Chronic Myeloid Leukemia Treatment
As one of the world¡¯s top cancer centers, MD Anderson brings together internationally renowned physicians with a specialized support team to customize your care. These highly experienced experts communicate and collaborate daily, ensuring you receive comprehensive leukemia treatment.
Many of these doctors focus not just on leukemia, but on specific types of leukemia, giving them a deep level of knowledge and experience to draw on when designing treatment plans.
The goal of leukemia treatment is to put the disease into remission and ultimately cure the patient. For leukemia, complete remission usually means that the patient¡¯s bone marrow has no detectable microscopic evidence of the disease and his or her blood counts have returned to normal.
Even with normal blood counts, many leukemia patients require ongoing maintenance therapy to stay in remission. In some cases, patients in remission undergo a stem cell transplant to maintain remission.
Patients who remain in continued complete remission for an extended period of time are considered cured. This means they have an extremely low chance of recurrence. The exact amount of time it takes to be considered cured differs among leukemia types, but it is typically measured in years.
Chronic myeloid leukemia (CML) treatment plans
Treatment for CML can change depending on the phase of the disease: chronic, accelerated or blastic.
In the chronic phase, patients typically receive targeted therapy. Younger patients may also be candidates for a stem cell transplant. Some patients are also given chemotherapy to help lower elevated white blood cell counts.
In the accelerated and blastic phases, a stem cell transplant, targeted therapy and chemotherapy are primary treatments.
Chemotherapy
Chemotherapy drugs kill cancer cells, control their growth or relieve disease-related symptoms. Chemotherapy may involve a single drug or a combination of two or more drugs, depending on the type of cancer and how fast it is growing.
Learn more about chemotherapy.
Targeted therapy
Targeted therapy drugs are designed to stop or slow the growth or spread of cancer. This happens on a cellular level. Cancer cells need specific molecules (often in the form of proteins) to survive, multiply and spread. These molecules are usually made by the genes that cause cancer, as well as the cells themselves. Targeted therapies are designed to interfere with, or?target, these molecules or the cancer-causing genes that create them.
For CML, the most common targeted therapy drugs are tyrosine kinase inhibitors. These drugs are designed to interfere with a protein in the tyrosine kinase family that encourages cancer cells to multiply rapidly.
Learn more about targeted therapy.
Stem cell transplantation
A stem cell transplant (also known as a bone marrow transplant) is a procedure that replaces cancerous bone marrow with new, healthy bone marrow stem cells.?Stem cell transplants are usually given after an intense round of chemotherapy that kills the patient¡¯s existing bone marrow cells and prepares the body for transplant. Patients usually must stay in the hospital for three to four weeks after the procedure.
A stem cell transplant may be needed for patients whose leukemia has returned or has not responded to standard treatments. It may also be recommended if the patient has a high-risk form of leukemia that would make a cure with standard treatments unlikely. This treatment can be physically challenging, so it is typically not given to patients who are older or otherwise unhealthy.
Learn more about stem cell transplants.
Clinical trials
As a top-ranked cancer center, MD Anderson offers multiple clinical trials for CML. Many of these cannot be found anywhere else. Trials explore new drug combinations and new drugs, including targeted therapies and immunotherapies.
Learn more about clinical trials.
Learn more about chronic myeloid leukemia:
Chronic myeloid leukemia survivor: Why I joined a clinical trial at MD Anderson
When I was diagnosed with chronic myeloid leukemia (CML) in 2005, I was flabbergasted. There was no history of cancer in my family. I was only 42. And, I felt perfectly fine.
But routine blood work at my annual physical showed my white blood cell count was significantly elevated. It was more than four times the upper limit of the normal range. There was no question that I had leukemia. All that was left to decide was where to go for my treatment.
Why I chose MD Anderson
I¡¯m retired now. But I spent my entire career working in health care IT. Most of that involved traveling from one end of the country to the other, visiting various hospital systems. So, I was familiar with MD Anderson not just because of its stellar reputation, but also because I had been there myself in my professional capacity.
I knew that not all cancer centers are created equal. So, if I had this disease, I wanted to be seen by experts. Not necessarily because my local doctors wouldn¡¯t know what to do. Just because you go to a specialist for a reason. I wanted to see doctors who focused on chronic myeloid leukemia and treated that disease every single day.
