Managing and Treating Myelofibrosis (MF)

*Please note: This slide show is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always consult your doctor about any questions you may have regarding a medical condition.

Introduction to myelofibrosis (MF) management

When you have myelofibrosis, or MF, your bone marrow—the factory that makes blood cells—becomes scarred and can't work properly. This affects how your body makes red blood cells, white blood cells, and platelets.

Goals of MF treatment

The main goals of treating MF are to improve symptoms like fatigue and pain, reduce spleen size in people with enlarged spleens, improve blood cell counts, and help you feel better overall.¹

Goals of MF treatment

Most treatments help manage symptoms and improve how you feel every day. While a stem cell transplant can cure MF, it is not right for everyone who has MF.

Stem cell transplant for MF

Stem cell transplants are usually considered for people with higher-risk MF who are healthy enough to undergo the procedure. Your doctor will take your overall health into account and may use risk-scoring tools to help determine whether a transplant is a recommended option for you. If not, other treatments are available to manage your symptoms and help you feel as well as possible.^1,2

Risk assessment and scoring for MF

Doctors use scoring systems called IPSS, DIPSS, DIPSS-Plus, and MIPSS70+2.0 to learn how likely MF is to transform into leukemia.³ They count factors like age, symptoms, blood counts, and whether you have certain genetic changes. Your risk level—low, intermediate, or high—helps guide your treatment options.

The active surveillance approach

Low-risk people with no symptoms might just have checkups every 3 to 6 months. Doctors call this approach active surveillance.¹

During these visits, your doctor will check your blood counts, symptoms, and general health. If anything changes, you can start treatment as soon as necessary.

JAK inhibitors – The main treatment for MF

Many people with MF have mutations such as JAK2, CALR, or MPL. Four approved medications can block their activity: ruxolitinib, pacritinib, momelotinib, and fedratinib. These drugs are called JAK inhibitors. They calm the signals that cause inflammation and abnormal blood cell production. They can shrink an enlarged spleen and reduce symptoms like night sweats, itching, and fatigue.²

Choosing the right JAK inhibitor for MF

Each JAK inhibitor works a little differently. Your doctor will choose the best one for you.

Ruxolitinib is often used first for people with intermediate and high-risk MF.^1,2

Pacritinib is designed for people with very low platelet counts.

Momelotinib may help if you also have anemia, a low red cell count.

Fedratinib is commonly used after ruxolitinib or to better control spleen symptoms.¹

Managing anemia

More than half of people with myelofibrosis have anemia when they are first diagnosed.³

Managing anemia

Treatment depends on the cause. After ruling out causes like low iron or vitamin B12, your doctor may prescribe medications to boost red blood cell production.¹

Anemia treatment

You may receive a blood transfusion or medication to treat anemia symptoms.⁴ Options may include erythropoiesis-stimulating agents, or ESAs, luspatercept, or adjusting your JAK inhibitor therapy.¹

Other helpful medications

Hydroxyurea can help lower blood cell counts and reduce spleen size, though it is not specifically approved for myelofibrosis.⁵

Pegylated interferon may also help improve symptoms and manage blood counts.⁵

Managing an enlarged spleen

An enlarged spleen can cause pain and an early feeling of fullness when you eat. JAK inhibitors usually help shrink the spleen.²

If medication does not help, low-dose radiation or surgery to remove the spleen may, although these are less common options.

Supportive care for MF

Beyond medication, supportive care is important.⁶ This includes appropriate exercise to help manage fatigue, eating nutritious foods, not smoking, and treating anxiety or depression. You might need regular blood transfusions. You can work with your care team to adjust your treatment as needed.

Clinical trials offer hope for MF

Clinical trials study new treatments for myelofibrosis. They may give you access to treatments before they are widely available. Some are testing new medications or combinations of treatments, including drugs used with ruxolitinib, to see if they can improve symptoms or reduce spleen size more effectively.

Talk with your doctor about clinical trials you might qualify for. Organizations like the MPN Research Foundation can also help you find trials.

Living well with MF

Regular checkups, communicating with your doctor, and staying informed can help. Many effective treatments are available to help you control MF symptoms and live as well as possible.

References

1. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Myeloproliferative Neoplasms, Version 3.2024.
   https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1477
2. Harrison CN, Schaap N, Vannucchi AM, Kiladjian JJ, Tiu RV, Zachee P, Jourdan E, Winton E, Silver RT, Schouten HC, Passamonti F, Zweegman S, Talpaz M, Lager J, Shun Z, Mesa RA. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, multicentre study. Lancet Haematol. 2017 Jul;4(7):e317-e324.
3. Tefferi A. Primary myelofibrosis: 2021 update on diagnosis, risk-stratification and management. Am J Hematol. 2021 Jan;96(1):145-162.
4. Cervantes F, Alvarez-Larrán A, Hernández-Boluda JC, Sureda A, Torrebadell M, Montserrat E. Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature. Br J Haematol. 2004 Nov;127(4):399-403.
5. Kiladjian JJ, Mesa RA, Hoffman R. The renaissance of interferon therapy for the treatment of myeloid malignancies. Blood. 2011 May 5;117(18):4706-4715.
6. Mesa R, Miller CB, Thyne M, Mangan J, Goldberger S, Fazal S, Ma X, Wilson W, Paranagama DC, Dubinski DG, Boyle J, Mascarenhas JO. Myeloproliferative neoplasms (MPNs) have a significant impact on patients' overall health and productivity: the MPN Landmark survey. BMC Cancer. 2016 Feb 27;16:167.