Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.
Melphalan is an alkylating chemotherapy medicine, a nitrogen mustard that cross-links DNA. At high doses by drip it is used before an autologous stem cell transplant in multiple myeloma; at lower doses as tablets, for myeloma and some other cancers; and in isolated limb or liver perfusion for melanoma. It is sold as Alkeran and Evomela, among other names.
Melphalan breaks down spontaneously in the blood rather than through liver enzymes. It enters cells through the amino acid transporter SLC7A5 (LAT1), and studies have looked at SLC7A5 and at CTLA4 for side effects such as mouth sores (mucositis) after high-dose treatment. The findings come from a few studies, and there is no CPIC or DPWG guideline.
Every published finding on gene variants and Melphalan. Upload your DNA file to see which of these rows apply to you.
PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.
CTLA4 rs4553808: Level 3. Toxicity. 1.
SLC7A5 rs4240803: Level 3. Toxicity: Multiple Myeloma. 1.
Study findings and evidence levels: PharmGKB (CC BY-SA 4.0). Guidelines: CPIC and DPWG, as bundled in PharmCAT. Most studies were done mainly in people of European ancestry.
More drugs from the same category
Alkylating Agents (class) (Alkylating Agents)
Not a prescribing recommendation. This page is educational information about pharmacogenomic research and clinical guidelines - it is not medical or dosing advice, and not a prediction of how any medication will work for you. Never start, stop, or change a medication or dose based on it; talk to your doctor or pharmacist, who can order clinical-grade testing if appropriate.
Content updated September 2026.
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