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Myelodysplastic syndrome. Treatment and prognosis. 3
Myelodysplastic syndrome treatment and prognosis is reviewed by top expert in hematology oncology. How to use hypomethylating medications and chemotherapy? How house cell growth factors? Precision medicine for each patient with MDS. What is Rapid Heme Panel of 95 genetic tests for blood disorders? Dr. Anton Titov, MD. Myelodysplastic syndrome treatment and prognosis. Dr. Aric Parnes, MD. Video interview with top expert in hematology from Harvard Medical School. Treatment of MDS starts with improving blood production by bone marrow. Erythropoietin is used to increase red blood cells and GCSF (granulocyte colony stimulating factor) increases numbers of platelets. How molecular diagnosis of mutations in MDS helps to predict myelodysplastic syndrome progression? Second line of therapy in Myelodysplastic syndrome treatment is chemotherapy. Two medications are used to treat myelodysplastic syndrome. These medications are called hypomethylating medications. These are azacitidine (VIDAZA) and decitabine (Dacogen. Medical second opinion in MDS helps to make sure myelodysplastic syndrome diagnosis is correct and complete. Myelodysplastic syndrome treatment and prognosis are very different for each patient. Medical second opinion also helps to choose the best treatment for myelodysplastic syndrome. Medical second opinion also helps to clarify prognosis. Dr. Aric Parnes, MD. Myelodysplastic syndrome life expectancy depends on the stage of the disease and presence of specific genetic mutations. Dr. Anton Titov, MD. New genetic diagnostic test “Rapid Heme Panel” quickly and cheaply tests for 95 genetic mutations common in blood disorders. In patients with “deletion 5Q” or “5q minus” mutation a specific medication can be used. This is immune modulating medication lenalidomide (REVLIMID). Myelodysplastic syndrome survival rate depends on how successfully MDS is treated and on response to therapy. Get medical second opinion on myelodysplastic syndrome diagnosis. Myelodysplastic syndrome treatment is getting more personalized. MDS prognosis is improving for most patients. Myelodysplastic syndrome treatment and prognosis. Dr. Anton Titov, MD. Myelodysplastic syndrome (MDS) is also known as bone marrow failure disorder. This disease affects increasing number of patients. Dr. Anton Titov, MD. Perhaps risk factors for MDS increase as patients live longer. What is myelodysplastic syndrome? Who is at risk for myelodysplastic syndrome? What are the latest advances in treatment of MDS? Dr. Aric Parnes, MD Hematologist, Harvard Medical School. Myelodysplastic syndrome is “catch-all” or umbrella term that by itself does not mean much. “Myelo-” means “bone marrow”; “dysplasia” means “abnormal”. I say it’s called “syndrome” because we do not really understand it. Myelodysplastic disorder is abnormal bone marrow that produces blood ineffectively. This is really a stem cell disorder. There is a mutation in the stem cell that gives rise to all blood cells of a given lineage. After mutation happens, bone marrow is unable to make blood like it used to (before mutation occurred). The word “dysplasia” is significant for how myeloproliferative disorder is diagnosed. Dr. Aric Parnes, MD. You absolutely need a bone marrow biopsy to make this diagnosis. We need bone marrow biopsy for diagnosis of MDS because under the microscope we have to see dysplasia. Dysplasia is abnormally appearing blood. it is rarely possible to see dysplasia in the peripheral blood smear. Study of peripheral blood is not sufficient for MDS diagnosis because the end result of MDS is ineffective hematopoiesis. It means ineffective blood production. blood-making process in patients with myelodysplastic syndrome gets stuck inside the bone marrow. Dr. Anton Titov, MD. Clinical manifestation is that blood counts all decline. you get low white blood cells, low red blood cells and low platelets. Low blood counts are the cause of all the symptoms in MDS patients. if patients have low white blood cells (WBCs, leucocytes) they are susceptible to infection. Sometimes patients have low red cells (RBCs, erythrocytes), they are tired and anemic. Patients with anemia might be dizzy or short of breath. Patients with low platelets (PLTs, thrombocytes) are susceptible to bleeding. Platelets are the sticky blood cells that stop us from bleeding or bruising. The real concern is that patients with myelodysplastic syndrome have increased risk of developing acute leukemia. This risk depends on exactly what kind of genetic mutation is causing patient’s MDS. Dr. Aric Parnes, MD. We have now developed scoring systems for prognosis in myelodysplastic syndrome. Score points depend on how low is patient’s blood cell counts. Then patient gets certain number of points. Points are given for each of particular patient’s mutations after molecular diagnosis is done. Points are also assigned for specific bone marrow characteristics of the patient. Then all scores are added up. Total score allows us to predict the probability of acute leukemia in myelodysplastic syndrome patient. Scoring system also allows us to predict survival of patients