I was living in North Carolina at the time. I knew I¡¯d need a place to stay for an extended period if I required a stem cell transplant. My wife¡¯s brother and family live in Cypress, just northwest of Houston. She¡¯d feel more comfortable staying with them than she would at a hotel. I called MD Anderson.
Why I joined a clinical trial
The first thing my doctors did at MD Anderson was conduct their own tests to confirm my diagnosis. Then, they asked if I¡¯d consider joining a clinical trial. It involved a type of targeted therapy drug called imatinib, which was still relatively new at the time. Both my local doctor and my MD Anderson care team believed it would work well for me.
I felt very strongly that others had helped and sacrificed to get me to this point. So, I owed it to the patients who would come after me to do the same. It was my turn to pay it forward. I joined the clinical trial.
Why I¡¯ll keep taking this targeted therapy drug
Back in 2005, researchers were still trying to find the optimal dose of imatinib. So, the goal of my clinical trial was to see if patients could achieve remission faster by taking a higher dosage. Eventually, the data would show that a lower dose could still get patients across the finish line; it would just take them a little longer to get there. But it would also cause fewer and less severe side effects, which was great.
Imatinib ended up working so well for me that I stayed on it long after the clinical trial ended. I briefly went off of it once, in 2020, after the results of a new international study came out. It showed that some CML patients who had achieved complete remission on this drug could stop taking it and remain cancer-free. Leukemia specialist said I was a good candidate to try going off the medication. I gave it a shot.
It was really strange not taking that pill every day. By then, I¡¯d been taking imatinib every morning for 15 years straight. But when my white blood cell count started creeping back up again just 90 days after going off the drug, I wasn¡¯t too disappointed. The medicine still worked. So, I felt like either way, I¡¯d win.?
My life today, after 20 years of targeted therapy
I¡¯ve been on imatinib now for almost 20 years. And, aside from that one three-month period in 2020, I¡¯ve only missed maybe five doses. I never needed a stem cell transplant. And, I only come back to MD Anderson every other year now for check-ups.
So far, my only limitation is that I can¡¯t eat grapefruit. Which is no big sacrifice. I¡¯m also prone to muscle cramps, I get chilled more easily, and I have loose stools. But I¡¯ve learned to manage all of those side effects with diet, occasional potassium pills, and a sweater or jacket. I have no complaints.
What I tell others about clinical trials
I realize that clinical trials are not for everybody. It¡¯s a very personal decision. You have to weigh the pros and cons and then decide if it¡¯s right for you.
But in the end, I said yes. I felt like I owed it to society as a whole, and to the people coming after me. If I could help better their lives in some small way, then it was almost a moral obligation.
Today, I look at what I did as being pretty trivial. Essentially, I took pills twice a day and had a whole lot of bone marrow aspirations. So, I¡¯m no hero. I¡¯m just lucky. But I am proud to have contributed in some very tiny way. And I¡¯m grateful to still be alive today.
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What is minimal residual disease (MRD)?
After completing a course of treatment, there are few words that sound better to a patient than ¡°complete remission.¡± It¡¯s an indication that the treatment has worked, and there is no evidence of cancer based on scans or lab tests.
However, there is a different phrase that can be somewhat confusing to patients ¨C minimal residual disease (MRD). This term is used often by physicians when treating patients with blood cancers, such as leukemia, lymphoma or multiple myeloma.
MRD refers to cancer cells remaining after treatment that can¡¯t be detected by those same scans or tests. But what exactly does it mean for patients?
To learn more about minimal residual disease, we spoke with leukemia specialist , of MD Anderson¡¯s Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Moon Shot? team. Here¡¯s what he shared.
How do you explain minimal residual disease to patients?
Minimal residual disease is a small number of cancer cells left in the body after treatment. These cells have the potential to come back and cause relapse in our patients.
In leukemia, for example, we look for response after chemotherapy treatment by looking under the microscope for cancer cells present in a bone marrow biopsy. When there are no cancer cells present, and the bone marrow is making normal cells, we call that a complete response.
