccines (such as measles, mumps, rubella, oral polio, yellow fever, and BCG) must be avoided during treatment with Alkeran. Because the drug suppresses the immune system, live vaccines could cause serious or potentially fatal infections in immunocompromised patients. Inactivated vaccines may be given but may produce a reduced immune response.
- Combined oral contraceptives: If you are taking combined hormonal contraceptives (birth control pills containing both estrogen and progestogen), you should switch to an alternative effective contraceptive method. The combination of Alkeran treatment and hormonal contraceptives increases the risk of venous thromboembolism (blood clots). This elevated risk persists for 4–6 weeks after stopping the oral contraceptive pill.
- Family planning: Melphalan can damage DNA in reproductive cells (genotoxicity) and may cause permanent infertility in both men and women. If you plan to have children in the future, discuss fertility preservation options (such as sperm banking or egg freezing) with your oncologist before starting treatment.
Alkeran may increase the risk of developing other types of cancer, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). This risk is related to the cumulative dose and duration of treatment. Your oncologist will carefully evaluate the potential benefits of treatment against this risk before prescribing Alkeran, and will discuss the balance of benefits and risks with you.
Pregnancy and Breastfeeding
Alkeran poses serious risks to reproductive health and to an unborn child. The following guidelines are essential:
Pregnancy: Melphalan is teratogenic, meaning it can cause birth defects. It can also damage sperm, eggs, and a developing fetus. Alkeran should not be used during pregnancy. If you are already pregnant, inform your oncologist immediately before receiving treatment. Effective and reliable contraception must be used during treatment:
- Female patients: Use effective contraception during treatment and for at least 6 months after the last dose.
- Male patients: Use effective contraception during treatment and for at least 3 months after the last dose.
Breastfeeding: Do not breastfeed while taking Alkeran. Cytotoxic agents and their metabolites may pass into breast milk and could harm the nursing infant.
Fertility: Melphalan can significantly affect fertility in both men and women. In women, it can damage ovarian function, leading to premature ovarian insufficiency, infertility, and amenorrhea (absence of menstrual periods). In men, it can cause azoospermia (absence of sperm in semen), which may be temporary or permanent. Male patients are strongly recommended to seek advice regarding sperm cryopreservation before beginning treatment.
Driving and Operating Machinery
There are no specific studies on the effect of melphalan on driving ability or the use of machinery. However, because of potential side effects such as nausea, fatigue, and general malaise associated with chemotherapy, patients should assess their own ability to drive or operate machinery. If you experience side effects that impair your alertness or reaction times, avoid driving or operating heavy machinery and consult your oncologist.
How Does Alkeran Interact with Other Drugs?
Drug interactions can significantly affect the safety and efficacy of Alkeran. Some interactions may increase the risk of serious side effects, while others may reduce the effectiveness of the chemotherapy. Your oncologist and pharmacist will review all your current medications to identify and manage potential interactions.
Major Interactions
| Drug | Interaction | Clinical Significance |
|---|---|---|
| Live vaccines (e.g., MMR, oral polio, yellow fever, BCG) | Risk of severe or fatal vaccine-induced infection in immunocompromised patients | Contraindicated during treatment; wait until immune recovery before vaccination |
| Nalidixic acid (antibiotic) | Reported fatal hemorrhagic enterocolitis when combined with high-dose IV melphalan in pediatric patients | Avoid concurrent use; consider alternative antibiotics |
| Cyclosporine (immunosuppressant) | Increased risk of nephrotoxicity (kidney damage), especially following stem cell transplantation | Close monitoring of renal function required; dose adjustment may be necessary |
| Busulfan (chemotherapy) | Enhanced myelotoxicity and hepatic veno-occlusive disease risk in high-dose conditioning regimens | Requires careful dose scheduling and monitoring in transplant settings |
Other Considerations
Beyond the major interactions listed above, there are additional pharmacological considerations that your healthcare team will take into account:
- Other myelosuppressive agents: Concurrent use of other drugs that suppress bone marrow function (including other chemotherapy drugs, certain antibiotics like chloramphenicol, and some anticonvulsants) may compound the risk of severe blood count depression.
- Nephrotoxic drugs: Medications that can harm the kidneys, such as aminoglycoside antibiotics, amphotericin B, or NSAIDs (non-steroidal anti-inflammatory drugs), may increase melphalan levels and toxicity due to reduced renal clearance.
- Food interactions: Absorption of oral melphalan can be reduced by food. Some guidelines suggest taking Alkeran on an empty stomach to maximize absorption, though your oncologist will provide specific instructions based on your treatment protocol.
Always provide your oncologist and pharmacist with a complete list of all medications you are taking, including over-the-counter drugs, vitamins, supplements, and herbal products. This is essential for preventing dangerous interactions and ensuring the most effective treatment.
