Living with myelofibrosis (MF) and anemia significantly affects a person’s energy, daily plans, and peace of mind. Despite being a rare disease, MF research and new treatments are advancing at an extraordinary pace. A key consideration for MF patients is determining the best treatment plan for their specific situation.
According to Gabriela Hobbs, MD, clinical director of the Leukemia Service at Massachusetts General Hospital in Boston, the primary goal of MF treatments is to improve symptoms, including anemia. For patients with anemia, Dr. Hobbs’s priority is to resolve the anemia. This aligns with the highest priority of many patients with myeloproliferative neoplasms (MPNs), who want to reduce or eliminate the need for blood transfusions and ease chronic fatigue.
Treatment Options for Myelofibrosis and Anemia
There are several treatment approaches for MF and anemia, including Janus kinase (JAK) inhibitors, interferons, hydroxyurea, and stem cell transplants. JAK inhibitors are the most common treatment for symptomatic or high-risk myelofibrosis, with four FDA-approved drugs available: ruxolitinib (Jakafi), pacritinib (Vonjo), fedratinib (Inrebic), and momelotinib (Ojjaara).
Each JAK inhibitor has its own benefits and drawbacks. For example, ruxolitinib is often the first go-to therapy, but it can cause anemia as a side effect. Pacritinib, on the other hand, may help improve red blood cell counts and is less likely to worsen anemia. Momelotinib, the newest JAK inhibitor, was approved specifically for people with MF and anemia and may help reduce the need for transfusions.
Interferons, such as ropeginterferon alfa-2b (Besremi), are also used to treat MF, particularly in early-stage or lower-risk cases. Hydroxyurea, a decades-old chemotherapy drug, can relieve symptoms but may worsen anemia. Stem cell transplants are the only potentially curative treatment for MF, but they are high-risk and require specific health criteria.
A study conducted in 2025 found that the prospect of becoming transfusion-dependent contributes significantly to anxiety about MF-related health. The study’s findings highlight the emotional, physical, and logistical challenges associated with transfusions and the strong desire to minimize their frequency. Dr. Hobbs notes that it’s essential for patients and clinicians to appreciate the available treatment options and discuss switching to another medication if one isn’t improving anemia and its symptoms.
For patients with anemia, momelotinib may be the treatment of choice, particularly if it’s causing severe fatigue. Anemia treatments, such as luspatercept, erythropoiesis-stimulating agents (ESAs), and danazol, may also be used to help improve red blood cell production. With new therapies being studied and combination therapies being explored, patients with MF and anemia have more options than ever to manage their condition.
Personalized Treatment Plans
When anemia is present with myelofibrosis, one MF treatment may be more appropriate than another. The choice of treatment depends on various factors, including the patient’s symptoms, platelet counts, and overall health.
As research continues to advance, patients with MF and anemia can expect more personalized treatment options. Several studies are underway to find new MF treatments that won’t cause anemia or may even improve it. Clinical trials are also exploring combination therapies, adding newly developed drugs or existing anemia medications to established MF treatments. A patient’s care team can help determine whether a clinical trial could be right for them.
The National Comprehensive Cancer Network provides patient guidelines for MF, which can serve as a valuable resource for patients and clinicians. By working together, patients and their care teams can develop a personalized treatment plan that addresses their unique needs and goals. For example, a patient with severe anemia may require a different treatment approach than someone with mild anemia.
According to Dr. Hobbs, seeing a specialist familiar with MF is critical to exploring treatment options. The specialist can help patients understand the potential benefits and risks of each treatment and develop a plan that takes into account their individual needs and circumstances. With the right treatment plan, patients with MF and anemia can manage their condition and improve their quality of life.
Transplant Evaluation and Curative Options
Specialists use this information to determine whether a patient meets the criteria for a potentially curative approach.
The transplant process requires intensive conditioning therapy before the donor cells are infused, and the procedure carries a high level of complexity and risk.
Because of the demanding nature of the transplant, clinicians discuss the balance between potential long-term remission and the immediate risks of complications. Ongoing monitoring after the procedure focuses on graft success, infection prevention, and management of any residual disease activity.
Guidelines from leading oncology societies advise that every individual with high-risk disease should receive an evaluation for transplant eligibility, even if the patient ultimately chooses a different therapeutic path. This recommendation ensures that curative intent remains an option throughout the disease course.
Emerging Clinical Trials and Combination Strategies
Clinical trials now explore adding anemia-targeted agents to established myelofibrosis therapies. Researchers are testing whether combining a JAK inhibitor with a medication that stimulates red blood cell production can improve fatigue and reduce transfusion needs.
Combination studies also examine novel compounds that may modify disease biology while sparing blood counts. Early results suggest that some investigational drugs can lower bone marrow scarring and decrease the proportion of mutated cells, offering a potential shift in disease trajectory.
Patients interested in trial participation are encouraged to discuss eligibility criteria with their care team. Factors such as prior treatment history, current blood counts, and overall health influence trial suitability.