Cyclophosphamide (brand name Cytoxan) is a chemotherapy agent that can help suppress inflammation and has been used to treat certain forms of pulmonary fibrosis. It works by stopping or slowing the growth of certain types of cancer cells.

It usually is taken orally, but also may be administered intravenously (injected into the blood stream). The length of the treatment period depends on the types of other drugs being taken and how well the body responds to the treatment. The dosage also may be adjusted depending on the response to treatment and any side effects.

The most common side effects of Cytoxan include appetite loss, absence of menstruation, skin color changes, diarrhea, general feeling of unwellness, hair loss, nausea, skin rashes, stomach discomfort or pain, texture in nails, vomiting, and weakness.

Cytoxan for the treatment of pulmonary fibrosis

The potential benefits of Cytoxan for the treatment of pulmonary fibrosis has been tested in a number of studies.

A prospective study of 19 participants diagnosed with pulmonary fibrosis who did not respond to corticosteroids treatment, or experienced adverse effects, was published in June 2000. The participants were prescribed 2 mg per kg of Cytoxan a day. The results showed a sustained response in one participant while seven remained stable and 11 deteriorated while receiving the drug, suggesting that the effectiveness of Cytoxan therapy is limited in people with pulmonary fibrosis.

Nevertheless, a Phase 3 study called EXAFIP (NCT02460588) is assessing the effect of cyclophosphamide added to corticosteroid for the treatment of acute flares of pulmonary fibrosis.

Acute flares of pulmonary fibrosis are major events associated with the disease. The annual incidence rate of acute flares is between 5% and 10%, and the cause of death for one-third of pulmonary fibrosis patients who experience them.

The primary endpoint of the EXAFIP clinical trial is the early survival of participants at three months. Secondary endpoints include overall survival, respiratory mortality and morbidity, chest high-resolution CT scan features, prognosis of acute flares, time to visit after worsening, and blood biomarkers.

The estimated completion date of the EXAFIP trial is December 2018.

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