There is not one treatment that is effective for all myelofibrosis (MF) patients. Patients have varying symptoms and circumstances that require different treatment options. Some MF patients remain symptom-free for many years and do not require immediate treatment. All MF patients, however, need to be closely monitored.
There is no drug therapy that can cure MF. The only potential cure for MF is allogeneic stem cell transplantation. But this procedure is risky for older patients and those with other health problems. Because MF primarily affects older adults, a stem cell transplantation is not a treatment option for most MF patients. For most people with MF, treatment remains aimed at controlling disease symptoms and complications, enhancing quality of life and extending survival.
Low-Risk Myelofibrosis (MF) Patients
Asymptomatic Patients. Patients who are symptom-free (called “asymptomatic”) and do not have signs of anemia, an enlarged spleen or other complications, are generally not treated. Some people remain stable and symptom-free for many years. However, these low-risk patients need to be monitored closely with regular medical checkups and examinations to detect any signs and symptoms of disease progression.
Symptomatic Patients. For low-risk patients with symptoms, treatment may include
- Ruxolitinib (Jakafi®) or
- Interferon alfa (Intron A, Roferon-A, Pegasys), although a situation in which these drugs are used may be best in the setting of a clinical trial
Intermediate-Risk and High-Risk MF Patients
Intermediate-1 Risk (INT-1) Patients. Treatment may include:
- Ruxolitinib (Jakafi®) or
- Allogeneic stem cell transplantation (in some cases)
Intermediate-2 Risk (INT-2) and High-Risk Patients. Treatment may include:
- Ruxolitinib (Jakafi®) or
- Fedratinib (Inrebic®)
Doctors will determine on a case-by-case basis those who are candidates for allogeneic stem cell transplantation. For those eligible, the stem cell transplant may begin shortly after diagnosis. Patients who are not candidates for allogeneic stem cell transplantation are encouraged to participate in clinical trials.
For information about the drugs listed on this page, visit Drug Listings.
Stem Cell Transplantation
Allogeneic stem cell transplantation is the only current treatment with the potential to cure MF, but it also carries a high risk of life-threatening side effects. It is usually risky for older patients and those individuals with other health problems. Therefore, it is recommended for younger patients with no other pre-existing health problems. However, allogeneic stem cell transplantation can be used in older people when medically appropriate. Whether or not a patient is a candidate for transplantation is determined by medical indications and the availability of a donor.
Reduced-intensity or “nonmyeloablative” allogeneic stem cell transplantation is a type of transplant that is being used to treat some patients with MF. Compared with standard allogeneic stem cell transplantation, reduced-intensity transplant delivers lower doses of chemotherapy drugs and/or radiation to the patient in preparation for the transplant. This approach may benefit older and sicker patients who are unable to tolerate high doses of chemotherapy drugs used in standard allogeneic stem cell transplantation.
Patients should talk with their doctors about whether stem cell transplantation is a treatment option for them.
Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. Patient participation in clinical trials is important in the development of new and more effective treatments for MF and may provide patients with additional treatment options.
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Some examples of treatments being studied include:
- JAK Inhibitors are showing effectiveness in reducing spleen size and symptoms such as night sweats and fatigue, and possibly improving anemia. These new treatments include pacritinib (SB1518) and NS-018.
- Histone deacetylase (HDAC) inhibitors play an important role in the regulation of gene expression. A clinical study of panobinostat (Farydak®), which is approved by the FDA for the treatment of multiple myeloma), in combination with ruxolitinib (Jakafi®) in patients with MF is ongoing. Pracinostat is another HDAC inhibitor that is being studied.
- Antifibrotic agents interfere with the process of tissue repair and fibrosis. PRM-151 is an antifibrotic therapy that is being tested to see whether it prevents and/or reverses fibrosis in MF. Lysyl oxidase (LOX) and lysyl oxidase-like (LOXL) are also antifibrotic medications being studied in a clinical trial.
- Imetelstat is a telomerase inhibitor, which affects the ability of dividing cells to repair the loss of DNA that happens during cell division. It is being studied in MF to improve bone marrow fibrosis, bone marrow function and blood cell counts.
Certain factors affect prognosis and treatment options for patients with MF. The Dynamic International Prognostic Scoring System (DIPSS) Plus uses the following eight risk factors:
- Age older than 65
- Hemoglobin lower than 10 g/dL
- Leukocytes greater than 25 x109 /L
- Circulating blast cells equal to or greater than 1 percent
- Constitutional symptoms (fatigue, night sweats, fever, unexplained weight loss, etc)
- The need for red blood cell transfusions
- Platelet count less than 100 x109 /L
- Unfavorable karyotype (chromosome abnormalities)
The risk groups based on these eight risk factors are:
- Low-risk - Patients with no risk factors
- Intermediate 1 risk (INT-1) - Patients with one risk factor
- Intermediate 2 risk (INT-2) - Patients with two or three risk factors
- High-risk - Patients with four or more factors .
Researchers are also beginning to incorporate a patient’s mutational status in assessing a patient’s prognosis. For example, certain gene mutations in MF patients, such as the CALR mutation, are associated with better overall survival than those with JAK2 or MPL mutations. As researchers learn more about the genetic factors involved in MF, they will use this information for treatment planning.
Every patient’s medical situation is different and should be evaluated individually by a hematologist-oncologist who specializes in treating blood cancers. It is important for patients and the members of their medical team to discuss all treatment options, including treatments being studied in clinical trials.
To download lists of suggested questions to ask your healthcare providers, click here.
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