Pentostatin
Pentostatin is a purine analog antineoplastic agent used primarily to treat hairy cell leukemia, a rare form of blood cancer. It works by inhibiting adenosine deaminase, an enzyme essential for lymphocyte proliferation, thereby suppressing the growth of abnormal white blood cells. This medication is typically reserved for patients who have not responded to other treatments.
Pentostatin is an anticancer drug used to treat hairy cell leukemia by inhibiting lymphocyte growth.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L01XX08
- RxNorm
- 8011
- Generic
- Pentostatin
- Class
- atc-l01
- Brands
- Nipent
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Pentostatin is primarily indicated for the treatment of hairy cell leukemia, particularly in patients who have relapsed after or are resistant to alpha-interferon therapy. It may also be used in other lymphoid malignancies as determined by an oncologist.
Pentostatin is a potent inhibitor of adenosine deaminase, an enzyme critical for purine metabolism in lymphocytes. By blocking this enzyme, it disrupts DNA and RNA synthesis in rapidly dividing cells, leading to cell death.
Pentostatin is administered by intravenous infusion and requires careful monitoring of blood counts and organ function. It may cause significant immunosuppression, increased risk of infections, and potential kidney and liver toxicity. Patients should be adequately hydrated during treatment and avoid live vaccines while on therapy.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 25, 2026. · Read our editorial policy