with MDS. Dr. Anton Titov, MD. Dr. Anton Titov, MD. Is it possible to treat myelodysplastic syndrome at an early stage? that patients do not progress to acute leukemia. Dr. Aric Parnes, MD. The most basic treatment is supportive care. Sometimes patient has anemia and other symptoms (fatigue, shortness of breath) we can do blood transfusion. This makes patients with myelodysplastic syndrome feel better. Sometimes patient is bleeding because thrombocytes (platelets) are low. Then we can transfuse platelets. This will stop the bleeding. But that is not a long-term plan of treatment for patients with myelodysplastic syndrome. We have growth factor support as a first line therapy of patients with MDS. There are 3 three cell growth factors available. Granulocyte Colony Stimulating Factor (GCSF) is a growth factor to increase white blood cells. Erythropoietin (EPO) is a growth factor to increase red blood cells. Dr. Aric Parnes, MD. There are also two growth factors to increase platelet number. These Platelet Growth Factors are called thrombomimetics. They are: romiplostim (Nplate) and eltrombopag (Promacta / Revolade). But these growth factors do not work for very long. Sometimes myelodysplastic syndrome progresses. Dr. Anton Titov, MD. We have to move to stronger medications to treat patients. Next treatment line of MDS is chemotherapy. There are three medications that are used in myelodysplastic syndrome treatment. All three medications are very well tolerated. It is not like traditional chemotherapy that gets patients very sick. There are two broad categories of medications to treat MDS. One type is hypomethylating medications. These are azacitidine (VIDAZA) and decitabine (Dacogen). These two medications are infusion therapies. They are infused intravenously to patients over several days. Infusions are repeated every month. Dr. Aric Parnes, MD. The other broad category of myelodysplastic syndrome treatments is immune modulating medications. This is lenalidomide (REVLIMID), this is an analogue of thalidomide. Lenalidomide has a very specific indication for its use. This medication is used only in patients who have the cytogenetic abnormality “deletion 5q” (also called “5 q minus”). Myelodysplastic syndrome with deletion 5q means that a piece of chromosome #5 is missing in these patients. Patients with del(5q) do very well with lenalidomide treatment. This is a tablet, so lenalinomide is taken as a pill every day. Dr. Anton Titov, MD. Patients take lenalinomide by mouth. Dr. Parnes. That is correct. Perhaps we can emphasize that several gene mutations are known to happen in patients with myelodysplastic syndrome. Exact molecular diagnosis of mutations in MDS helps to predict progression of disease. It also helps to select personalized treatment for MDS patients. Dr. Aric Parnes, MD. That’s right. we are starting to understand to understand the precise genetic changes in myelodysplastic syndrome much better. Dr. Anton Titov, MD. Understanding molecular genetics helps to predict prognosis and to personalize treatment of MDS. We have now developed a diagnostic panel of genes. We use it to screen patients and establish precise molecular diagnosis. We call it the Rapid Heme Panel. It is a collection of 95 oncogenes. These oncogenes are important for many hematological diseases. Our Rapid Heme Panel allows us to make much more precise diagnosis than cytogenetics technology. Cytogenetics is actually a very crude test to identify gene mutations. Cytogenetics is also called karyotype. Cytogenetics test looks at actual chromosome itself. The goal is to see if there are missing segments of the chromosome. But even small missing segment of chromosome contains thousands of genes. you do not know this gene is the problem. Dr. Aric Parnes, MD. Myelodysplastic syndrome happens with 5q deletion. del(5q) is a good example. “5q-” means that a small segment of chromosome 5 is missing. The exact gene that causes the 5q- myelodysplastic syndrome was identified only several years ago. Dr. Benjamin Ebert from Harvard Stem Cell Institute found that missing gene was the RPS14. we understand the roles these genes play in myelodysplastic syndrome. There is a race now to find more targeted treatments for myelodysplastic syndrome treatment. We have specific medication (lenalidomide) to treat 5q minus myelodysplastic syndrome. But what about treatment for all other MDS mutations? That is coming. Dr. Anton Titov, MD. This is an example of “precision medicine”. It is also known as “P4 medicine”: personalized, predictive, preventive and participatory. Dr. Aric Parnes, MD. Yes, exactly. now we have three real medications to treat myelodysplastic syndrome. But there will be 100 medications to treat myelodysplastic syndrome in the future. Dr. Anton Titov, MD. Myelodysplastic syndrome treatment and prognosis. Video interview with top hematology expert. New genetic tests and medications to treat MDS. Lenalinomide. Myelodysplastic syndrome treatment and prognosis. Treatment of MDS starts with improving blood production by bone marrow. How growth factor, hypomethylating agents and chemotherapy is personalized for each patient with MDS?
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