What are Treatments for Myelofibrosis?

Myelofibrosis is a type of cancer that affects the bone marrow's ability to generate new blood cells caused by scar tissue within the marrow. This condition can lead to symptoms like weakness and fatigue. Common findings in lab tests or physical examination include anemia and an enlarged liver and spleen. 

Myelofibrosis is a chronic disease that can impact in quality of life. However, with appropriate management and treatment, symptoms can be controlled. Treatment options for myelofibrosis include medication, blood transfusions, stem cell transplants, and other supportive therapies.

What Medications are Approved for Myelofibrosis?

Medications are often used to manage symptoms and improve the quality of life for patients with myelofibrosis. But they are also helpful in eliminating abnormal cells or targeting specific sites of the cell that may be causing myelofibrosis to spread. 

• Ruxolitinib (Jakafi): This was the first drug approved by the FDA specifically for myelofibrosis. It works by blocking the JAK1 and JAK2 enzymes, which regulate blood and immune cell production. Ruxolitinib can reduce spleen size, alleviate symptoms, and improve survival in patients with myelofibrosis.
• Fedratinib (Inrebic): is a JAK inhibitor approved by the FDA for treatment of adult patients with intermediate-2 or high-risk primary or secondary myelofibrosis
• Momelotinib (Ojjaara): Is also a JAK inhibitor which function is to increase the red blood cell production and reduce inflammation that could lead to more fibrosis in the bone marrow. To know more about momelotinib we have a comprehensive article here. 
• Pacritinib (Vonjo): is for patients who have myelofibrosis and low platelet levels (thrombocytopenia)
• Interferon-α: This therapy can help reduce the size of the spleen and potentially stop the bone marrow from becoming fibrotic. It's often used in younger patients with a lower risk of disease progression. To know more about interferons role in myelofibrosis click here. 
• Hydroxyurea: This chemotherapy drug can help control an enlarged spleen and high white blood cell counts in myelofibrosis.

When Should Myelofibrosis Patients Receive a Blood Transfusion?

Blood transfusions can help alleviate anemia symptoms like weakness, fatigue, easy bruising, or difficulty breathing. Transfusions of red blood cells or platelets can help alleviate symptoms and prevent bleeding problems. However, not all patients will receive transfusions. The indication for it depends on lab values and other chronic conditions for example, if the hemoglobin levels are less than 5 g/dL and if a patient has risk factors like heart failure, heart disease, arrhythmia, and COPD or lung failure, they can receive a transfusion if their hemoglobin is less than 6 g/dL.

Can Myelofibrosis Be Cured With a Stem Cell Transplant? 

Stem cell transplant, also known as bone marrow transplant, is the only treatment currently known to potentially cure myelofibrosis. This procedure involves replacing your diseased bone marrow with healthy bone marrow from a donor. However, stem cell transplants come with significant risks, including graft-versus-host disease and infections, and are typically reserved for younger patients and those with severe disease.

What are the Supportive Therapies for Myelofibrosis? 

Supportive therapies aim to relieve symptoms and improve quality of life. These may include:

• Erythropoiesis-stimulating agents: These drugs can help stimulate the bone marrow to produce more red blood cells
• Iron chelation therapy: This treatment is used to remove excess iron from the body, a common problem in patients who require frequent blood transfusions
• Immunomodulators: like thalidomide, lenalidomide and pomalidomide can be used to improve anemia and spleen size, however they require close monitoring because of the side effects and withdrawal reactions. 
• Androgens: hormones like nandrolone, fluoxymesterone, methandrostenolone, and oxymetholone improve anemia in 30% to 60% of patients. They are particularly helpful in patients who don’t have a spleen and can also help improve low platelet levels 

Want to Learn More About Myelofibrosis?

Expanding knowledge for all patients and discovering the best time to start treatment is possible thanks to research. And you can actively participate by staying informed with the latest myelofibrosis news, recruiting trials, surveys and more! 
 

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Sources: 

• Diagnosis and evaluation of prognosis of myelofibrosis: A British Society for Haematology Guideline
• Myelofibrosis
• How I treat myelofibrosis
• Use of Interferon Alfa in the Treatment of Myeloproliferative Neoplasms: Perspectives and Review of the Literature
• Bone marrow fibrosis in myelofibrosis: pathogenesis, prognosis and targeted strategies