What Is the Correct Dosage of Alkeran?
The dosing of Alkeran is carefully calculated and individualized by your treating oncologist. Chemotherapy dosing depends on many factors, including your body weight, overall health status, kidney function, blood cell counts, and the specific type and stage of cancer being treated. Your oncologist will regularly monitor your blood counts and may adjust the dose or delay treatment cycles based on how your body responds.
Adult Dosage
Multiple Myeloma
Standard dose: 0.15 mg per kg body weight per day for 4 consecutive days.
Treatment cycle: This 4-day course is repeated every 6 weeks (42 days).
Combination therapy: Melphalan is frequently combined with prednisone (the MP regimen) or with other agents such as thalidomide (MPT regimen) or bortezomib (VMP regimen) in patients not eligible for autologous stem cell transplantation.
Dose adjustments: Your oncologist will adjust doses based on your blood counts at the nadir (lowest point, usually 2–3 weeks after the start of each cycle) and before the next cycle. Treatment may be delayed if blood counts have not recovered sufficiently.
Advanced Ovarian Cancer
Standard dose: 0.2 mg per kg body weight per day for 5 consecutive days.
Treatment cycle: This 5-day course is repeated every 4 to 8 weeks, depending on the degree of bone marrow recovery.
Dose adjustments: The interval between cycles may be lengthened if bone marrow recovery is slow. Your oncologist will monitor complete blood counts and adjust the schedule accordingly.
| Indication | Dose | Duration | Cycle Interval |
|---|---|---|---|
| Multiple Myeloma | 0.15 mg/kg/day | 4 days | Every 6 weeks |
| Ovarian Cancer | 0.2 mg/kg/day | 5 days | Every 4–8 weeks |
Patients with Kidney Impairment
Patients with impaired kidney function require special attention when taking Alkeran. Melphalan is partially eliminated through the kidneys, and reduced renal function can lead to higher blood levels of the drug and increased toxicity. Your oncologist will typically reduce the dose and extend the interval between treatment cycles in patients with significant renal impairment. Regular monitoring of both kidney function and blood counts is especially important in this patient group.
Elderly Patients
Older adults may be more sensitive to the effects of melphalan, particularly bone marrow suppression. Age-related decline in kidney function is common and may affect drug clearance. While there is no specific age-related dose recommendation, oncologists typically start with the lower end of the dosing range and adjust based on individual tolerance and blood count response. Careful monitoring of complete blood counts and renal function is essential in elderly patients.
How to Take Alkeran
- Swallow the tablets whole with a glass of water. Do not split, crush, or chew the tablets.
- The film coating protects handlers from direct contact with the cytotoxic agent; as long as the coating is intact, there is no risk from normal handling.
- Some treatment protocols recommend taking Alkeran on an empty stomach to optimize absorption. Follow your oncologist's specific instructions.
- Take the tablets at the same time each day during a treatment course for consistent blood levels.
Missed Dose
If you forget to take a dose of Alkeran, contact your oncologist for guidance. Do not take a double dose to compensate for a missed one. Your oncologist will advise you on whether to take the missed dose or wait until the next scheduled dose, depending on how much time has elapsed.
Overdose
If you take more Alkeran than prescribed, or if a child accidentally ingests the medication, seek emergency medical attention immediately. Contact your local poison control center or emergency department. Bring the medication packaging with you so healthcare professionals can identify the drug and dose ingested. Overdose can lead to severe bone marrow suppression, gastrointestinal toxicity, and other serious complications that require intensive medical management.
What Are the Side Effects of Alkeran?
Like all chemotherapy drugs, Alkeran can cause side effects, though not everyone experiences them. The most significant side effect is bone marrow suppression, which reduces the production of blood cells and increases vulnerability to infections, bleeding, and anemia. Your oncologist will monitor your blood counts regularly throughout treatment and adjust your dose as needed.
It is important to contact your oncologist or seek immediate medical attention if you experience signs of a severe allergic reaction (hives, facial/throat swelling, difficulty breathing), signs of infection (fever, persistent sore throat, even with a normal temperature), unusual bruising or bleeding, severe shortness of breath, or chest pain.
