Cyclophosphamide is a powerful medication used to treat various types of cancer and severe, life-threatening autoimmune conditions.
Cyclophosphamide is a conventional cytotoxic chemotherapy and potent immunosuppressant drug. It belongs to a class of medicines known as alkylating agents, which work by interfering with the replication of DNA, thereby stopping rapidly dividing cells—such as cancer cells or overactive immune cells—from multiplying.
Cyclophosphamide is used for people with:
Various blood cancers and solid tumours, including leukaemias, lymphomas, breast cancer, and ovarian cancer.
Severe autoimmune and vasculitic conditions, such as Granulomatosis with Polyangiitis (GPA), Microscopic Polyangiitis (MPA), and severe lupus nephritis (kidney inflammation caused by systemic lupus erythematosus).
Conditioning regimens prior to a bone marrow or stem cell transplant.
The dose of cyclophosphamide is highly individualised, calculated based on the patient’s body surface area, kidney function, and the specific condition being treated:
Formulations: It can be given either as an intravenous infusion (drip) in a hospital day-unit or as oral tablets taken at home.
Administration advice: Tablets must be swallowed whole, typically first thing in the morning with plenty of fluid. Patients are advised to drink at least 2 to 3 litres of water a day and empty their bladder frequently to protect the bladder lining.
Note: It is often given alongside a protective medication called Mesna to prevent bladder irritation and bleeding.
Cyclophosphamide has a significant side-effect profile that requires careful medical management.
Haemorrhagic cystitis (irritation, inflammation, and bleeding from the bladder lining, causing blood in the urine).
Bone marrow suppression (decreased production of white blood cells, red blood cells, and platelets, causing an increased risk of infection, anaemia, and bruising).
Nausea, vomiting, and temporary hair loss (alopecia).
Infertility (it can permanently impact fertility in both men and women).
Increased long-term risk of secondary cancers, particularly bladder cancer.
As an alkylating agent, cyclophosphamide is converted by the liver into active metabolites that bind to cellular DNA.
This binding chemically alters the DNA structure, preventing the cell from replicating its genetic material and dividing.
In cancer treatment, this process triggers cell death in rapidly multiplying malignant cells. In autoimmune diseases, it selectively suppresses the overactive lymphocytes (white blood cells) that are driving inflammation and tissue destruction.
Your healthcare team should monitor:
Full blood counts (FBC) frequently to check for low white blood cell levels (neutropenia) and manage infection risks.
Urinalysis (regular urine tests) to check for microscopic blood, an early sign of bladder irritation.
Kidney and liver function through regular blood tests.
Fluid intake and output, ensuring adequate hydration during treatment cycles.
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