# Pentostatin

Source: https://onco.cc/drugs/pentostatin/  
OnCo record `pentostatin` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

An intravenous drug given every other week that produces long remissions in hairy cell leukaemia by poisoning the leukaemic lymphocytes' purine metabolism; cladribine is the one-week alternative.

## Summary

Pentostatin was approved in the United States in 1991 for hairy cell leukaemia, initially after interferon alfa had failed and then as first-line treatment, on the strength of a randomised comparison in which it produced far more complete remissions than interferon (Grever, JCO 1995). It is given as a short intravenous infusion of 4 mg/m² every other week until maximal response. It is also used in T-cell leukaemias and lymphomas and, off label, in graft-versus-host disease and reduced-intensity transplant conditioning. Prolonged lymphopenia with infection risk is the main toxicity, and renal impairment requires dose reduction.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Also known as: Deoxycoformycin; 2'-deoxycoformycin
- Tags: gap-fill; generic
- Brand: Nipent
- Modality: Purine analogue, adenosine deaminase inhibitor
- Mechanism: Binds adenosine deaminase almost irreversibly, so deoxyadenosine triphosphate accumulates in lymphocytes, blocks DNA synthesis and triggers apoptosis.
- Approvals: US 1991: Hairy cell leukaemia (initially alpha-interferon-refractory; later untreated disease)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Pentostatin
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pentostatin
- Grever 1995, JCO: pentostatin versus interferon alfa in hairy cell leukaemia: https://doi.org/10.1200/JCO.1995.13.4.974

## Connected records

- drugs: [Cladribine](https://onco.cc/drugs/cladribine/)
- cancers: [Hairy cell leukaemia](https://onco.cc/cancers/hairy-cell-leukemia/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/)
- targets: [Adenosine deaminase (ADA)](https://onco.cc/targets/ada/)
- trials: [Fludarabine, Cyclophosphamide, and Rituximab Versus Pentostatin, Cyclophosphamide, and Rituximab in Previously Untreated or Treated B-Cell Chronic Lymphocytic Leukemia Patients](https://onco.cc/trials/nct00254163/), [Methotrexate or Pentostatin for Graft-versus-host Disease Prophylaxis in Risk-adapted Allogeneic Bone Marrow Transplantation for Hematologic Malignancies](https://onco.cc/trials/nct01188798/), [Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia](https://onco.cc/trials/nct01059786/)
- key papers: [Randomized comparison of pentostatin versus interferon alfa-2a in previously untreated patients with hairy cell leukemia: an intergroup study](https://onco.cc/key-papers/paper-grever-j-clin-oncol/)

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