A closer look at treatment for myelofibrosis

Myelofibrosis (MF) is a type of cancer that causes scarring (fibrosis) in the bone marrow. Bone marrow is the soft, spongy tissue found at the center of most bones in the human body. This tissue contains a dense network of blood vessels, and it is responsible for the production of the majority of the body’s supply of blood cells.

Most cases of MF are not curable, and treatment will focus on controlling symptoms and complications, and helping the person with MF live as long as possible with the best possible quality of life.

Treatment for MF will vary from person to person, and treatment recommendations will depend on several different factors. Here, we look at what some of those factors are, along with the treatment options you may discuss with a healthcare team.

Low-risk and high-risk MF

MF is not staged like certain other cancers. Instead, healthcare providers use a scoring system. The scoring system can take into account a person’s age, blood cell counts, symptoms, results of genetic testing, and whether they have required a blood transfusion for anemia. Based on these factors, MF will be assigned a score that is low risk, high risk, or somewhere in between.

This score is used to guide treatment recommendations for MF.

Low-risk MF

If a person is low risk and does not have any symptoms, a healthcare team may recommend a “watchful waiting” approach. A person will have regular checkups and exams to monitor the disease to watch for signs of progression. If the disease progresses, treatment can begin. This can help avoid side effects from treatment.

If MF is scored at low risk, but is causing symptoms, a healthcare team may recommend watchful waiting or treatment with an anti-cancer drug, such as an immunotherapy or chemotherapy.

Intermediate and high risk MF

Treatment for intermediate and high-risk MF usually involves immunotherapy drugs called JAK inhibitors. These medications act on Janus-associated kinases (JAKs). These are proteins that play an important role in regulating the growth of blood cells.

MF is associated with mutations to the genes that contain the instructions for making JAKs, which creates JAKs that do not work correctly, which results in abnormal blood cells that replicate too rapidly. Blocking JAK proteins helps reduce the number of abnormal blood cells. This can help control the disease and ease symptoms.

In addition to JAK inhibitors, treatment may include other anti-cancer medications, such as other immunotherapies and chemotherapy.

Treatment for anemia

Anemia occurs when a person has low amounts of red blood cells. This is common among people with MF, and treatment often includes therapies to address or prevent anemia. This can include blood transfusions, as well as medications to help boost red blood cell numbers.

Enlarged spleen

While most of the body’s white blood cells are made in bone marrow, some are made in the spleen. When bone marrow becomes diseased or scarred, the spleen responds by producing more white blood cells. This can cause the spleen to become enlarged—and overactive. An enlarged spleen can cause abdominal pain, bloating, and problems with appetite and eating.

An enlarged spleen can also contribute to anemia. Another one of the spleen’s functions is to filter out damaged red blood cells. If the spleen is overactive, it can remove healthy red blood cells, reducing the number of red blood cells in circulation.

JAK inhibitors can help treat an enlarged spleen. In some cases, radiation therapy may be used. In severe cases, a healthcare team may recommend surgically removing the spleen.

Can MF be cured?

Currently, the only potential cure for MF is a procedure called an allogenic stem cell transplant, where a person receives stem cells collected from the bone marrow of a donor.

Many people will not be candidates for this procedure. It typically involves chemotherapy and/or radiation therapy. This is used to destroy any diseased cells before introducing donor stem cells. Chemotherapy and radiation therapy also weaken the immune system, which helps prevent the body from rejecting the stem cells after transplantation. However, there are reduced-intensity transplants, which utilize lower doses of chemotherapy and radiation therapy, and may be a treatment option for more people.