Very Common
May affect more than 1 in 10 patients
- Bone marrow suppression (reduced red blood cells, white blood cells, and platelets)
- Nausea and vomiting
- Diarrhea
- Mouth sores and inflammation (stomatitis)
- Hair loss (alopecia)
- Absence of menstrual periods in women (amenorrhea)
Common
May affect up to 1 in 10 patients
- Secondary blood cancer (acute leukemia)
- Elevated blood urea levels (in patients with kidney impairment being treated for myeloma)
Rare
May affect up to 1 in 1,000 patients
- Allergic reactions (hives, itching, facial/throat swelling, difficulty breathing, chest tightness)
- Lung disease (interstitial pneumonitis, pulmonary fibrosis) – symptoms include dry cough, shortness of breath, and fever
- Liver damage (hepatitis, jaundice – yellowing of the skin and eyes)
- Skin rash and itching
Frequency Unknown
Reported but frequency not established
- Deep vein thrombosis (DVT) – blood clot in the leg veins, causing pain, swelling, and warmth
- Pulmonary embolism (PE) – blood clot in the lungs, causing chest pain, sudden shortness of breath, cough, dizziness, or sweating
- Azoospermia in men (absence of sperm in semen)
- Acute kidney injury (rapid decline in kidney function)
Managing Side Effects
Many side effects of Alkeran can be managed with supportive care. Your oncology team may prescribe anti-emetic (anti-nausea) medications to help with nausea and vomiting. Maintaining good oral hygiene and using gentle mouthwashes can help prevent or reduce the severity of stomatitis. It is important to stay well-hydrated and eat a balanced diet to support your body during treatment.
To reduce the risk of infection when your white blood cell count is low, practice good hand hygiene, avoid crowded places, and contact your oncologist immediately if you develop a fever (even a low-grade fever of 38°C / 100.4°F or above) or other signs of infection. Your oncologist may prescribe prophylactic antibiotics or growth factors to support your immune system during periods of low blood counts.
- Signs of allergic reaction: hives, swelling of face, tongue, or throat, difficulty breathing
- Signs of blood clot: sudden chest pain, unexplained shortness of breath, leg pain or swelling
- Signs of severe infection: fever (even low-grade), chills, persistent sore throat
- Unusual bleeding or bruising, very dark or tarry stools, blood in urine
- Yellowing of skin or eyes (jaundice)
Reporting Side Effects
If you experience any side effects, including those not listed here, report them to your healthcare provider. You can also report suspected adverse drug reactions directly to your national pharmacovigilance authority. Reporting side effects helps regulatory agencies continuously monitor the benefit-risk balance of medicines and contributes to drug safety for all patients.
How Should You Store Alkeran?
Proper storage of Alkeran is essential to maintain the drug's effectiveness and to prevent accidental exposure. Unlike most oral medications, Alkeran requires refrigerated storage, which is an important detail to remember:
- Temperature: Store in a refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze.
- Container: Keep the tablets in their original amber glass bottle with the child-resistant cap securely fastened.
- Safety: Store well out of sight and reach of children. Alkeran is a cytotoxic drug and accidental ingestion by a child requires immediate emergency medical treatment.
- Expiration: Do not use Alkeran after the expiration date (marked "EXP" on the packaging). Expired chemotherapy drugs may be less effective or potentially harmful.
- Disposal: Do not dispose of unused or expired Alkeran tablets in household waste or down the drain. As a cytotoxic drug, it requires special handling. Return unused tablets to your pharmacist for safe disposal according to local regulations for hazardous pharmaceutical waste.
While the film coating on intact Alkeran tablets prevents harmful exposure during normal handling, it is advisable to wash your hands after handling the tablets. If a tablet is broken or damaged, avoid direct skin contact with the contents. Use gloves to handle broken tablets and clean any contaminated surfaces. Caregivers who are pregnant should not handle chemotherapy medications.
What Does Alkeran Contain?
Active ingredient: Each film-coated tablet contains 2 mg of melphalan. Melphalan is the pharmacologically active component responsible for the anticancer effect of the drug.
Inactive ingredients (excipients): The tablet core contains microcrystalline cellulose (filler/binder), crospovidone (disintegrant), colloidal anhydrous silica (flow agent), and magnesium stearate (lubricant). The film coating consists of hypromellose, macrogol, and titanium dioxide (color additive E 171).
Appearance: Alkeran tablets are white to off-white, round, biconvex, film-coated tablets. They are embossed with the letter "A" on one side and "GX EH3" on the other side for identification.
Packaging: The tablets are supplied in amber (brown) glass bottles with child-resistant caps, available in pack sizes of 25 or 50 tablets. The amber glass provides protection from light, which is important for maintaining the chemical stability of melphalan.
Marketing authorization holder: Aspen Pharma Trading Limited, 3016 Lake Drive, Citywest Business Campus, Dublin 24, Ireland.
Manufacturer: Excella GmbH & Co. KG, Nürnberger Strasse 12, 90537 Feucht, Germany.
Frequently Asked Questions About Alkeran
Alkeran (melphalan) is a chemotherapy drug used primarily to treat multiple myeloma, a cancer of plasma cells in the bone marrow, and advanced ovarian cancer (epithelial ovarian carcinoma). It belongs to the class of alkylating agents and works by damaging the DNA of cancer cells, preventing them from dividing and growing. In multiple myeloma, it is commonly used in combination with prednisone (the MP regimen) or with newer agents in various combination protocols. Melphalan has been a cornerstone of myeloma treatment for over 60 years.
