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There is no single 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. However, all MF patients 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, who are primarily affected by MF, and those with other health problems, so it’s 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.
Treatment planning
Certain factors can affect a patient’s prognosis (a person’s chance of recovery or the likely outcome of their disease). These are called “prognostic factors,” and they help doctors predict how a patient’s disease is likely to respond to treatment. These factors help doctors plan the most appropriate treatment for each patient. In addition, they may help determine whether allogeneic stem cell transplantation should be considered as a treatment option.
Prognostic scoring systems are used to evaluate treatment options for patients. There are multiple scoring systems available to help doctors predict the prognosis of patients with MF based on assessment of their risk factors.
The four most common ones are:
- Dynamic International Prognostic Scoring System (DIPSS)
- DIPSS Plus
- Mutation-Enhanced International Prognostic Scoring System 70 (MIPSS-70)
- MIPSS70-plus Version 2.0
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.
The MIPSS70-plus Version 2.0 categorizes patients into four risk groups based on eight risk factors including age, blood counts, symptoms, and genetic mutations. For each factor that a person has, one point is assigned. The points are totaled to determine the score and corresponding risk group for the patient, as follows:
- 0 points = very low risk
- 1 to 2 points = low risk
- 3 to 4 points = intermediate risk
- 5 to 8 points = high risk
- 9 points or more = very high risk
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.
Learn more about communicating with your blood cancer specialist or find a list of suggested questions to ask your healthcare providers.
Types of treatment for MF
Open each section below to learn more.
For patients without anemia, the treatment approach is based on the patient’s prognostic level as well as the presence and severity of MF symptoms.
Lower risk without symptoms
Patients who are symptom-free and have no signs of anemia, an enlarged spleen, or other complications at the time of diagnosis are generally not treated. Some people remain stable and symptom-free for many years.
However, these patients still need to be monitored closely with regular medical checkups and tests to detect any signs and symptoms of disease progression. Treatment as part of a clinical trial is recommended if symptoms appear.
Lower risk with symptoms
The treatment approach for low-risk category patients who have symptoms of MF may be observation only. In certain circumstances, the doctor may prescribe cytoreductive treatment (a medication to lower blood cell counts) to help relieve the patient’s symptoms.
Treatment options include:
- Participation in a clinical trial
- Ruxolitinib (Jakafi®)
- Peginterferon alfa-2a (Pegasys®)
- Hydroxyurea (Hydrea®) to relieve symptoms caused by high blood counts
- Pacritinib (Vonjo®) if the platelet count is less than 50,000
- Momelotinib (Ojjaara)
- Fedratinib (Inrebic®)
Higher risk and platelets are very low (below 50,000)
Treatment options include:
- Allogeneic stem cell transplant
- Participation in a clinical trial
- Pacritinib (Vonjo®) (preferred regimen) or momelotinib (Ojjaara)
Higher risk and platelets are within the low to high range (50,000 or higher)
Treatment options include:
- Allogeneic stem cell transplant
- Participation in a clinical trial
- Ruxolitinib (Jakafi®), fedratinib (Inrebic®), momelotinib (Ojjaara), pacritinib (Vonjo®)
Most people with MF develop anemia within one year after diagnosis. For patients with anemia, the treatment approach depends on whether MF symptoms are present and, if present, on how well drug therapy is controlling these symptoms. Some of the recommended treatment options are listed below
When there are no MF symptoms, treatment may include:
- Participation in a clinical trial
- Luspatercept-aamt (Reblozyl®)
- Erythropoiesis (EPO)-stimulating agents (ESA) if EPO level is lower than 500 mU/mL
- Momelotinib (Ojjaara)
- Pacritinib (Vonjo®)
- Receive red blood cell transfusions
When a JAK inhibitor drug is controlling MF symptoms, treatment may include:
- Participation in a clinical trial
- Luspatercept-aamt (Reblozyl®), erythropoiesis-stimulating agent (ESA) or ruxolitinib (Jakafi®)
- Switch current JAK inhibitor to either momelotinib (Ojjaara) or pacritinib (Vonjo®)
- Receive red blood cell transfusion
When MF symptoms are not controlled, treatment may include:
- Participation in a clinical trial
- Momelotinib (Ojjaara)
- Pacritinib (Vonjo®)
- Luspatercept-aamt (Reblozyl®), erythropoieses-stimulating agent (ESA) or ruxollitinib (Jakafi®)
- Receive red blood cell transfusions
For information about the drugs listed on this page, visit our cancer drug listing.
