The drugs used to treat myelofibrosis depend on several factors including the symptoms of the patient, presence of gene mutations, and which type of blood cells are being produced at abnormal rates. Myelofibrosis is a rare blood disease in which bone marrow scarring is caused by the abnormal production of blood cells. Stem cells are not able to mature and they build up within the bone marrow. There is only one curative treatment for myelofibrosis, which is an allogenic stem cell transplantation, which injects healthy stem cells from a donor to repair the bone marrow.
Other treatments are used to help with the symptoms that the patient is experiencing. In the early stages, some patients do not experience any symptoms at all. These patients often do not receive treatment until symptoms arise. Symptoms myelofibrosis patients experience include:
- Unexpected weight loss
- Night sweats
- Loss of appetite or feeling of fullness
- Easy bleeding and bruising
To determine which drug should be used to treat myelofibrosis, the doctor takes several factors into considerations like age, symptoms, and gene mutations. A gene mutation test on the bone marrow or blood sample can reveal mutations in various genes related to myelofibrosis. A mutation in JAK1/JAK2 is found in 50% of all myelofibrosis cases. Those who have tested positive for JAK1/JAK2 will be eligible to receive ruxolitinib (Jakafi), an FDA approved targeted therapy. This treatment binds to the cancerous cells with the mutation and attacks them. Targeted therapies can be more effective than other standard treatments and they avoid healthy cells, which can lead to less side effects for patients.
Other Drugs used to treat myelofibrosis include:
- Inrebic (fedratinib), the only other FDA approved targeted therapy for myelofibrosis
Standard treatments used for myelofibrosis patients include:
- Active surveillance/watchful waiting
- Allogenic Stem Cell Transplantation
Clinical trials are also an option for many patients, which utilize many of the available drugs for myelofibrosis and are available for enrollment. Before therapies can be approved by the FDA, they must go through the different phases of clinical trials to ensure they are safe and are effective. Therefore, clinical trials provide early access to treatment studies for patients. Talk to your doctor if you are interested in learning if a clinical trial is right for your case.