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Nucleoside metabolic inhibitor

Clofarabine is an intravenous medication used to treat certain types of leukemia in children. It works by stopping the growth of cancer cells by interfering with their DNA. Because it can lower blood cell counts and affect organs like the liver and kidneys, it requires close medical supervision.

How it worksClofarabine works by getting inside cells and turning into an active form that stops the cell from making new DNA. It also interferes with the enzymes that repair DNA and disrupts the cell's energy centers to trigger cell death.

Brand names:CLOLAR

Used for
Relapsed or refractory acute lymphoblastic leukemia in pediatric patients aged 1 to 21 years old.
Conditions
Acute lymphoblastic leukemia
Class
AntineoplasticNucleoside metabolic inhibitorHematologicImmune SystemHepaticRenalIntegumentary

How to take

FormsIntravenous infusion
RoutesIntravenous

The standard dose for children is 52 mg/m² given as an IV infusion over 2 hours once daily for 5 days. This is usually part of a 28-day cycle, with cycles repeating every 2 to 6 weeks.

The dose may need to be lowered if there is kidney trouble. Do not mix other medicines in the same IV line during administration. Patients often need extra support like IV fluids and special treatments to manage side effects during the 5-day period.

Key warnings

  • ·Severe drop in blood cells that can lead to infection or bleeding.
  • ·Risk of serious infection due to bone marrow suppression.
  • ·Risk of serious bleeding in the brain, gut, or lungs.
  • ·Potential for severe liver or kidney damage.
  • ·Risk of life-threatening skin reactions.
  • ·Risk of tumor lysis syndrome during treatment.

Side effects

Common

VomitingNauseaDiarrheaFever with low white blood cell countsItchingHeadacheRashFatigueAbdominal painChills

Serious

  • ·Severe or prolonged low blood cell counts (myelosuppression)
  • ·Serious bleeding (brain, stomach, or lung)
  • ·Severe infections (sepsis)
  • ·Tumor lysis syndrome (high uric acid levels)
  • ·Severe liver damage (hepatotoxicity)
  • ·Kidney failure
  • ·Severe intestinal inflammation (enterocolitis)
  • ·Severe skin reactions (Stevens-Johnson syndrome or toxic epidermal necrolysis)

Seek help if

  • ·Signs of infection
  • ·Signs of bleeding
  • ·Signs of liver failure
  • ·Signs of skin reactions

Interactions

  • ·Leukeran
  • ·Thiotepa
  • ·Myleran
  • ·Cosmegen
  • ·Ameluz
  • ·Valchlor
  • ·DTIC-Dome
  • ·Depocyt
  • ·Bicnu
  • ·Purixan
  • ·Thioguanine
  • ·Grafapex

Special populations

PregnancyClofarabine can cause harm to an unborn baby. Animal studies showed it caused death or physical abnormalities in developing fetuses. It should only be used during pregnancy if the benefits to the mother are greater than the risks to the baby. Women of reproductive age should use effective birth control during treatment and for 6 months after the last dose. Men should also use effective contraception during treatment.

PediatricClofarabine is specifically indicated for pediatric patients between the ages of 1 and 21 who have relapsed or refractory acute lymphoblastic leukemia.

GeriatricThe safety and effectiveness of Clofarabine have not been established for patients aged 65 and older.

Talk to your doctor

Monitor

  • ·Complete blood counts
  • ·Platelet counts
  • ·Liver enzymes
  • ·Bilirubin levels
  • ·Creatinine levels
  • ·Signs of infection
  • ·Signs of skin reactions

Questions to ask

  • ·What are the risks of infection while on Clofarabinine?
  • ·How often will my blood counts be monitored?
  • ·What should I do if I develop a fever?
  • ·How will this affect my kidney or liver function?

Pharmacokinetics

Half-life5.2 hours
Protein binding47% bound to plasma proteins, predominantly to albumin
MetabolismSequentially metabolized intracellularly to the 5’-monophosphate metabolite by deoxycytidine kinase and mono- and di-phospho-kinases to the active 5’-tripolphosphate metabolite.

Practical

OverdoseThere are no known human overdoses reported, but high doses in studies have caused severe liver issues, vomiting, and skin rashes.

Ingredients

Inactive

Water for Injection, USPSodium Chloride, USP

Educational summary derived from FDA labeling, AI-assisted. Not medical advice — consult a healthcare professional. Updated 2026-04-25.