Cyclophosphamide
Cyclophosphamide is a potent medication primarily used in the treatment of various cancers and autoimmune diseases. As an alkylating agent, it works by interfering with the replication of rapidly dividing cells, including cancer cells and certain immune cells.

Key Takeaways
- Cyclophosphamide is a chemotherapy drug and immunosuppressant.
- It functions by damaging the DNA of rapidly dividing cells, preventing their growth and spread.
- The drug is used to treat a range of cancers, including lymphomas and leukemias, as well as severe autoimmune conditions.
- Common side effects include nausea, hair loss, and bone marrow suppression, requiring careful monitoring.
- Its administration requires close medical supervision due to its potent effects and potential for serious adverse reactions.
What is Cyclophosphamide?
Cyclophosphamide is a cytotoxic chemotherapy drug belonging to the class of nitrogen mustard alkylating agents. It is a prodrug, meaning it must be metabolized by the liver into its active forms, phosphoramide mustard and acrolein, to exert its therapeutic effects. This medication is widely recognized for its role in oncology, where it targets and destroys rapidly proliferating cells, such as those found in tumors. Beyond cancer treatment, it also functions as an immunosuppressant, making it valuable in managing certain severe autoimmune disorders.
Globally, cancer remains a significant health challenge, with chemotherapy, including agents like cyclophosphamide, forming a cornerstone of treatment strategies for many types of malignancies. According to the World Health Organization (WHO), cancer is a leading cause of death worldwide, underscoring the critical need for effective therapeutic agents.
How Does Cyclophosphamide Work?
The **cyclophosphamide mechanism of action** involves its active metabolites forming covalent bonds with DNA strands within cells. This process, known as alkylation, primarily occurs at the guanine bases of DNA. By cross-linking DNA strands and causing breaks, cyclophosphamide disrupts the normal function of DNA and RNA synthesis, thereby inhibiting cell division and inducing programmed cell death (apoptosis). This action is most pronounced in cells that divide rapidly, such as cancer cells and certain immune cells, which makes it effective against various malignancies and autoimmune conditions.
The drug’s impact on DNA replication and transcription prevents cells from growing and multiplying, ultimately leading to their destruction. While highly effective against cancerous and overactive immune cells, this mechanism also affects healthy, rapidly dividing cells in the body, such as those in the bone marrow, hair follicles, and gastrointestinal tract, leading to many of its characteristic side effects.
Cyclophosphamide Uses and Side Effects
Cyclophosphamide is a versatile medication with a broad spectrum of clinical applications. It is primarily used to treat various types of cancer, addressing the question of what is cyclophosphamide used for. These include lymphomas (such as Hodgkin’s and non-Hodgkin’s lymphoma), leukemias (like acute myeloid leukemia and chronic lymphocytic leukemia), multiple myeloma, breast cancer, ovarian cancer, and certain solid tumors. In addition to its antineoplastic properties, cyclophosphamide is also utilized as an immunosuppressant for severe autoimmune diseases, including systemic lupus erythematosus, rheumatoid arthritis, vasculitis, and nephrotic syndrome, where it helps to suppress an overactive immune system.
Like all potent medications, cyclophosphamide carries a risk of **cyclophosphamide side effects**, which can range from mild to severe. These adverse reactions are largely due to its non-selective action on rapidly dividing cells. Common side effects include:
- Nausea and vomiting
- Hair loss (alopecia)
- Bone marrow suppression, leading to a decrease in white blood cells (increasing infection risk), red blood cells (anemia), and platelets (bleeding risk)
- Fatigue
- Mouth sores (mucositis)
- Diarrhea or constipation
More serious side effects can include hemorrhagic cystitis (bladder inflammation and bleeding), cardiotoxicity (heart damage), pulmonary fibrosis (lung scarring), and an increased risk of secondary cancers later in life. Due to these potential adverse effects, patients receiving cyclophosphamide require close medical supervision, including regular blood tests and monitoring for organ function, to manage and mitigate risks effectively.