Cyclophosphamide chemotherapy treatment


Cyclophosphamide is one of the old warhorses of chemotherapy. Administered intravenously or orally in tablet form, the drug has been around since the end of the 1950s and has found its way into treatment for a host of cancers, in both FDA-approved and off-label indications.

Because cyclophosphamide has been around for so long, more is known about it—in terms of safety and efficacy—than is known about most anti-cancer drugs. The drug is considered carcinogenic and is frequently implicated in secondary cancers.

Drug profile

  • Class: Alkylating agent
  • Mechanism of action: Cyclophosphamide adds a molecule called an alkyl group to DNA, which causes DNA damage leading to cell death.
  • Treatment type: Chemotherapy
  • US approval: 1959
  • Synonyms: Cytoxan, Endoxan, Revimmune
  • FDA Use-in-Pregnancy Rating: Category D (What is this?)

What cyclophosphamide is effective for and why

Cyclophosphamide is among the most utilized drugs in chemotherapy, with FDA-approved indications in many different types of cancer: Breast cancer, all four major leukemia subtypes (ALL, AML, CLL, CML), several subtypes of both Hodgkins and non-Hodgkins lymphoma, along with multiple myeloma, neuroblastoma, retinoblastoma, and ovarian cancer.

Cyclophosphamide has several off-label uses as well, including indications against bladder cancer, cervical cancer, endometrial cancer, testicular cancer, osteosarcoma, penile cancer, lung cancer, and prostate cancer.

While it is sometimes used as a single agent, more often it is in combination chemotherapy regimens, notably, CVP, FAC, CHOP, R-CHOP, BEACOPP, EPOCH and MACOP-B, to name a few.

Cyclophosphamide side effects: Overview

Serious side effects associated with cyclophosphamide tend to be very dose-related—the higher the dose, the higher the risk, most notably for cardiac toxicity, nausea/vomiting, hemorrhagic cystitis, myelosuppression, and secondary cancers. The most common types of secondary cancers are bladder cancer and myeloproliferative and lymphoproliferative cancers, or cancers of the bone marrow or lymph system (i.e. multiple myeloma, various subtypes of non-Hodgkins lymphoma). Such secondary cancers seem to occur most often in people being treated for lymphoproliferative cancers in the first place.


  • Boyiadzis, Michael M. et al. Hematology-Oncology Therapy. 2007. New York: McGraw Hill, Medical Publishing Division.
  • Cancer Drug Information, The National Cancer Institute
  • Cancer Drug Manual, BC Cancer Agency
  • Guide to Cancer Drugs, American Cancer Society
  • Perry, Michael C, Editor. Companion Handbook to the Chemotherapy Sourcebook. 1999. Baltimore; Williams & Wilkins.

Significant studies relating to cyclophosphamide


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