Treatment

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. 

Treatment for Patients Without Anemia 

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®)

Treatments for Patients with Anemia

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 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.
 

Clinical Trials

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. 

Click here to read more about clinical trials.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

There are clinical trials for MF patients in a number of different situations, whether they are newly diagnosed, have advanced-stage disease, or are intolerant or resistant to their current medications. Sometimes, a clinical trial is the best option for a patient.

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 patient 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. 

You can visit this Primary Myelofibrosis Prognostic Scores Calculator. 
 

To download lists of suggested questions to ask your healthcare providers, click here.

Related Links