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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.
 

Low-Risk Myelofibrosis (MF) Patients

Low 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 may be started if symptoms appear.

Low 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:

  • Ruxolitinib (Jakafi®)
  • Interferon alfa (Intron® A, Roferon®-A, and Pegasys®), although the best use of these medications may be in a clinical trial
  • Hydroxyurea (Hydrea®)

Intermediate-Risk and High-Risk MF Patients

Intermediate-1 Risk (INT-1) Patients With Symptoms. Treatment may include:

  • Pacritinib (Vonjo™)
  • Ruxolitinib (Jakafi®) 
  • Allogeneic stem cell transplantation (in some cases)

Intermediate-2 Risk (INT-2) and High-Risk Patients. Treatment may include:

  • Pacritinib (Vonjo™)
  • Ruxolitinib (Jakafi®)
  • Fedratinib (Inrebic®)
  • Allogeneic stem cell transplantation (for eligible patients, the transplant may begin shortly after diagnosis) 
  • Treatment for anemia 

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.
 

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.


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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. Drugs being researched for use in treatment of MF include: 

  • Momelotinib, luspatercept and sotatercept. Use of these drugs is being studied to help patients with chronic anemia.
  • Bomedemstat (IMG-7289). This drug is being studied to help patients with high platelet counts.
  • JAK inhibitors. In current clinical trials, drugs called “JAK inhibitors” are showing effectiveness in reducing spleen size and symptoms such as night sweats and fatigue, and possibly improving anemia. Pacritinib (SB1518) is being studied to lower platelet counts, and momelotinib is being studied for the treatment of anemia.
  • Antifibrotic agents. These 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 patients with MF. Lysyl oxidase (LOX) and lysyl oxidase-like (LOXL) are other antifibrotic medications being studied in a clinical trials.
  • Telomerase inhibitor. A drug called “imetelstat” is being studied for use in MF treatment to improve bone marrow fibrosis, bone marrow function and blood cell counts.
  • Panobinostat, pracinostat, umbralisib, bomdestat and navitoclax. For patients who no longer respond to ruxolitinib, researchers are studying the use of these drugs, either in combination with ruxolitinib or as single-agent therapies.

Treatment Planning

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
  • Constitutional symptoms 
  • Hemoglobin lower than 10 g/dL
  • White blood cell count greater than 25 x109 /L
  • Peripheral blood blasts greater than 1 percent
  • Platelet count less than 100 x109 /L
  • Transfusion need
  • 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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