There are now many treatment options for people with chronic myelogenous leukemia.

Chronic myelogenous leukemia (CML), also known as chronic myeloid leukemia, is a cancer of the white blood cells. CML begins in the bone marrow, where blood cells are made. This cancer is caused by a mutation (change) in the genes of the bone marrow cells. Human cells contain 46 chromosomes. These coiled structures contain our genes, the blueprints for the body and its functions. In CML, the ABL gene on chromosome 9 joins to the BCR gene on chromosome 22. They form a new BCR-ABL “fusion gene” that changes chromosome 22. This altered chromosome is called the Philadelphia (Ph) chromosome.

With a Ph chromosome, white blood cells grow and divide too quickly, crowding out healthy cells in the bone marrow. From the bone marrow, CML cells enter the bloodstream and can spread throughout the body.

There are three phases of CML: chronic (the leukemia is slow growing and responds more easily to treatment), accelerated (cancer cells start to grow more quickly) and blast crisis (leukemia is fast growing and treatment is needed right away). Almost everyone who gets CML has the Ph chromosome. The BCR-ABL fusion gene mutation happens gradually during a person’s lifetime. It cannot be passed on from parents to children.

Often, the symptoms of CML are not very specific: feeling run down or tired, night sweats, loss of appetite or easy bleeding. Doctors may find an enlarged spleen (located below the ribs on the left side) when examining a person with CML. A spleen can enlarge because it is overactive in removing and destroying abnormal blood cells. The best way to diagnose CML is with a blood or bone marrow test.

Since 2001, when the U.S. Food and Drug Administration (FDA) approved the first targeted treatment for CML, there has been a revolution in the way doctors treat this cancer. Unlike chemotherapy, targeted treatments focus on specific cell mechanisms thought to be important for cancer growth. Because of this focus, targeted treatments tend to spare healthy cells and cause less severe side effects than chemotherapy. Now, there is a large and growing number of people with CML who are in remission (no signs or symptoms of cancer) and doing well.

Among the many treatment options available for CML are:

Imatinib (Gleevec). This drug dramatically changed the outlook for people with CML. In most patients, imatinib, taken as a pill every day, makes the cancer go into longterm remission. It works by shutting down the function of an abnormal protein produced by the Ph chromosome. This protein causes CML cells to grow and divide. In eight out of 10 people treated with imatinib, the Ph chromosome disappears completely. Some patients treated with imatinib have now been in remission for more than 10 years. However, this drug is not effective for all patients, so doctors sometimes must turn to other treatments.

Nilotinib (Tasigna) and dasatinib (Sprycel). Like imatinib, nilotinib and dasatinib are taken in pill form and treat CML by blocking the cells’ growth and division. When used as a first-time treatment, both drugs have been shown to be more effective than imatinib for some patients. These medications can also benefit people whose CML stops responding to imatinib and those who cannot continue on imatinib because of its side effects.

Bosutinib (Bosulif). Similar to nilotinib and dasatinib, bosutinib was developed for people with CML who cannot benefit from taking imatinib. Like all of these targeted treatments, bosutinib causes side effects that must be carefully monitored by patients and their doctors. Symptoms include nausea, diarrhea or vomiting; low red and white blood cell counts due to an “overcorrection” of blood cell production in the bone marrow; and problems with liver function.

Ponatinib (Iclusig). Approved recently by the FDA, ponatinib controls CML in a slightly different way than the other drugs. Because of this, ponatinib can be beneficial even for CML with a specific gene mutation known as T315I. This mutation makes CML cells resist treatment with imatinib, nilotinib, dasatinib and bosutinib. Doctors are very encouraged by having this option available. Ponatinib benefits patients for whom these other treatments did not work and allows most to go into remission.

Chemotherapy. Older medications can still be useful, such as hydroxyurea (Droxia, Hydrea and others) to control high blood cell counts. Doctors now mostly prescribe hydroxyurea for CML only if all other treatments have stopped working. Often, the vitamin folic acid is taken with this drug to decrease some of its side effects, such as nausea and vomiting.

Immune system boosters. Before imatinib was approved, the standard treatment for CML was interferon. There are several types of interferon, which block the division of cancer cells and can slow tumor growth. Researchers are studying a number of other medications designed to strengthen the body’s immune response to CML, including vaccines and T-cell therapy. T-cell treatment “reprograms” a patient’s or donor’s immune cells so they can recognize and destroy CML cells.

