Understanding Myelofibrosis and Your Treatment Options

This article is for people who are living with myelofibrosis and their caregivers or anyone who wants to learn more about myelofibrosis. The goal of this activity is to help you or your caregiver talk to your doctor and healthcare team about your treatment plan.

You will learn about:

• What myelofibrosis is and types of myelofibrosis

• Its possible symptoms and health effects

• How myelofibrosis may be managed

• Reviewing your treatment plan with your doctor and healthcare team

• Questions to ask your doctor and healthcare team

Medicines listed in this activity may not be approved by the US Food and Drug Administration (FDA) for myelofibrosis but are recommended by the National Comprehensive Cancer Network (NCCN).

Myelofibrosis belongs to a group of rare blood cancers known as myeloproliferative neoplasms (MPNs). MPNs change how your bone marrow (the spongy tissue inside your bones) makes blood cells.

In myelofibrosis, changes in your genes (part of your cell's DNA) cause the bone marrow to make abnormal cells and fibers. The fibers clump together and form scar tissue.

As the scar tissue grows, your bone marrow has less room to make all the new red blood cells (RBCs), white blood cells (WBCs), and platelets (cells that form blood clots) that you need. This can cause symptoms and other health problems.

You can have primary or secondary myelofibrosis. Primary myelofibrosis is when you've never had an MPN before.

Secondary myelofibrosis is when you had an MPN known as essential thrombocythemia (ET) or polycythemia vera (PV) that changed into myelofibrosis. Secondary myelofibrosis is also called post-ET myelofibrosis or post-PV myelofibrosis.

Primary and secondary myelofibrosis can cause similar symptoms and are managed in similar ways. Both can turn into acute myeloid leukemia, a fast-growing blood cancer.

Most myelofibrosis symptoms happen when your bone marrow can't make enough normal RBCs, WBCs, or platelets. Other symptoms result from swelling of your spleen (splenomegaly) or liver (hepatomegaly), which are organs that help make blood cells during a shortage.

Common health problems and symptoms due to myelofibrosis include:

• Anemia (RBC shortage): severe tiredness (fatigue), weakness, shortness of breath, pale skin, and confusion

• Neutropenia (WBC shortage): frequent infections and trouble fighting them

• Thrombocytopenia (platelet shortage): easy bleeding or bruising

• Thrombocythemia (too many abnormal platelets): blood clots and stroke

• Splenomegaly and hepatomegaly: stomach and back pain, bloating, and feeling full after eating little food

• Others: night sweats, itchiness, fever, bone pain, lack of hunger, and weight loss

Your doctor and healthcare team will work with you on a treatment plan. Treatment will depend on your symptoms, health issues, and risk of myelofibrosis getting worse.

Many people are asymptomatic (no symptoms) at first and don't require treatment. Instead, your doctor may watch you closely and start treatment when you develop symptoms. Myelofibrosis grows slowly, and you may be asymptomatic for years.

A bone marrow transplant is the only treatment that sometimes cures myelofibrosis. It has serious risks, however, and isn't an option for everyone.

Some myelofibrosis treatments are used to improve symptoms. They do this by shrinking your spleen or increasing your bone marrow's ability to make RBCs or platelets. Treatment options include medicines, surgery to remove your spleen, radiation therapy, and blood transfusions.

Medicines for myelofibrosis include Janus kinase (JAK) inhibitors, chemotherapy, androgens and other hormones, steroids, and medicines to aid your immune system. Your doctor may recommend more than 1 medicine to help manage myelofibrosis and its symptoms.

It's important to know how treatment might affect you. Ask your doctor or healthcare team about symptoms to watch for, how to report them, and how to recognize an emergency.

A JAK inhibitor is a medicine commonly used to treat symptomatic myelofibrosis. JAK inhibitors block the signals that cause scarring in your bone marrow.

JAK inhibitors can cause your spleen to shrink, which may help with symptoms. JAK inhibitors can't cure myelofibrosis, but they may slow down bone marrow damage.

There are a few JAK inhibitors, and each is slightly different, with different side effects. JAK inhibitors are taken until they stop working or you develop side effects that can't be managed.

Your doctor and healthcare team can help you decide if you should take a JAK inhibitor.

You may notice new or worsening symptoms over time if:

• You haven't started treatment and your myelofibrosis is getting worse

• Treatment isn't working as well as before

• Treatment is causing a health problem or side effect

• You have a new health problem

Tracking symptoms and health changes in a diary can alert your doctor and healthcare team to problems early. Always tell them right away if your symptoms change. You may need to review your treatment plan.

Reviewing your treatment plan with your doctor and healthcare team gives you an opportunity to:

• Bring up any symptoms or concerns you have

• Think again about your treatment goals

• Review possible side effects and how to manage them

• Discuss plans for if your myelofibrosis gets worse or you need to change treatment

Never stop treatment without first talking to your doctor or healthcare team member. Suddenly stopping certain treatments may cause myelofibrosis symptoms to return quickly and cause life-threatening side effects.

Questions you can ask your doctor about your myelofibrosis treatment plan include:

• What treatments can be used and what side effects are possible?

• When and how should I report changes in my symptoms?

• How will you know if my myelofibrosis is getting worse?

• What if my side effects won't go away?

• How can I make sure I have the best quality of life possible?

Congratulations!

You have successfully completed the program Understanding Myelofibrosis and Your Treatment Options.

View Additional Materials on this topic that you may find useful:

Chronic Myeloproliferative Neoplasms

Treating Myelodysplastic/Myeloproliferative Neoplasms

Primary Myelofibrosis

Talking With Your Healthcare Team

Questions to Ask Your Doctor About Treatment