Myelofibrosis (MF)

What is myelofibrosis?

Researchers believe that myelofibrosis is caused by abnormal stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… in the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . These abnormal cells grow quickly and cause scaring in the bone marrow and chronic inflammation. If the marrow is scarred, it can’t produce blood cells correctly or perform its normal functions. As a result, the liver and spleen try to make these cells, which in turn causes swelling and malfunction of these organs.

Relation to bone marrow failure diseases:

Myelofibrosis can develop without having had any other condition. This is called primary myelofibrosis or chronic idiopathic idiopathic: Usually refers to any condition with no known cause. myelofibrosis.

It can also develop in people who have polycythemia vera or essential thrombocytosis. This is called secondary myelofibrosis. Patients with MDS, leukemia or even lymphoma can also have fibrosis fibrosis: (fie-BRO-suss) Scarring of tissue. Fibrosis of the bone marrow is an feature seen in some types of unclassified myeldysplastic syndrome (MDS). in their bone marrows.

What causes myelofibrosis?

The exact cause of myelofibrosis in any given patient is not known. There are, however, known risk factors that increase the chances of getting myelofibrosis:

• People in their 50s, 60s or older are more likely to get myelofibrosis than younger people.
• Having another blood cell disorder, like essential thrombocythemia or polycythemia vera, as these disorders can evolve to myelofibrosis over time
• The presence of the JAK2 mutation mutation: Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow.
• Exposure to certain industrial chemicals, such as toluene and benzene benzene: A chemical that is widely used by the chemical industry in the United States to make plastics, resins, nylon and synthetic fibers. Benzene is found in tobacco smoke, vehicle emissions, and gasoline fumes. Exposure to benzene may increase the risk of developing a bone marrow failure disease. Benzene…
• Exposure to high levels of radiation

What are the symptoms of myelofibrosis?

Common symptoms include:

• Abdominal fullness, pain or feeling full before finishing a meal (because of an enlarged spleen)
• Bone pain
• Fatigue
• Itching
• Night sweats
• Bruising
• Easy bleeding
• Increased likelihood of getting an infection
• Pale skin
• Shortness of breath with exercise
• Weight loss without trying

How do you diagnose myelofibrosis?

There are a number of tests and tools doctors use to diagnose and confirm myelofibrosis. They may include:

• Physical exam and history
• Blood tests:
  • A complete blood count with a differential that looks at the total number and type of blood cells in your blood
  • A blood smear that allows your doctor to look at your blood under a microscope
• Bone marrow examination, including aspiration and biopsy to see how your bone marrow looks (required for diagnosis)
• Gene testing to look at abnormalities in your cell DNA

How do you treat myelofibrosis?

Many patients don’t need treatment at first. As the disease progresses and symptoms get worse, a number of treatments may be used:

• Supportive care Supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… with blood transfusions blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… , blood cell growth factors growth factors: Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts. Red… , androgens androgens: Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH. Androgens that may be used include: Danazol (Danacrine®) Fluoxymestrone (Halotestin®) Oxymetholone … (male hormones) and antibiotics antibiotics: The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have neutropenia. Because of their low white count, they may get infections easily and have trouble getting rid of infections. If you have neutropenia,… to increase blood counts and stop infections
• Ruxolitinib to decrease enlarged spleen size and reduce other symptoms such as fatigue
• Immune modulating drugs such as thalidomide thalidomide: Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy. Thalidomide is being studied, either as a single agent or in combination with other medicines, for treating of myelodysplastic syndromes (MDS). These clinical trials… and the related drugs lenalidomide lenalidomide: Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS… and pomalidomide, which in some patients may improve blood cell counts and relieve enlarged spleens
• Interferon, another type of immune modulating drug
• Stem cell transplants, which replace blood-forming stem cells in bone marrow with healthy stem cells from a donor
• Surgical removal of the spleen which may be needed in rare cases

Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… , also called research studies. These may offer good treatment options for some patients. These studies also help researchers better understand the value of promising new treatments or procedures.