Stem cell/bone marrow transplant. This treatment may be used for some people whose CML advances or if several medicines have not been effective. To be eligible for a transplant, patients need a well-matched donor—ideally a close relative such as a brother or sister. Because the risks associated with bone marrow transplants increase with age, this procedure is usually recommended for younger patients who have already tried a number of other treatments that either did not work or caused side effects that could not be tolerated.

Because so many people with CML are treated for years and even decades, they face a number of challenges, including coping with the side effects of their medication and staying on a drug for a long time (discussed on page 10). Doctors now have a great deal of experience helping patients manage their symptoms.

Fatigue (extreme tiredness not helped by sleep) is one of the most common side effects for people living with CML. In some cases, doctors can lower the dose of the drug, as long as it does not make the treatment less effective. If you are experiencing fatigue, talk to your doctor about whether taking a smaller dose is right for you.

There are a number of other tips for reducing fatigue:
• Take several short naps or breaks.
• Take short walks or do some light exercise, if possible.
• Try easier or shorter versions of the activities you enjoy.
• Ask your family or friends to help you with tasks you find difficult or tiring.
• Save your energy for things you find most important.
• Talk to an oncology social worker or oncology nurse.

These professionals can also help you manage fatigue. They can work with you to manage any emotional or practical concerns that may be causing symptoms and help you find ways to cope.

Digestive tract symptoms are also common but usually not severe: mild nausea, occasional vomiting, loss of appetite or diarrhea. Your doctor can prescribe medications for digestive tract side effects. The following tips also may help.

Nausea and vomiting:
• Avoid food with offending odors, as well as overly sweet, greasy, fried or highly seasoned food.
• Eat meals cold or at room temperature, which often makes food more easily tolerated.
• Nibble on dry crackers or toast. These bland foods are easy on the stomach. Having something in your stomach when you take medication may help ease nausea.

Loss of appetite:
• Because it’s important to maintain your weight, eat small meals throughout the day. That’s an easy way to take in more calories and protein.
• To keep from feeling full early, avoid liquids with meals or take only small sips (unless you need liquids to help swallow). Drink most of your liquids between meals.
• Be as physically active as you can. Sometimes, taking a short walk an hour or so before meals can help you feel hungry.
• Keep high-calorie, high-protein snacks on hand such as hard-boiled eggs, peanut butter, cheese, ice cream, granola bars, liquid nutritional supplements, puddings, nuts, canned tuna or trail mix.
• Eat your favorite foods any time of the day. For example, if you like breakfast foods, eat them for dinner.

Diarrhea:
• Drink plenty of water. Ask your doctor about using drinks such as Gatorade which provide electrolytes as well as liquid. Electrolytes are body salts that must stay in balance for cells to work properly.
• Over-the-counter medicines such as loperamide (Imodium A-D and others) and prescription drugs are available for diarrhea but should be used only if necessary. If the diarrhea is bad enough that you need medicine, discuss it with your doctor or nurse.
• Avoid sweetened foods and alcohol.
• Choose fiber-dense foods such as whole grains, fruits and vegetables, all of which help form stools.

Skin rash. People with CML who are affected by skin rash usually start to experience this side effect within the first week or two of treatment. But the rash is often mild and can be treated with antihistamines or, rarely, topical steroids. Before you treat a rash with over-the-counter medications, talk to your doctor. It’s important to closely monitor this and other symptoms to make sure they do not worsen and affect your health or quality of life.

Taking medication over many years means maintaining a schedule and staying on track. That’s especially important for people living with CML. Doctors have found that even a 10 percent reduction in how often patients take their drug can lower the treatment’s effectiveness. Patients give different reasons for not sticking with their treatment plan: “I’m feeling good, so maybe I don’t need this drug” or “This drug makes me feel nauseated, so I don’t take it as often as I should.”

With the number of treatment options available, your doctor can find the right drug for you to best manage your CML. With the help of your health care team and the following tips, you should be able to stay on your medication and stay healthy:

Get a plan on paper. When you start taking your pills, have your doctor provide written instructions that you can take home and review.

Fit the drug into your usual routine. Check with your doctor about the best way to take your pills, including the time of day, before or after meals, or with food. It is always best to swallow your pills with water. If you are taking many pills, ask your doctor or pharmacist how best to space your pills apart. Some pills cannot be taken together, so check with your health care team to learn the best way to take your pills.

Take your prescribed pills the same time every day.

Consider using a pill sorter (available at drug stores) to sort your week’s medications. Fill it with your week’s supply of medicine.

