Myelofibrosis Treatments

There are many treatment options for myelofibrosis patients to consider because cases can vary for everyone. The treatment decision will be made by the doctor after considering several factors that include age, symptoms, and which type of blood cells are being affected.

Myelofibrosis belongs to a group of blood disorders called Myeloproliferative Neoplasms (MPNs) that cause scarring of the bone marrow due to abnormal production of red blood cells, white blood cells, and platelets. Tests for blood and bone marrow can be used to diagnose any of the six MPNs, which include:

• Chronic myelogenous leukemia
• Polycythemia vera
• Primary myelofibrosis
• Essential thrombocythemia
• Chronic neutrophilic leukemia
• Chronic eosinophilic leukemia

Primary myelofibrosis (PMF) refers to patients who have had no prior MPNs or autoimmune disorders. Secondary myelofibrosis (SMF) occurs when a patient has had a previous disorder, usually essential thrombocythemia or polycythemia vera.

Roughly half of all myelofibrosis cases have a gene mutation in the JAK2 protein, which has a role in the growth of cells. If the mutation is present, the case is likely to be more aggressive than those who do not have it, due to abnormal JAK2 mutated cells growing faster than others. The JAK2 V617F test is used when someone is suspected of having an MPN. The test can verify if there is a JAK2 mutation present, which confirms the diagnosis of myelofibrosis.

Myelofibrosis Treatments

Myelofibrosis treatments vary because the disease can be aggressive in some patients, while indolent in others. Patients who do not experience symptoms, or are asymptomatic, are often under active surveillance, also known as watchful waiting. This is because aggressive treatments may cause more damage to the body than it would help the disease.

Allogenic Stem Cell Transplantation (ASCT) remains the only curative treatment for myelofibrosis patients. Healthy stem cells are taken from a donor and injected into the patient to grow into healthy blood cells. However, the procedure can take time for recovery and has side effects. Stem cell transplants are sometimes too risky for some patients, a physician would make the final decision. For ineligible patients, they will receive palliative care to treat their symptoms and improve quality of life. These treatments include:

• Chemotherapy
• Splenectomy
• Radiation therapy
• Immunotherapy
• Targeted therapies
• Clinical Trials

Clinical Trials

Ruxolitinib, or Jakafi, is the first FDA approved myelofibrosis treatment for patients. Ruxolitinib is a targeted therapy that locates cells with the JAK2 mutation and attacks them. Therefore, not as many healthy cells will be damaged when compared to standard treatments. This treatment can only be used for those who have tested positive for a JAK2 mutation. Targeted therapies prevent abnormal cells from growing and reproducing, while ignoring healthy cells.

New treatment methods and options are continuously being evaluated in clinical trials. Clinical trials are available for enrollment for myelofibrosis patients around the United States. These trials provide early access to treatments because they have not yet been approved by the FDA and are not yet widely used. Ruxolitinib was approved in 2019, and new treatments will be available once they are studied and approved. Talk to your doctor to see if clinical trials could be beneficial for your myelofibrosis case.