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 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.
There are clinical trials for MF patients whether they are newly diagnosed, have advanced-stage disease, or are intolerant or resistant to their current medications.
Read more about clinical trials.
The goal of palliative, or supportive, care is to prevent or treat the following MF symptoms and improve the quality of life for patients.
Anemia
Anemia is observed in more than 50 percent of patients with MF at the time of diagnosis. Before considering treatment options, it is important for doctors to rule out and treat the most common causes of anemia such as bleeding, iron deficiency, vitamin B12 deficiency, and folic acid deficiency.
Blood transfusions are recommended for patients with anemia that is causing symptoms. Blood transfusions can increase a patient’s red blood cell count and ease symptoms such as fatigue and weakness. Additional treatment options may be considered, based on the patient’s serum erythropoietin (EPO) level.
Erythropoietin is a hormone needed for normal red blood cell production. In the body, it is made mainly by the kidneys.
Treatment recommendations for patients who have MF with anemia include:
- Darbepoetin alfa or epoetin alfa, which are drugs called “erythropoietinstimulating agents (ESAs).” They are made in the laboratory and work by stimulating the bone marrow to make red blood cells.
- Danazol or other androgen drugs, which are synthetic versions of male hormones (androgens) that may help increase red blood cell production.
- The immunomodulators, thalidomide (Thalomid®) and lenalidomide (Revlimid®), are both given by mouth to help improve red blood cell counts. These drugs may be combined with prednisone, a steroid.
- Momelotinib (Ojjaara), a JAK inhibitor that can improve anemia as well as MF symptoms
- Luspatercept-aamt (Reblozyl®), an erythroid maturation agent that is currently under study in clinical trials.
Enlarged spleen (Splenomegaly)
Many patients with MF have enlarged spleens that may cause symptoms such as abdominal discomfort, pain under the left ribs and a feeling of fullness without eating or after eating a small amount.
There are several options for dealing with the painful effects of an enlarged spleen, which include:
- Ruxolitinib (Jakafi®), which has been shown to reduce spleen size in some patients
- Hydroxyurea (Hydrea®), which may reduce the size of an enlarged spleen and relieve related symptoms
- Peginterferon alfa-2a (Pegasys®). This therapy can control an enlarged spleen.
- Interferon alfa (Intron A, Roferon-A, Pegasys), which can control spleen enlargement
- Surgical removal of the spleen (splenectomy), which may be considered if other forms of therapy have not reduced the pain or complications associated with an enlarged spleen. Benefits and risks of this procedure need to be weighed.
- Radiation therapy, which uses high powered X-rays to shrink the spleen. When other treatment methods have failed and surgical removal of the spleen is not a viable option, radiation therapy can be used to help reduce the size of the spleen
- Embolization of the spleen. This minimally invasive treatment is an alternative to the surgical removal of all or part of the spleen. While the patient is sedated, the doctor injects embolizing agents through a catheter into an artery to block blood flow to the spleen and reduce its size.
Thrombocytosis and leukocytosis
Some patients who have MF suffer from thrombocytosis (in which the bone marrow produces too many platelets) or from leukocytosis (in which the bone marrow produces too many white blood cells). Hydroxyurea (Hydrea®) may be given to reduce the high platelet and white blood cell counts.
It may also help treat other symptoms, including an enlarged spleen, night sweats and weight loss. Patients with low blood cell counts or severe anemia should not take hydroxyurea.
For information about the drugs listed on this page, visit our cancer drug listing.
Get free clinical trial support! Visit our Clinical Trial Support Center (CTSC).
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Treatment outcomes
Among patients with MF, the prognosis or likely outcome of the disease varies widely. Each patient’s risk factors are evaluated individually to determine their prognosis. Some people with MF may survive for decades following their diagnosis.
It is important to know that outcome data can show how groups of people with an MPN responded to treatment in the past, but it cannot always determine how any particular person will respond. For these reasons, patients are advised to discuss information about survival with their doctors.
Follow-up care
Find more information about follow-up care, including what to expect, long-term and late effects of treatment, survivorship clinics, and other resources, such as The National Comprehensive Cancer Network (NCCN) treatment guidelines.
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