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Mayo Clinic Q&A: Treatment-free Remission For Chronic Myeloid Leukemia

DEAR MAYO CLINIC: My 32-year-old daughter was recently diagnosed with chronic myeloid leukemia. It's a lot to take in. We've heard about treatment-free remission. That sounds hopeful. What does that mean? How is it achieved?

ANSWER: It is a lot to take in. Some background information may help you and your daughter understand this diagnosis and potential treatment.

Leukemias are cancers of the blood cells. Chronic myelogenous leukemia (CML) is an uncommon type of cancer of the bone marrow, which produces blood cells. "Myelogenous" refers to the type of blood cells this leukemia affects. "Chronic" means this cancer is more slowly progressing than other severe forms of leukemia.

CML is a genetic problem that causes overproduction of a protein that allows some blood cells to grow out of control. But the gene change that causes CML is not hereditary. Parents do not pass the gene to their children.

According to the American Cancer Society, nearly 9,000 people are diagnosed with CML each year in the U.S. This type of leukemia typically affects older adult males, but both men and women can be diagnosed with CML. It is rarely seen in children.

Some people with CML may experience symptoms such as bone pain, bleeding easily and feeling full after eating a small amount. But many people do not have symptoms until later stages. It is diagnosed with a blood test.

People first diagnosed with CML likely will begin targeted treatment to eliminate blood cells that are overproducing a protein called tyrosine kinase. Medications called tyrosine kinase inhibitors (TKIs) are first-line treatments. Each medication has its own side effects, such as swelling or nausea, that differ for each patient and are discussed in the early stages of choosing treatment. At later stages, other treatments may include chemotherapy or a bone marrow transplant.

Patients we consider for treatment-free remission include those in a low-risk category who take one kind of medication during their treatment course and are in what's called "molecular remission" for at least two years. Molecular remission means the gene that causes CML cannot be found in the patient's blood or bone marrow.

During and after treatment, regular tests are necessary to monitor the patient's blood. When no leukemia is detected, patients may be able to end treatment. Even patients in treatment-free remission must be monitored regularly, for life, and work closely with a hematologist or oncologist. That means testing monthly, progressing to every other month, then every three months, to every six months.

If a patient were to relapse, the disease would be discovered early, and treatment using the same kind of medication would be resumed. Studies over the last 10 to 15 years have shown when restarting medication early, the chance of achieving remission is almost 100%.

There are certain criteria before stopping TKI treatments. This is important as it affects women and family planning. Managing CML, pregnancy and childbirth is challenging but possible with the help of the health care team.

At Mayo, we started this treatment-free remission approach in 2015. Many patients have been off treatment for years. For more information, visit the CML Patient Education website. — Kebede Begna, M.D., Hematology, Mayo Clinic, Rochester

Mayo Clinic Q&A is an educational resource and doesn't replace regular medical care. Email a question to MayoClinicQ&A@mayo.Edu. For more information, visit www.Mayoclinic.Org .

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A Clinical Trial Saved My Life: Surviving Chronic Myeloid Leukemia

Mel Mann shares his personal story of survival after being diagnosed with chronic myeloid leukemia in 1995. Given only three years left to live, Mel was shocked and worried about his young daughter and wife. The only possibility for a cure was the difficult-to-find bone marrow transplant. A chance encounter at a donor drive opened Mel's eyes to the possibility of clinical trials. He enrolled in one in 1998 that gave him access to cutting-edge medicine unavailable to most patients.

Now, almost 28 years later, Mel is still alive thanks to that experimental treatment. He advocates strongly for clinical trials not just because they saved his life, but because they offer hope to patients who have run out of options. As he says, clinical trials give you "the opportunity to get tomorrow's medicine today." By bringing trials to more diverse and rural populations, more people can potentially benefit. Mel wants clinical trials offered as a standard part of informed consent so that other families may be spared the grief of a terminal prognosis. Read on for Mel's perspective on clinical trials as a survivor. 

This interview has been edited and condensed for clarity.

I think clinical trials are important because they allow you to get tomorrow's medicine today. 

In January 1995, I was diagnosed with chronic myeloid leukemia. I was stationed in the military in the Michigan area around Detroit and I went to the doctor because I had back pain and fatigue. The doctor diagnosed me with chronic myeloid leukemia and gave me 3 years to live. That was the prognosis. And, I was in shock because I had a 5-year-old daughter [at the time], and it was going to be hard on my wife and my family. 

Dealing with my diagnosis

I was just in shock, and he said the only possible cure was a bowel marrow transplant. And it was hard to find a donor. No one in my family matched. So I did a bunch of [bone marrow] drives around the country in the military, at, colleges, churches, [etc].

I was doing a a bone marrow drive down in Columbus, Georgia and a gentleman [told me] he had hairy cell leukemia and was near death. He had tried a clinical trial, and it worked for him and suggested that I do the same thing. They're saving lives.

How clinical trials have changed

Back in 1998, when I started doing clinical trials, it was a lot different than what it is now. The goal of clinical trials now is to bring the clinical trial to the patient, which is better because it helps with a more diverse clinical trial participant community. It helps the rural people [and] it helps the people who cannot afford to travel back and forth.

Why I advocate for clinical trials

So [that creates] a bigger field of people to participate and have an opportunity to take advantage of this state-of-the-art treatment. I'm the world's longest-living person out of hundreds of thousands of people on the [chronic myeloid leukemia] drug I received, and the drug has helped millions of people [with] different types of cancer. And, so I had a good outcome [and] my daughter grew up and she became a doctor. It saved my life and that's why I advocate so strongly, about clinical trials. 

I only [had] 3 years to live, and it's almost 28 years later, and I'm still here, and I'm doing well. So I strongly recommend that everyone is informed about clinical trials as part of their treatment.

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