However, we know that if we don¡¯t do further treatment, a portion of these patients will experience a relapse. That means there were some leukemia cells hiding that we weren¡¯t able to detect under the microscope. That is minimal residual disease, or perhaps a better term is measurable residual disease. Typically, these cells don¡¯t cause any symptoms, but they have the potential to lead to a relapse.
If we can¡¯t detect minimal residual disease under the microscope, how do we test for it?
We now have much more sensitive assays available to us that allow us to quantify MRD. These could include next generation genetic sequencing, where we can analyze bone marrow samples for genetic mutations. If there are mutations present, that means there is minimal residual disease, even though we can¡¯t see anything under the microscope.
We can also use a technique called flow cytometry, which allows us look in the same samples for abnormal proteins on the surface of cells. By determining how many cells have abnormal proteins detected, we can get a better sense of residual cancer cells. Using these new assays, we routinely try to quantify whether a patient has MRD following standard treatment.
What are the implications for a patient who has evidence of minimal residual disease after treatment?
That¡¯s difficult to say, because it¡¯s not the same across all types of blood cancers. Some patients with MRD will have different responses than others. In general, if a patient has MRD, we need to do additional treatments to work toward the best outcome. If we do nothing, we know that the residual cells will cause a relapse.
It also depends on the timing of the MRD test. In my leukemia patients, if there is MRD after the first cycle of chemotherapy treatment, it tells me that I probably need to give more treatment ¡ª either a different medication or a different course of treatment. If there is still MRD after many rounds of chemotherapy, that is an indication that the patient may need to have a stem cell transplant, when otherwise it might not have been appropriate.
Ultimately, MRD is a marker that we need to be more aggressive in our treatment to try and prevent the cells from coming back.
What can cancer researchers learn from the residual cancer cells?
We can learn a great deal. These cancers can adapt to treatment, meaning the cancer we start with is not the same as what we have after treatment. By studying the minimal residual disease, we can learn more about what is left after treatment.
That helps us to do several things. First, it allows us to modify our treatment, either by adding medications that target specific vulnerabilities in the cancer cells, including medications that are especially good at killing even residual cells, or doing a stem cell transplant, which is able to take care of residual cells.
Currently, I work with a wonderful team through the MDS and AML Moon Shot to study these residual cancer cells in order to find new vulnerabilities. Through our research, we¡¯re hoping to identify new treatments that we can use in the future to specifically eliminate minimal residual disease.
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Understanding the types of leukemia
How many types of leukemia are there? What makes each one unique, and are they treated any differently??
We went to leukemia specialist for answers to these questions and more. Here¡¯s what he shared.?
How many different types of leukemia are there?
Quite a few. Leukemia is not just a single disease. It¡¯s actually a diverse group of blood-based cancers that originate in the bone marrow.??
All leukemias fall into one of two major categories: acute or chronic.??
- Acute leukemias are blood cancers that generally appear suddenly or over a very short period of time. The classic scenario is someone who was just fine at their annual physical, but is diagnosed with leukemia a few months later.?
- Chronic leukemias generally develop much more slowly, so they¡¯re usually found in one of two ways: either a young patient is being evaluated for something else, or an older patient is having problems with recurrent infections, bleeding or abnormal lab work.??
Examples of younger patients¡¯ situations might include an athlete getting lab work done before having knee surgery, or a person having blood tests done in order to obtain a new life insurance policy. Young patients with chronic leukemia often have no symptoms, so they tend to be diagnosed unexpectedly.?
Is leukemia broken down any further, beyond those two categories?
Yes. They¡¯re further subtyped by the kind of white blood cell involved: either myeloid or lymphocyctic. The four main types of leukemia are:?
- Acute myeloid leukemia (AML) is most commonly diagnosed among people in their 60s and 70s. It only affects about 21,000 people a year, but it¡¯s among the most aggressive of all cancers. Still, there is much cause for hope. Because we can now subdivide these cancers in a way that we previously couldn¡¯t, using molecular markers, protein receptors and other attributes. This allows us to treat them using personalized targeted therapy. And there¡¯s been a ton of progress in this area over the last five years or so, with much of it led by our own along with ., and These cancers may also be treated with chemotherapy, stem cell transplants or immunotherapy. We have a large number of clinical trials available as well.
