Myelofibrosis Treatment

There may be several doctors involved in your care. In addition to your hematologist/oncologist you might also have a surgeon, radiation oncologist, and/or a stem cell transplant specialist. Other key players on your medical team include support persons, such as your oncology nurse, nurse navigator, oncology social worker and pharmacist. Each of these professionals can help guide you to information and resources that may help you both during and after your treatment. If you do not meet these professionals upfront, ask your doctor how you might arrange an appointment with them. In addition, you may want to contact your insurance carrier to ask for a case manager to be assigned to you to assist in managing your treatment coverage options. 

There are many approaches to treatment depending on your MF diagnosis and its progression.  Some common types of treatment include: 

Watch and Wait
Patients who are symptom free and do not have signs of anemia, an enlarged spleen or other complications are generally not treated at the time of diagnosis. Some people remain stable and symptom free for many years. However, it is very important that such patients be closely monitored through regular medical check-ups and examinations to detect any signs or symptoms of disease progression.

Drug Therapies
There are various drugs used in the treatment of myelofibrosis, and many of them have very different approaches to slowing the disease process and/or controlling the effects of the disease. With any drugs, side effects will likely exist; your doctor or nurse should review these with you before starting.

These are drugs that kill cancer cells that divide rapidly. These agents also attack other fast growing cells in the body, such as nails and hair and may cause other more serious side effects. These drugs may be given by mouth (orally) or into the vein (intravenously).

Janus - associated kinase (JAK) inhibitor

This class of drug inhibits enzymes called “JAK1” and “JAK2”, which are involved in regulating blood. It is important to note that JAK inhibitors work in both patients that have a JAK mutation and those that do not have a JAK mutation. The JAK inhibitor is the first FDA approved therapy for patients with MF. Their focus is to treat and lessen some of the symptoms of MF, including an enlarged spleen, night sweats, itching, and bone or muscle pain. An increased risk of bleeding and infection may occur as may bruising, dizziness and headaches.

Immunomodulators (IMiDs) 

The drugs in this class work against cancer cells by affecting the functions of the immune system. Certain immunomodulators are used to treat MF patients for anemia. Another immunomodulator has been used to treat an enlarged spleen, bone pain and high platelet count in selected MF patients. Other immunomodulators are under study in clinical trials. 

Androgen therapy
These drugs are synthetic forms of male hormones, and can promote red blood cell production. They are used to relieve the symptoms of severe anemia in MF patients. These drugs can be toxic to the liver, and, therefore, require close monitoring of liver functions through blood tests and ultrasound. They may also cause facial hair growth and other masculinizing effects in women.

Recombinant erythropoietin 
This treatment helps regulate red blood cell production. It is given by intramuscular (into the muscle) or subcutaneous (under the skin) injection. However, the response in anemia-related symptoms in MF patients has been limited.

Also, known as “steroids”, these drugs are used to treat various conditions. They are used in MF patients to treat significant anemia. A number of side effects may occur when using glucocorticoids but are usually manageable with short term use.

These drugs are used to prevent bone loss in cancer patients, and may relieve bone pain, as well as increase blood counts in MF patients. These drugs are given intravenously. Short term use may cause upset stomach.

Anagrelide hydrochloride
This drug, given orally, may be used in MF patients who have an extremely high platelet c 

Radiation Therapy
Radiation uses high-powered beams, such as X-rays, to kill cells. Radiation therapy can help reduce the size of the spleen, when surgical removal isn’t an option. It is also used to treat bone pain and tumors outside the bone marrow.

Surgical Removal of the Spleen (Splenectomy)
If your spleen is enlarged and causing harmful complications and pain, and other interventions have not been successful, you may benefit from having your spleen surgically removed. Your doctor will consider the risks and benefits of your particular situation and determine if this is a feasible option. If surgery is done, you should be closely monitored for complications.

Stem Cell Transplantation
This is the only type of treatment that has the potential to cure myelofibrosis. However, it is a very high risk treatment, and can only be considered for patients deemed appropriate, and is dependent on the availability of a suitable donor.

Allogeneic stem cell transplant
In this procedure, the patient receives high doses of chemotherapy or radiation to destroy the diseased bone marrow. The donor stem cells (from someone other than the patient) are then infused into the patient.

Non-myeloblative stem cell transplant
This transplant uses lower doses of chemotherapy or radiation prior to the infusion of donor cells. This type of transplant can be used in older and sicker patients, who may not be eligible for an allogeneic transplant. Headache, diarrhea, weakness, nausea and dizziness may occur.

Clinical Trials
Because doctors are still trying to find more effective treatments for myelofibrosis, clinical trials are an important option to consider. Clinical trials are research studies to test promising new drugs or treatment approaches. Participating in a clinical trial may give you the opportunity to be among the first to benefit from the most advanced treatment available. If you are interested in a clinical trial, ask your healthcare team about studies that you might qualify for. 

The latest research in myelofibrosis involves looking at the effectiveness of several different JAK-2 inhibitors in patients with MF. This treatment stems from the knowledge that the mutation of the JAK2 gene is one mutation that may lead to the development of myelofibrosis. JAK2 inhibitor drugs are used for all mutations, including those who test JAK2 negative. Some of the benefits of such therapies may include: reducing spleen size, improving anemia, platelet count, and other symptoms, including fatigue, night sweats, weakness, and shortness of breath. Click here for more information on clinical trials and how to participate in one.

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