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Cladribine (Mavenclad)

Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.

About Cladribine

What it is

Cladribine is a purine analogue that builds up in lymphocytes and kills them, because these cells have much of the enzyme that activates it and little of the one that inactivates it. As tablets taken in two short courses a year apart, it is used for highly active relapsing multiple sclerosis; given by drip, it treats hairy cell leukaemia. It is sold as Mavenclad and Leustatin.

How genes come into it

Cladribine is activated inside cells by deoxycytidine kinase and acts partly by blocking ribonucleotide reductase. Studies, mainly in leukaemia, have looked at the ribonucleotide reductase genes RRM1, RRM2 and RRM2B and at NT5C1A for response. The findings come from a few studies, and there is no CPIC or DPWG guideline.

What research found

Every published finding on gene variants and Cladribine. Upload your DNA file to see which of these rows apply to you.

Rated evidence (9 rated variants)

PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.

RRM2B rs1265138: Level 3. Efficacy: Leukemia, Myeloid, Acute. 1.

RRM2 rs1130609: Level 3. Efficacy: Leukemia, Myeloid, Acute. 1.

RRM2 rs5030743: Level 3. Efficacy: Leukemia, Myeloid, Acute. 1.

RRM1 rs2898950: Level 3. Efficacy: Leukemia, Myeloid, Acute. 1.

Response (6 findings)

Studies of how well it worked in people carrying a variant.

RRM1 rs1042919: Genotype AT is associated with decreased response to cladribine and cytarabine in children with Leukemia, Myeloid, Acute as compared to genotype AA.

RRM1 rs1561876: Genotype GG is associated with decreased response to cladribine and cytarabine in children with Leukemia, Myeloid, Acute as compared to genotypes AA + AG.

RRM1 rs2898950: Genotypes AC + CC is associated with increased response to cladribine and cytarabine in children with Leukemia, Myeloid, Acute as compared to genotype AA.

RRM2 rs1130609: Genotypes GT + TT is associated with decreased response to cladribine and cytarabine in children with Leukemia, Myeloid, Acute as compared to genotype GG.

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.

Related Drugs

More drugs from the same category

Methotrexate (Antimetabolites)

5-FU (Antimetabolites)

Alimta (Antimetabolites)

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.

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Content updated September 2026.

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