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Trade/other name(s): Hycamtin, topotecan hydrochloride

Why would this drug be used?

This drug is used to treat ovarian cancer, cervical cancer, and small cell lung cancer. Your doctor may use it to treat other types of cancer as well.

How does this drug work?

Topotecan is a chemotherapy drug. It is a type of plant alkaloid known as a topoisomerase I inhibitor. It is thought to work by blocking the action of an enzyme in cells called topoisomerase I. Cells need this enzyme to keep their DNA in the proper shape when they are dividing into 2 cells. Blocking this enzyme leads to breaks in the DNA, which leads to cell death. Because cancer cells divide more rapidly than normal cells, they are more likely than normal cells to be affected by topotecan.

Before taking this medicine

Tell your doctor…

  • If you are allergic to anything, including medicines, dyes, additives, or foods.
  • If you have ever had kidney or liver problems. This drug is cleared from the body by the kidneys and liver. Reduced function in either of these organs may result in more drug than expected staying in the body, which could lead to worse side effects. Your doctor may need to adjust your dose accordingly.
  • If you have any other medical conditions such as heart disease, diabetes, high blood pressure, scarring of the lungs (fibrosis), gout, or infections. These conditions may require that your medicine dose, regimen, or timing be changed.
  • If you are pregnant, trying to get pregnant, or if there is any chance of pregnancy. This drug may cause problems with the fetus if taken at the time of conception or during pregnancy. Men and women who are taking this drug should use some kind of birth control during treatment. It is important to check with your doctor about what kinds of birth control can be used with this medicine. In pregnant women, treatment with this drug should be used only if the potential benefit to the mother outweighs the risk to the fetus.
  • If you are breast-feeding. While no studies have been done, this drug may pass into breast milk and affect the baby. Breast-feeding is not recommended during treatment with this drug.
  • If you think you might want to have children in the future. This drug may affect fertility. Talk with your doctor about the possible risk with this drug and the options that may preserve your ability to have children.
  • About any other prescription or over-the-counter medicines you are taking, including vitamins and herbs. In fact, keeping a written list of each of these medicines (including the doses of each and when you take them) with you in case of emergency may help prevent complications if you get sick.

Interactions with other drugs

Certain drugs, including cyclosporine, ketoconazole, ritonavir, and saquinavir, may increase the amount of topotecan in the blood. Your doctor may need to watch you carefully if you are taking any of these drugs.

There may be more drugs that interact with topotecan. Check with your doctor, nurse, or pharmacist about your other medicines, herbs, and supplements, and whether alcohol can cause problems with this medicine.

Interactions with foods

No serious interactions with food are known at this time. Check with your pharmacist, nurse, or doctor about medicines, herbs, and vitamins that you are taking.

Tell all the doctors, dentists, nurses, and pharmacists you visit that you are taking this drug.

How is this drug taken or given?

Topotecan is either given by an infusion into a vein (IV) over 30 minutes or taken as capsules. The capsules may be taken with or without food, and should be swallowed whole with a glass of water. Do not break, crush, open, or chew capsules. The typical schedule is once a day for 3 to 5 days, which is usually repeated every 3 weeks. The dose and schedule will depend on a number of factors, including your body size, your blood counts, how well your kidneys are working, and the type of cancer being treated.


You may have nausea and vomiting on the day you receive this drug or in the first few days afterward. Your doctor may give you medicine before your treatment to help prevent nausea and vomiting. You will likely also get a prescription for an anti-nausea medicine that you can take at home. It is important to have these medicines on hand and to take them as prescribed by your doctor.

This drug may cause sores in the mouth or on the lips, which often occur within the first few weeks after starting treatment. This can cause mouth pain, bleeding, or even trouble eating. Your doctor or nurse can suggest ways to reduce this, such as changing the way you eat or how you brush your teeth. If needed, your doctor can prescribe medicine to help with the pain.

This drug may cause diarrhea, which in some cases may be severe enough to require you to be in the hospital. Call your doctor or nurse if you have diarrhea. Your doctor will be able to prescribe medicine to help stop the diarrhea and avoid dehydration.

Your doctor will likely test your blood often before and during your treatment, to check for the effects of the drug on blood counts or on blood chemistry. Based on the test results, you may be given medicines to help treat any effects. Your doctor may also need to reduce or delay your next dose of this drug, or even stop it altogether. Keep all your appointments for doctor visits and lab tests.

This drug can lower your white blood cell count, especially in the weeks after the drug is given. This can increase your chance of getting an infection. Be sure to let your doctor or nurse know right away if you have any signs of infection, such as fever (100.5° or higher), stomach or belly pain, chills, pain when passing urine, a new cough, shortness of breath, or bringing up sputum.

This drug may lower your red blood cell count. If this occurs, it is usually a few months after starting treatment. A low red blood cell count (known as anemia) can cause shortness of breath, or make you to feel weak or tired all the time. Your doctor may give you medicines to help prevent or treat this condition, or you may need to get blood transfusions.

This drug may lower your platelet count in the weeks after it is given, which can increase your risk of bleeding. Speak with your doctor before taking any drugs or supplements that might affect your body's ability to stop bleeding, such as aspirin or aspirin-containing medicines, warfarin (Coumadin), or vitamin E. Tell your doctor right away if you have unusual bruising, or bleeding such as nosebleeds, bleeding gums when you brush your teeth, or black, tarry stools.

Tell your doctor right away if you notice any cough, shortness of breath, or trouble breathing. This drug can cause scarring in the lungs and your doctor will need to check you right away.

Tell your doctor or dentist that you are on this drug if you are planning to have surgery or any procedure.

Do not get any immunizations (vaccines), either during or after treatment with this drug, without your doctor's OK. This drug may affect your immune system, which could make vaccinations ineffective, or could even lead to serious infections. Try to avoid contact with people who have recently received a live virus vaccine, such as the oral polio vaccine or smallpox vaccine. Check with your doctor about this.

Possible side effects

You will probably not have most of the following side effects, but if you have any talk to your doctor or nurse. They can help you understand the side effects and cope with them.


  • low white blood cell count with increased risk of infection*
  • low blood platelet count with increased risk of bleeding*
  • low red blood cell count (anemia)*
  • nausea and/or vomiting*
  • hair loss or thinning, including face and body hair

Less common

  • sores in the mouth or on the lips*
  • diarrhea*
  • constipation
  • abdominal pain*
  • loss of appetite
  • fever*
  • feeling tired (fatigue)
  • feeling weak
  • skin rash
  • shortness of breath
  • cough
  • headache


  • abnormal blood tests which suggest that the drug is affecting the liver (Your doctor will discuss the importance of this finding, if any.)
  • pain or redness at the injection site
  • numbness or tingling in the hands and feet
  • lung damage with cough, shortness of breath, fever
  • allergic reaction, with symptoms like trouble breathing, itchy welts (hives), swelling in the mouth or throat, flushing, and dizziness
  • death due to infection, diarrhea, lung failure, or other causes

*See the "Precautions" section for more detailed information.

There are other side effects not listed above that can also occur in some patients. Tell your doctor or nurse if you develop these or any other problems.

FDA approval

Yes – first approved in 1996.

Disclaimer: This information does not cover all possible uses, actions, precautions, side effects, or interactions. It is not intended as medical advice, and should not be relied upon as a substitute for talking with your doctor, who is familiar with your medical needs.

Last Medical Review: 11/29/2011
Last Revised: 11/29/2011