Myelofibrosis (MF) is a myeloproliferative neoplasm (MPN) that causes scarring in the bone marrow leading to a change in the production of blood cells in the body. While myelofibrosis is a rare disease, it is often progressive and requires treatment. Receiving a new myelofibrosis diagnosis can feel overwhelming, but there are a number of treatment options available.
People with myelofibrosis may experience a variety of symptoms, including anemia, increased bleeding risk, infections, an enlarged spleen, itching, night sweats, bone and muscle pain, abdominal discomfort, feeling of fullness, or inflammation.
Because treatments are not one size fits all, visiting a subspecialty treatment center that is focused solely on the treatment of MPNs, like the Weill Cornell Medicine Silver MPN Center, can often offer patients the full scope of available therapies prior to beginning a treatment plan. Coming to a specialty MPN Center can be an important next step after an initial myelofibrosis diagnosis in order to ensure that all potential treatment options are reviewed and discussed. Due to the range of symptoms a person may experience, different therapies may be prescribed or recommended, and in some cases, participating in a clinical trial may be the right treatment choice.
Learn more about some of the different therapies that may be available.
Ruxolitnib is a Food and Drug Administration (FDA) approved medication that is a commonly prescribed treatment for myelofibrosis. About 50% of myelofibrosis patients have mutations of the JAK2 gene that can be targeted by this therapy option to relieve symptoms and decrease enlarged spleens.
Erythropoiesis-stimulating agents (ESAs) are injected under the skin to help stimulate red blood cell production caused by anemia in some myelofibrosis patients. If anemia a is large concern, ESAs can be prescribed to decrease anemia and the corresponding symptoms.
Interferons are naturally occurring proteins in the body made to fight infections and have been developed as drugs to treat a number of disorders. Although they are not FDA approved for the treatment of MPNs, they have been shown to be an effective option for some and may be able to control blood counts, reduce clotting risk, and reverse scarring of the bone marrow.
Hydroxyurea is an oral chemotherapy medication that has the ability to reduce spleen size and the number of blood cells in the body. Slowing down the production of the abnormal cells can help with some patients scarring and disease symptoms.
The Weill Cornell Medicine Silver MPN Center participates in and leads a number of clinical trials aimed at determining new therapies or combinations of therapies that may be able to better treat myelofibrosis and reduce side effects. Because treatment plans for myelofibrosis can vary amongst patients, therapies in development through clinical trials may ultimately be the right choice for an individual even as the first line of therapy. Speaking to a member of our team can help determine if taking part in a clinical trial is the best option.
Prednisone, or similar anti-inflammatory steroid hormones, can be used to manage myelofibrosis symptoms, increase appetite, and improve overall blood counts in some patients. Clinical trials have also shown that combining prednisone with the oral medication lenalidomide may also help myelofibrosis symptoms.
An allogeneic stem cell transplant is the only known therapy with the ability to cure myelofibrosis but is not appropriate for every patient due to its aggressive nature and compatibility requirements. Many steps are essential before a patient can undergo a transplant. Should a person meet the requirements for a transplant, Weill Cornell Medicine/NewYork-Presbyterian Hospital has one of the best Bone Marrow and Stem Cell Transplant (BMT) programs in the world. The physicians at the Silver MPN Center collaborate very closely with the Weill Cornell BMT program team to ensure this treatment approach is considered for appropriate patients.
It’s important to remember that not every treatment option is right for every person and discussing the available options with a subspecialized physician expert is the best way to manage symptoms and the disease, especially after a new myelofibrosis diagnosis. Research for new treatment options is ongoing at the Silver MPN Center in an effort to continue to help improve myelofibrosis outcomes and quality of life.
At the Weill Cornell Medicine Silver MPN Center, our team of experts recognizes the nuances that come with each myelofibrosis diagnosis and is able to tailor treatments to each individual patient depending on their symptoms, disease progression, and whether or not they’ve received treatment previously. We also offer second opinions and care for patients who have received prior treatments or are undergoing care with other physicians. By leveraging the latest science and precision medicine we are able to partner with our patients to determine next steps in their course of care.