Myeloproliferative neoplasms, or MPNs, are a group of chronic blood cancers with the potential to rapidly progress to a more advanced stage or to an acute leukemia. Though our understanding of why these cancers occur is still evolving, we believe these MPNs can arise from a common cause: genetic alterations within the stem cell that change the way these blood cells grow and divide. Scientists are unraveling the mysteries of these rare cancers, bringing new hope for patients through research and specialized treatment.
mCancerPartner recently talked to Marie Huong Nguyen, M.D., a hematology/oncology MPN specialist at the U-M Comprehensive Cancer Center. Dr. Nguyen leads multiple clinical trials at U-M to develop new therapies in MPNs. Dr. Nguyen’s MPN and Systemic Mastocytosis Clinic focuses on the diagnosis, treatment and follow-up of patients with many different types of MPNs.
mCancerPartner: What are myeloproliferative neoplasms, or MPNs?
Dr. Nguyen: MPNs are a group of rare blood cancers. Symptoms vary depending on the subtype, but MPNs have many characteristics in common. Each arises from a problem in blood cell production that leaves the bone marrow unable to function normally. The bone marrow is where our body’s blood cells are produced. If not diagnosed and treated, MPNs can progress into an advanced stage or transform into acute leukemia.
Here is a list of the MPNs treated in our MPN and Systemic Mastocytosis Clinic at the U-M Comprehensive Cancer Center:
- Polycythemia Vera (PV)
- Essential Thrombocythemia (ET)
- Myelofibrosis (MF)
- Chronic Myeloid Leukemia (CML)
- Chronic Myelomonocytic Leukemia (CMML)
- Hypereosinophilic Syndromes (HES)
- Systemic Mastocytosis (SM) and Mast Cell Disease
We are one of only a few centers of excellence dedicated to the treatment of systemic mastocytosis in the United States, and the only one in Michigan. We are also one of the few centers with a comprehensive research program dedicated to clinical trials research in MPNs.
mCancerPartner: Are MPNs cancer, pre-cancer, or does it depend?
Dr. Nguyen: Cancer experts used to classify MPNs as disorders that had the possibility of developing into cancer. Now that we have a better understanding of genetic mutations and how these cancers arise, we know that they are cancers, not disorders, because the body is unable to regulate or control the production of abnormal cells which continue to grow independently.
MPNs can be viewed as “chronic” cancer because patients can live for a long time without severe symptoms. But once severe symptoms develop, the MPN can be life-threatening. Some MPNs can be more severe than others. The prognosis for advanced stage myelofibrosis, systemic mastocytosis, and CMML, for example, can be very serious, but there is hope: through dedicated research, the number of treatments in clinical trials has grown tremendously. And that means that timely diagnosis, treatment, and consideration for a clinical trial are critical to successfully fighting these diseases.
mCancrerPartner: Is it possible to characterize the signs and symptoms for such a broad list of cancers?
Dr. Nguyen: Yes, these diseases can share a number of symptoms which include fatigue, unintentional weight loss, night sweats, bone pain and loss of appetite. Patients can have massively enlarged liver and spleens which cause abdominal pain and difficulty eating. Patients can also have increased infections from suppressed immune systems and abnormal blood counts. In the case of mast cell disease, patients can have a characteristic rash called urticarial pigmentosa and symptoms can mimic allergic reactions.
mCancerPartner: How are MPNs treated? Can people recover?
Dr. Nguyen: The answer is a bit complex because these diseases can have different treatment and prognosis:
- PV, ET: The goal is to lower the platelet count or hemoglobin to protect against strokes, blood clots or heart attack. A very small number of these patients can progress to MF and acute leukemia but most patients can live relatively normal lives.
- Myelofibrosis: This cancer can develop suddenly or more slowly over time. Patients may have an enlarged spleen or liver, and treatment can be focused on reducing spleen and liver symptoms and controlling blood counts if they are high. Some patients may instead have low blood counts (like anemia) and require blood transfusions to survive. There is a robust clinical trials program in Dr. Nguyen’s clinic to combat myelofibrosis.
- Systemic Mastocytosis: This is a difficult disease to treat and new drugs are currently in development at U-M. To control allergy symptoms, we give antihistamines and mast cell stabilizers but we also want to give medications that kill the cancerous mast cell that causes these symptoms. This has been limited in the past to chemotherapy or other treatment that can reduce the number of mast cells; however, more recently, new medications are now available in clinical trials.
- CML: The United States Food and Drug Administration has approved drugs that will lower CML Clinical trials are currently underway for patients who have developed resistance to these medications.
For some of these blood cancers such as PV or ET, patients can live long and healthy lives using current standard therapy. But especially in the case of advanced myelofibrosis and systemic mastocytosis, patients may survive only for several years. It depends on how advanced the disease is when diagnosed, how much organ damage has taken place, and how well patients have responded to standard treatment. Some may be candidates for a bone marrow transplant, but often they are too sick, weak or elderly to qualify. Patients may also develop resistance or intolerance to treatment. Finally, though standard treatments may control the disease, they may not be curative and patients can progress or relapse over time.
That’s why new and more targeted therapies are needed through clinical trials. Now that we understand the genetic complexity of MPNs, we can target new drugs to attack the cancer cells, but without the toxicity of older, traditional therapies. Research is rapidly advancing in the field of blood cancers.
Because MPN patients may be too sick to await FDA approval of these drugs, enrolling in a clinical trial is one way patients can have early access to the newest, most cutting edge treatments that the scientific community has to offer. And some MPN research doesn’t involve taking a new drug. These can be as simple as a one-time donation of a small amount of blood or bone marrow so researchers can study these diseases. Enrolling in a clinical trial is also an opportunity to help others who may develop an MPN in the future.
Take the next step:
- Learn more about MPN clinical trials at the U-M Comprehensive Cancer Center here.
- Learn more from MPN Advocacy & Education International, which features an interview with Dr. Nguyen. Topics covered include her MPN clinical trials, as well as clinical approaches to treatment.
- Listen to Dr. Nguyen talk about the results of one of her clinical trials for patients with CML.
- Still have questions? Call the nurses at the University of Michigan Cancer AnswerLine™. They can help patients or their loved ones find a clinical trial, provide insights into the newest and latest cancer treatments, or help new MPN patients set up their first appointment in Dr. Nguyen’s Myeloproliferative Neoplasms and Systemic Mastocytosis Clinic at the Cancer Center. Feel free to call at 1-800-865-1125 or send an e-mail.
Marie Huong Nguyen, M.D, is a hematology/oncology MPN specialist at the U-M Comprehensive Cancer Center. She is a founding member of the National Comprehensive Cancer Network (NCCN) Myeoproliferative Neoplasms Panel which is charged with making recommendations for best practice in the diagnosis, treatment and management for MPNs. Dr. Nguyen’s research is focused on developing novel therapy to treat myeloproliferative neoplasms, particularly for patients with myelofibrosis and mast cell disease. Her MPN and Systemic Mastocytosis Clinic focuses on the diagnosis, treatment and follow-up of patients with many different types of MPNs.
The University of Michigan Comprehensive Cancer Center’s 1,000 doctors, nurses, care givers and researchers are united by one thought: to deliver the highest quality, compassionate care while working to conquer cancer through innovation and collaboration. The center is among the top-ranked national cancer programs, and #1 in Michigan according to U.S. News & World Report. Our multidisciplinary clinics offer one-stop access to teams of specialists for personalized treatment plans, part of the ideal patient care experience. Patients also benefit through access to promising new cancer therapies.