Treatment Options

The treatment plan that your doctor recommends for myelofibrosis will be determined largely by the types of symptoms you’re experiencing, if any—two thirds of people with this condition are symptomless when they are first diagnosed. If you currently don’t have symptoms, you may be a candidate for a strategy known as “watchful waiting,” in which you won’t receive active therapies. Instead, your doctor will monitor you closely with regular checkups and laboratory tests.

If you begin to develop myelofibrosis symptoms, your doctor will recommend a treatment plan based on the specific problems you are experiencing, as well as your age, blood cell counts, and other factors. This article describes the various treatments that doctors use to control symptoms of myelofibrosis and slow its progress. While these therapies can improve quality of life, they also carry certain risks and can cause side effects.

Drug Therapies

Initial treatment for most patients with myelofibrosis will be drug therapy. A class of medicines known as JAK inhibitors has revolutionized the treatment of myelofibrosis, but older medications still have a role in managing symptoms for many patients.

JAK inhibitors: These drugs block a protein that becomes overactive in myelofibrosis, promoting formation of scar tissue in bone marrow. Studies show that JAK inhibitors can reduce spleen enlargement, a common and potentially painful problem in this disease, while improving other symptoms and overall quality of life. Several JAK inhibitors are available for treating myelofibrosis, including ruxolitinib (Jakafi), fedratinib (Inrebic), and pacritinib (Vonjo), though the latter two are intended for patients with specific circumstances. Other JAK inhibitors for treatment of myelofibrosis are under investigation in clinical trials. Some common side effects include diarrhea and fatigue, and patients should be monitored for anemia and internal bleeding.

Hydroxyurea: This medicine belongs to a class of drugs called cytoreductive therapies, which hinder bone marrow’s ability to make blood cells. Hydroxyurea can help treat an enlarged spleen and has other benefits. Hydroxyurea (or, less often, other cytoreductive therapies) may be used to treat moderate spleen-related symptoms or if JAK inhibitors are not an option.
Androgen therapy: Treatment with a synthetic version of male hormones, or androgens, can promote red blood cell production and improve severe anemia, a common symptom of myelofibrosis. Potential downsides to androgen therapy include liver damage and masculinizing effects in female patients.
Thalidomide and lenalidomide: The related medications thalidomide (Thalomid) and lenalidomide (Revlimid) may be prescribed with the goal of improving common myelofibrosis symptoms, including anemia, spleen enlargement, and internal bleeding. These drugs carry several warnings, including that they must be used with extreme caution by women of child-bearing age.
Recombinant erythropoietin: This treatment may be used if a patient has anemia related to low levels of erythropoietin, a hormone that’s necessary for production of red blood cells. Recombinant erythropoietin is a synthetic version of the naturally occurring hormone.
Anagrelide hydrochloride: If a patient with myelofibrosis has a related condition called thrombocytosis, he or she produces too many blood platelets, which can cause dangerous clots in blood vessels. This drug reduces platelet count.

Radiation Therapy

This treatment, also called radiotherapy, uses beams of intense energy to shrink tumors and relieve symptoms in many forms of cancer. In myelofibrosis, radiation therapy may be recommended to shrink an enlarged spleen, though some doctors suggest that it should be reserved for patients who can’t undergo surgery to treat the problem (see Splenectomy, next item). The benefits of radiation therapy for myelofibrosis are usually temporary.

Surgical Removal of the Spleen (Splenectomy)

Surgical removal of the spleen, or splenectomy, is often considered for patients who develop severe symptoms related to myelofibrosis, which can cause this organ to become enlarged. Doctors will attempt to use medication first to reduce the size of an enlarged spleen, but that isn’t always possible. Symptoms of an enlarged spleen that may require surgical treatment for relief can include abdominal pain and discomfort, blood in the stool, internal bleeding, and others. Post-surgical risks include infection, excessive bleeding, and blood clots, which increase the risk for strokes and a dangerous condition called pulmonary embolism (blood clot in the lungs). Some patients develop an enlarged liver.

Stem Cell Transplantation

While other treatments for myelofibrosis can relieve symptoms, stem cell transplants can often send the disease into remission. Stem cells have the unique ability to develop into other, specialized cells. In this procedure (also called a bone marrow transplant), a patient first undergoes chemotherapy or radiation therapy to destroy diseased bone marrow. Then, healthy stem cells are infused into bone marrow with the goal of restoring a normal balance of blood cells in a patient with myelofibrosis. Patients must meet strict criteria to be candidates for stem cell transplants. Serious and even life-threatening side effects are a risk with stem cell transplants, including graft-versus-host disease, in which the patient’s immune cells perceive the infused stem cells as foreign bodies and wage an attack on them.

Here are two terms you may hear mentioned in association with stem cell transplantation for myelofibrosis:

Allogeneic stem cell transplant: This is the most common form of stem cell transplant for myelofibrosis. In this procedure, the patient receives healthy stem cells from a well-matched donor.
Non-myeloablative stem cell transplant: Also called reduced-intensity stem cell transplant, this version of the procedure uses lower doses of chemotherapy or radiation, or none at all, in some cases.

Clinical Trials

For many patients with myelofibrosis, clinical trials offer an additional treatment option. A clinical trial is a research study in which scientists evaluate the effectiveness and safety of innovative medical treatments, including new drugs or novel combinations of drugs. All medications approved by the U.S. Food and Drug Administration, the European Medicines Agency, and other regulatory agencies around the world must be tested first in clinical trials. Several new therapies for the treatment of myelofibrosis are being studied in clinical trials underway at sites around the world. Massive Bio specializes in helping cancer patients find clinical trials of promising new treatments. Our artificial intelligence-powered platform, SYNERGY-AI, uses over 170 proprietary algorithms to extract data from patients’ medical records, then matches them to clinical trials whose criteria and availability align with their cancer type, treatment history, and location.

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