Place reminders on your calendar to refill your prescriptions several days before they run out.

If you are going on a trip, be sure to have plenty of medication with you, including extra pills in case your stay is extended or your return is delayed. Keep your medication with you at all times, in your personal bag, rather than checking it with luggage, which could be lost. If you are on a road trip, make sure your pills don’t sit for long periods in a hot car trunk or in a locked car.

Know what to do if you miss a dose. Discuss this with your health care team. Do not take a double dose to make up for a missed one.

If you have trouble swallowing pills, ask your oncologist, nurse or pharmacist for suggestions on what to do. Your health care team can advise you on the best method to use.

Know what to expect. Medications often have side effects. Review with your health care team the expected side effects. If you experience any unusual or unexpected symptoms, call your doctor immediately. Do not try to treat these side effects on your own.

Do not stop taking your medication without talking to your doctor.

As you manage your CML, it’s important to remember that you are a consumer of health care. The best way to make decisions about health care is to educate yourself about your diagnosis and who is on your health care team, including nurses, social workers and patient navigators.

Here are some tips for improving communication with your health care team:

Start a health care journal. Having a health care journal or notebook will allow you to keep all of your health information in one place. You may want to write down the names and contact information of the members of your health care team, as well as any questions for your doctor. Keep a diary of your daily experiences with cancer and treatment. You can separate your journal or notebook into different sections to help keep it organized.

Prepare a list of questions. Before your next medical appointment, write down your questions and concerns. Because your doctor may have limited time, you should ask your most important questions first, and be as specific and brief as possible.

Bring someone with you to your appointments. Even if you have a journal and a prepared list of questions or concerns, it’s always helpful to have support when you go to your appointments. The person who accompanies you can serve as a second set of ears. He or she may also think of questions to ask your doctor or remember details about your symptoms or treatment that you may have forgotten.

Write down your doctor’s answers. Taking notes will help you remember your doctor’s responses, advice and instructions. If you cannot write down the answers, ask the person who accompanies you to do that for you. If you have a mobile device, you can use it to take notes. Writing notes will help you review the information later.

Record your visit if your doctor allows it. Recording the conversation with your doctor gives you a chance to hear specific information again or share it with family members or friends.

Q. Should I be taking the new oral drugs, which may not work for me, or consider getting a bone marrow transplant?

A. This is one of the most important decisions doctors make in treating CML. Most patients do benefit from the newer oral treatments, and there are several to choose from. If none of the new therapies works or if a patient has moved on to a second or third drug and the cancer is still resisting treatment, doctors then consider recommending a bone marrow transplant. The lesson we have learned is not to wait to do a transplant until the cancer advances greatly.

Doctors need to monitor their patients’ progress and make careful decisions along the way. The transplant option is a good one, especially for a younger person with a matched donor. However, most people with CML are middle aged or older, and transplants pose more challenges for them. Although we have made great advances in managing side effects and can reduce the risk of infection and other complications, bone marrow transplants can be difficult procedures.

Q. I took ponatinib [Iclusig] for several weeks, but because of the side effects I experienced, I had to stop using it. I am now waiting to begin on a lower dose. Will it still be effective?

A. So far, relatively few people have used this medication, but we do know that for those who cannot tolerate the starting dose, which is generally 45 milligrams (mg), it is effective at lower doses. We learned this from clinical trials that were conducted to study the safety and effectiveness of ponatinib. For people who experience side effects, the best strategy is to lower the dose and try using it again, under their doctors’ care.

Q. I’m feeling so tired all the time, but I’m not sure if it’s because of the CML drug I’m taking or some other reason. What should I do?

A. You bring up a good point: some symptoms may not be related to the drug, which is why it is so important to understand the cause of your fatigue. In some cases, it may be anemia due to a low red blood cell count or an infection, for example. So you need to discuss your symptoms with your doctor or nurse. They can treat the underlying cause with medication. Once the cause of your symptoms has been treated, there are a number of things you can do to prevent or reduce fatigue. Follow the tips listed on page 7 and consider using “complementary” techniques such as yoga, meditation, guided imagery or tai chi. All of these methods encourage deep breathing. Oxygen helps the brain and body function better.

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This e-booklet was made possible by Bristol-Myers Squibb.

The information presented in this publication is provided for your general information only. It is not intended as medical advice and should not be relied upon as a substitute for consultations with qualified health professionals who are aware of your specific situation. We encourage you to take information and questions back to your individual health care provider as a way of creating a dialogue and partnership about your cancer and your treatment.