- Acute lymphocytic leukemia (ALL) is most common among children, though adults can develop it, too. It¡¯s a fairly rare cancer, affecting fewer than 10,000 people a year. At MD Anderson, we¡¯ve developed highly curable regimens for this disease, under the guidance of and Some patients don¡¯t even need a stem cell transplant to get long-term results. The type of blood cell involved (T-cell or B-cell) determines the treatment, which could be a combination of chemotherapy, immunotherapy, or targeted therapy.??
- Chronic myeloid leukemia (CML) is extremely rare, with only several thousand diagnoses each year. All patients with CML will have the Philadelphia chromosome translocation, making their leukemia ¡°Philadelphia-positive.¡± Historically, this has been a very deadly disease, with median survival rates of only 5 to 7 years after diagnosis. But now, with tyrosine kinase inhibitors like imatinib, dasatinib, nilotinib, ponatinib, and bosutinib, we¡¯re seeing patients going into complete molecular remission over time. This is one area in which oral targeted therapy has really revolutionized the lives of many leukemia patients.?
- Chronic lymphocytic leukemia (CLL) is one of the most common leukemias among adults. Previously, CLL patients had to be treated with intravenous chemotherapies. Today, oral targeted therapy agents, including BTK inhibitors and venetoclax, have been showing a lot of promise. And the vast majority of patients with this disease can now take oral chemotherapy as a part of their treatment regimens, instead of just IV-based drugs. . and . have been pioneering this combination-therapy approach here.??
What is Philadelphia-positive leukemia?
Philadelphia-positive leukemia is characterized by the translocation of chromosomes 9 and 22. This happens when a part of chromosome 9 breaks off and attaches itself to chromosome 22, which results in the generation of abnormal cells.
Philadelphia-positive was one of the first types of leukemia to prove amenable to oral chemotherapy, and the leukemia team here at MD Anderson was the first to combine that approach with immunotherapy, which has led to some very high cure rates.?Interestingly, the Philadelphia chromosome occurs in 100% of CML cases, but it¡¯s also present to a lesser extent in AML and ALL.?
What are the other types of leukemia that people should know about?
- Myelodysplastic syndrome (MDS): This used to be called ¡°pre-leukemia,¡± because it could sometimes lead to leukemia. But today, doctors understand it as a unique blood cancer in and of itself. It happens when the bone marrow produces too many immature blood cells, causing a shortage of platelets, a condition known as thrombocytopenia. MDS can be life-threatening and requires its own separate approach. is leading our investigation in this field.
- Myeloproliferative neoplasms (MPN): This used to be called myeloproliferative disorder (MPD), but various discoveries led to a better understanding of it as a family of blood cancers in and of itself, so it was renamed. MPN is usually treated with oral chemotherapy, but our is the worldwide leader in this field, and several of his clinical trials are exploring combination treatments.?
- Aplastic anemia: This is a stem cell disorder caused by bone marrow failure. It is typically treated with a combination of chemo and immunotherapy. Our leader in this area, Dr. Kadia, has been conducting clinical trials that address the unique aspects of this disease.
- Blastic plasmacytoid dendritic cell neoplasm (BPDCN): This was originally thought to be a type of lymphoma, but now it¡¯s considered its own subtype of leukemia/dendritic cell cancer. BPDCN results from an overexpression of the CD123 protein. It usually appears as a skin disorder, which is quite uncommon, but our group developed the first ¡ª and so far, only ¡ª drug to be approved by the Food and Drug Administration?(FDA) to treat it: tagraxofusp-erzs.
- Hairy cell leukemia: This is a rare form of chronic leukemia that only affects about 1,000 people a year. We¡¯ve had a lot of success in treating it with a combination of rituximab and cladribine that was pioneered here under Dr. Ravandi.
- Chronic Myelomonocytic Leukemia (CMML): This has now been recognized as it is own subtype in the MDS/MPN area, and is an emerging field of scientific breakthroughs through clinical trials, led here by
What¡¯s the one thing you want people to know about leukemia treatment right now?
Some of these leukemia types are either rare or ultra-rare, but each one has developed its own field of study due to various discoveries. Many patients can live for decades now with some of these subtypes, thanks to our personalized approach to treatment. And our common goal among all of these diseases is to maximize patients¡¯ response to treatment while also improving their quality of life.?
or by calling 1-877-632-6789.