Myelofibrosis Clinical Trials

Myelofibrosis clinical trials are studies that help advance cancer research. These trials aim to hopefully have more therapies and treatment combinations approved in order to improve outcomes for myelofibrosis patients. Myelofibrosis clinical trials can help provide the most favorable treatment for some patients. Myelofibrosis is a bone marrow disorder that affects the body by disrupting the normal production of red blood cells. It occurs when blood stem cells develop a genetic mutation, and the mutation is passed on to new cells with the replication and division during cell production. Symptoms of myelofibrosis include:

• Fatigue
• Pain below the left ribs
• Easy bruising
• Easy bleeding
• Night sweats
• Fever
• Bone pain
• Anemia

Myelofibrosis Diagnostic Tests

Myelofibrosis can be diagnosed using a variety of tests including a physical exam, blood tests, imaging tests, bone marrow examination, and gene tests.

• Physical exam: your doctor will check vital signs like pulse, blood pressure, lymph nodes, spleen and abdomen.
• Blood tests: a complete blood count will show low levels of red blood cells, which is common in people with myelofibrosis. White blood cell and platelet counts will be checked as well.
• Imaging tests: X-rays and an MRI may be used.
• Bone marrow examination: bone marrow biopsy and aspiration can confirm a myelofibrosis diagnosis.
• Gene tests: a sample of blood or bone marrow can be analyzed for gene mutations associated with myelofibrosis.

Myelofibrosis Treatment Options

Treatment options will vary depending on each case. For myelofibrosis patients who aren’t showing signs of anemia, an enlarged spleen, or other complications, treatment won’t be necessary. Depending on the complication, different treatment options are recommended:

Patients with anemia:

• Blood transfusions: transfusions can increase the red blood cell count to ease uncomfortable symptoms associated with anemia.
• Androgen therapy: consumption of the male hormone androgen can promote red blood cell production.
• Thalidomide and related medications: Medications such as thalidomide, lenalidomide, and pomalidomide can improve blood cell counts and help with an enlarged spleen.

Patients with enlarged spleen:

• Targeted drug therapy: A drug that targets a gene mutation specific to myelofibrosis can help reduce an enlarged spleen. These drug therapies are being studied in clinical trials.
• Chemotherapy: May reduce the size of an enlarged spleen, as well as reduce symptoms
• Surgical remover of spleen (splenectomy): If the enlarged spleen is causing much discomfort and other treatments do not work, the spleen can be removed.
• Radiation therapy: High-powered beams are used to target and kill cancer cells.

Other treatment options available for myelofibrosis treatments include:

• Bone marrow transplant: This transplant replaces diseased bone marrow with healthy blood stem cells.
• Supportive care: Also known as palliative care, this treatment focuses on relieving pain and other symptoms in patients with serious illness.

Clinical trials: Clinical trials can use a combination of these therapies as well as new therapies. Clinical trials help advance research to improve treatment, prevention, and diagnostic options for myelofibrosis. As of now, there are 56 myelofibrosis clinical trial studies recruiting in the United States.

Resources:

https://www.mayoclinic.org/