The most common side effects of Alkeran include bone marrow suppression (leading to low counts of white blood cells, red blood cells, and platelets), nausea, vomiting, diarrhea, mouth sores (stomatitis), and hair loss (alopecia). Bone marrow suppression is the most clinically significant side effect and requires regular blood count monitoring. Women may also experience amenorrhea (absence of menstrual periods). Your oncologist will monitor your blood counts before and during each treatment cycle and adjust your dose as necessary.
Alkeran tablets must be stored in a refrigerator at 2–8°C (36–46°F). Do not freeze. Keep them in the original amber glass bottle with the child-resistant cap securely closed. Store out of sight and reach of children. Do not use the tablets after the expiration date marked on the packaging. Unlike most oral medications, Alkeran requires refrigerated storage to maintain chemical stability. Unused or expired tablets should be returned to your pharmacist for safe disposal.
Alkeran must not be used during pregnancy as it is teratogenic and can cause serious birth defects and harm to a developing fetus. Women of childbearing potential must use effective contraception during treatment and for at least 6 months after the last dose. Male patients should also use effective contraception during treatment and for at least 3 months afterward. Breastfeeding is contraindicated during Alkeran treatment. Patients who wish to have children in the future should discuss fertility preservation options with their oncologist before starting treatment.
Yes, Alkeran can interact with several medications. Live vaccines (such as MMR, oral polio, and yellow fever) are contraindicated as they may cause serious infection in immunocompromised patients. Nalidixic acid (an antibiotic) has been associated with fatal hemorrhagic enterocolitis when combined with high-dose melphalan. Cyclosporine may increase the risk of kidney damage, and busulfan (another chemotherapy drug) may cause enhanced toxicity when combined with melphalan. Always tell your oncologist about all medications you are taking, including over-the-counter drugs, supplements, and herbal products.
Regular complete blood count (CBC) monitoring is essential while taking Alkeran. Your oncologist will check white blood cell counts (especially neutrophils), platelet counts, and hemoglobin levels before each treatment cycle and at regular intervals during treatment. Blood counts typically reach their lowest point (nadir) approximately 2–3 weeks after starting each cycle. Your dose may be adjusted or the next cycle delayed if blood counts have not recovered sufficiently. Kidney function tests (serum creatinine, eGFR) may also be monitored, particularly in patients with pre-existing renal impairment.
References
This article is based on the following evidence-based sources and international guidelines:
- European Medicines Agency (EMA). Alkeran Summary of Product Characteristics (SmPC). Available at: www.ema.europa.eu. Accessed January 2026.
- U.S. Food and Drug Administration (FDA). Alkeran (melphalan) Prescribing Information. Available at: www.fda.gov. Accessed January 2026.
- National Comprehensive Cancer Network (NCCN). Clinical Practice Guidelines in Oncology: Multiple Myeloma. Version 4.2025. www.nccn.org.
- Dimopoulos MA, et al. Multiple myeloma: EHA-ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology. 2021;32(3):309-322. doi:10.1016/j.annonc.2020.11.014
- British National Formulary (BNF). Melphalan drug monograph. Available at: bnf.nice.org.uk. Accessed January 2026.
- World Health Organization (WHO). WHO Model List of Essential Medicines – 23rd List, 2023. Geneva: WHO; 2023.
- Facon T, et al. Melphalan and prednisone plus thalidomide versus melphalan and prednisone alone or reduced-intensity autologous stem cell transplantation in elderly patients with multiple myeloma (IFM 99-06). Lancet. 2007;370(9594):1209-1218.
- San Miguel JF, et al. Bortezomib plus melphalan and prednisone for initial treatment of multiple myeloma. New England Journal of Medicine. 2008;359(9):906-917.
- Palumbo A, Anderson K. Multiple Myeloma. New England Journal of Medicine. 2011;364(11):1046-1060. doi:10.1056/NEJMra1011442
- International Agency for Research on Cancer (IARC). IARC Monographs on the Identification of Carcinogenic Hazards to Humans: Melphalan. Volume 100A. Lyon: IARC; 2012.
Editorial Team
This article has been written and reviewed by medical professionals with expertise in oncology, hematology, and clinical pharmacology. Our editorial process follows international medical standards and the GRADE evidence framework.
iMedic Medical Editorial Team – specialists in oncology and hematology with experience in evidence-based content development for patient education.
iMedic Medical Review Board – an independent panel of board-certified physicians who review all content according to international guidelines (EMA, FDA, NCCN, ESMO, BNF).
Evidence standard: All medical claims in this article are based on Level 1A evidence (systematic reviews and meta-analyses of randomized controlled trials) or established international clinical practice guidelines. This article has no pharmaceutical company sponsorship or advertising influence.
Last reviewed: | Published: