Medication
Myelofibrosis (MF) treatment is based on your symptoms. If you don't feel bad right now, you may be able to just watch and wait without treating your cancer. Regular checkups and blood tests will make sure you don't have complications like anemia or an enlarged spleen.
Procedures
There are currently no medications that cure myelofibrosis. Allogeneic hematopoietic stem cell transplantation is the only treatment that may cure MF or significantly prolong the survival of people with MF. Stem cell transplants involve replacing abnormal stem cells in the bone marrow with an infusion of stem cells from a healthy donor.
Therapy
Myelofibrosis is a rare type of blood cancer in which the bone marrow (the soft, spongy tissue inside most bones) is replaced by fibrous scar tissue. It is considered a form of chronic leukemia.
Nutrition
If your primary doctor suspects that you have myelofibrosis — often based on an enlarged spleen and abnormal blood tests — you're likely to be referred to a doctor who specializes in blood disorders (hematologist). Because appointments can be brief, and because there's often a lot of information to discuss, it's a good idea to be prepared.
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How is myelofibrosis (MF) treated?
What medications are used to treat multiple myelofibrosis?
What is myelofibrosis?
What kind of doctor should I see if I have myelofibrosis?
What is the best treatment for myelofibrosis?
The drugs fedratinib (Inrebic) pacritinib (Vonjo), and ruxolitinib (Jakafi) are approved to treat MF. Most people with MF have a mutation, or change, in one of their genes that tell their body how to make blood cells. These inhibitors are used to block the processes those faulty genes.
What is the life expectancy with myelofibrosis?
Primary myelofibrosis, also known as idiopathic myelofibrosis or myelofibrosis with myeloid metaplasia, is a rare disease19, 20 usually affecting elderly people. Median survival ranges from 4 to 5.5 years in modern series6, 7, 8, 9, 10, 11, 12, 13, 14 (Figure 1).
How is bone marrow fibrosis treated?
There is no drug therapy that can cure MF. The only potential cure for MF is allogeneic stem cell transplantation. But this procedure is risky for older patients and those with other health problems. Because MF primarily affects older adults, a stem cell transplantation is not a treatment option for most MF patients.
Does myelofibrosis go away?
Some people with myelofibrosis have no symptoms and might not need treatment right away. Others with more-serious forms of the disease might need aggressive treatments right away.
Can you live 20 years with myelofibrosis?
Results from a retrospective study suggest that identification of patients with primary myelofibrosis likely to survive 20+ years can be made on the basis of age, sex, blood counts, and symptoms, without inclusion of genetic risk factors. This study was published in the American Journal of Hematology.
What is the longest someone has lived with myelofibrosis?
20+ Years and alive with primary myelofibrosis: Phenotypic signature of very long-lived patients. Am J Hematol. 2019 Mar;94(3):286-290.
Are there any new treatments for myelofibrosis?
In 2019, the FDA approved the JAK-2/FLT-3 inhibitor fedratinib (Inrebic) patients with intermediate- or high-risk myelofibrosis based on data from the JAKARTA trial (NCT01437787), in which 37% (n = 35/96) of patients treated with the recommended 400-mg dose achieved at least a 35% reduction in spleen volume vs 1 ...
What medications are used to treat myelofibrosis?
Two JAK inhibitors, ruxolitinib (Jakafi®) and fedratinib (Inrebic®), are approved to treat myelofibrosis. These drugs (e.g. thalidomide, lenalomide) fight cancer cells by helping the immune system work normally.
Can myelofibrosis be reversed?
A process called allogenic stem cell transplantation is the only cure.
What happens if myelofibrosis is left untreated?
One of the biggest complications with myelofibrosis, especially if left untreated, is that patients can develop progressive abdominal distension and, as result of that, abdominal pain and early satiety. Once patients develop early satiety, they develop weight loss.
Does exercise help myelofibrosis?
Your goal is to build up to one of these: About 150 minutes a week (roughly 20 minutes a day) of moderate exercise, like walking, swimming, or yoga. About 75 minutes a week of more vigorous exercise, like jogging, dancing, swimming, or riding your bike.
What is myelofibrosis caused from?
Myelofibrosis is associated with a genetic mutation in blood stem cells. However, researchers aren't sure what causes this mutation. When the mutated cells replicate and divide, they pass the mutation on to new blood cells. Eventually, the mutated cells overtake the bone marrow's ability to produce healthy blood cells.
What is MF treatment?
Myelofibrosis Symptoms. Myelofibrosis Diagnosis. Myelofibrosis Treatment. Myelofibrosis Complications. Myelofibrosis (MF) treatment is based on your symptoms. If you don't feel bad right now, you may be able to just watch and wait without treating your cancer. Regular checkups and blood tests will make sure you don't have complications like anemia ...
What are the things that can help your doctor plan your treatment?
Things like your age, blood cell counts, levels of immature blood cells called blasts, and symptoms like anemia or severe weight loss can help your doctor plan your treatment. One way to do this is to rank your risk of serious health problems using a scoring formula.
What is the best treatment for anemia?
These can give you more red blood cells and ease symptoms from severe anemia. Chemotherapy. The chemotherapy drug cladribine ( Leustatin ), helps with anemia symptoms, but it can also make you more likely to get infections and have trouble getting your blood to clot. Erythropoietins.
What is the best treatment for blood clots?
Erythropoietins. An injection of the hormone epoetin alfa ( Epogen, Procrit) helps you make more red blood cells. This treatment could make it more likely for you to get blood clots. Immunomodulators. Blood cancer drugs like lenalidomide ( Revlimid) and thalidomide ( Thalomid) treat anemia in MF.
Does MF cause bruising?
The medications can ease some MF symptoms like enlarged spleen, bone pain, itching, and night sweats. But they can have side effects, like a decrease in platelets or making your anemia worse. You might also have bruising, dizziness, or headaches.
What is the best treatment for myelofibrosis?
They might also recommend other treatments, including chemotherapy or radiation, to shrink it back down. Stem Cell Transplants. The only treatment that can cure myelofibrosis is a stem cell transplant , but it doesn’t work for everyone.
What is the first sign of myelofibrosis?
This condition is known as anemia. If you have anemia, your doctor might try to treat it with: In other cases, the first sign of myelofibrosis is that your spleen is larger than it should be. Your spleen is an organ near your liver and stomach that filters out and destroys old or damaged blood cells.
What is the best treatment for spleen swelling?
Your doctor may recommend the chemotherapy treatment hydroxyurea to help ease that swelling. If your spleen swells, your doctor also might give you a drug called ruxolitinib (Jakafi).
How do you know if you have myelofibrosis?
When you have myelofibrosis, your body tries to make blood cells in places like your spleen or liver.
Does myelofibrosis cause bruising?
It stops the genes that may be causing your myelofibrosis from working. It also may help with some of your symptoms, like fever, night sweats, and weight loss. But it can cause side effects like anemia, dizziness, and bruising. It also may weaken your immune system and make you more likely to get infections.
Can myelofibrosis cause shortness of breath?
In many cases, myelofibrosis doesn’t cause any symptoms until it's damaged your body’s ability to make red blood cells. When that happens, it’s harder for your cells to get oxygen, which leaves you feeling weak and short of breath. This condition is known as anemia.
What are the best medications for myelofibrosis?
This includes anemia, enlargement of the spleen, night sweats, itching, and bone pain. Medications to treat MF include: corticosteroids, such as prednisone. erythropoiesis-stimulating agents.
What drugs are used to treat MF?
One such class of drugs is histone deacetylase (HDAC) inhibitors. They play a role in gene expression and could treat MF symptoms when paired with ruxolitinib. Other trials are testing antifibrotic agents to see if these drugs prevent or reverse fibrosis in myelofibrosis.
What is MF in cancer?
Takeaway. Myelofibrosis (MF) is a rare type of cancer where a buildup of scar tissue keeps your bone marrow from making enough healthy red blood cells. This can cause symptoms like extreme fatigue and bruising. MF can also cause a low number of platelets in your blood, which can lead to bleeding disorders.
What is the name of the immunomodulator for MF?
immunomodulators, including thalidomide (Thalomid), lenalidomide (Rev limid), and pomalidomide (Pomalyst) Ruxolitinib is the first medication approved by the Food and Drug Administration (FDA) for the treatment of intermediate and high-risk MF. Ruxolitinib is a targeted treatment and a JAK2 inhibitor.
How does MF develop?
MF develops when a stem cell that produces blood cells is damaged. It starts producing immature blood cells that build up and cause scarring. This keeps your bone marrow from producing healthy blood cells. A stem cell transplant, also known as a bone marrow transplant, is a potentially curative treatment that addresses this problem.
What age can you get MF?
Doctors usually only recommend the procedure for people with intermediate- and high-risk MF who are under age 70 and have no other preexisting health conditions. A new type of reduced-intensity (nonmyeloablative) allogeneic stem cell transplantation requires lower doses of chemotherapy and radiation.
Why do you need a blood transfusion?
You may need a blood transfusion if you’re anemic due to MF. Regular blood transfusions can increase your red blood cell count and lessen symptoms such as fatigue and easy bruising.
What is myelofibrosis in cancer?
What is myelofibrosis? Myelofibrosis is a rare type of blood cancer in which the bone marrow (the soft, spongy tissue inside most bones) is replaced by fibrous scar tissue. It is considered a form of chronic leukemia.
What tests are performed to check for mutations in myelofibrosis?
Bone marrow biopsy: A sample of bone marrow may be removed for examination under a microscope. Imaging tests: An ultrasound test may be performed to check for enlargement of the spleen.
What are the cells that are overproduced in myelofibrosis?
One characteristic of myelofibrosis is the overproduction of giant cells called megakaryocytes. Megakaryocytes normally release tiny fragments called platelets. Platelets play an important role in the formation of blood clots at the site of an injury.
What is secondary myelofibrosis?
If it occurs as the result of a separate disease , it is known as secondary myelofibrosis (e.g. scar tissue in the bone marrow as a complication of an autoimmune disease). The bone marrow contains immature blood-forming cells that may develop into three types of specialized blood cells: red blood cells, white blood cells, or platelets.
How many people have myelofibrosis?
Myelofibrosis is a rare condition, with about 1.5 cases reported per 100,000 people each year in the United States. It occurs in both men and women. People of any age can have myelofibrosis, although it is more likely to be diagnosed in people over age 50.
Is myelofibrosis inherited?
Although myelofibrosis is not inherited, it is associated with DNA changes in certain genes. Proteins called janus-associated kinases (JAKs) play a role in myelofibrosis. JAKs regulate the production of blood cells in bone marrow by signaling the cells to divide and grow.
Does myelofibrosis show symptoms?
A person with myelofibrosis might not have any symptoms for many years. About one-third of patients do not show symptoms during the early stages of the disorder. However, during the course of the disease, they may experience the following symptoms or conditions:
What is the best treatment for MF splenomegaly?
The JAK inhibitors are effective in both JAK2-positive and JAK2-negative MF; one of them, ruxolitinib, is the current best available therapy for MF splenomegaly and constitutional symptoms.
What is a MF?
Myelofibrosis (MF), formerly known as idiopathic MF, MF with myeloid metaplasia, or agnogeneic myeloid metaplasia, is one of the classical BCR-ABL1-negative chronic myeloproliferative neoplasms (MPNs), a group also including essential thrombocythemia (ET) and polycythemia vera (PV). 1 Either appearing de novo (primary MF [PMF]) or following a previous ET or PV (post-ET or post-PV MF), 2 the disease is essentially the same. MF is a clonal proliferation of a pluripotent hematopoietic stem cell, 3 in which the abnormal cell population releases several cytokines and growth factors in the bone marrow that lead to marrow fibrosis and stroma changes and colonizes extramedullary organs such as the spleen and liver. 4 Discovery of the V617F mutation of the Janus kinase ( JAK) 2 gene in 60% of patients with PMF or post-ET MF and 95% of those with post-PV MF represented an important step in the understanding of the pathogenesis of MF. 5-7 Mutations in the thrombopoietin receptor gene ( MPL) were subsequently found in 3% to 8% of patients with PMF and post-ET MF, 8 whereas mutations in the calreticulin gene ( CALR) have been observed in half of patients with PMF and post-ET MF lacking JAK 2 and MPL mutations. 9, 10 Mutations shared by other myeloid neoplasms are found in some patients. 11 However, the genetic trigger of MF is unknown.
Which drugs improve anemia?
Androgens. Nandrolone, fluoxymesterone, methandrostenolone, and oxymetholone improve anemia in 30% to 60% of patients. 20, 21 Factors associated with a favorable response are female gender, previous splenectomy or lack of huge splenomegaly, and normal karyotype.
Is allo-sct a curative treatment?
At present, there is no curative treatment other than allogeneic hemopoietic stem cell transplantation (allo-SCT), which can be applied to a minority of patients. Therefore, treatment remains essentially palliative and aimed at controlling disease symptoms and complications and improving the patients’ quality of life.
Abstract
Myelofibrosis (MF) is a BCR-ABL1 –negative myeloproliferative neoplasm that is mainly characterised by reactive bone marrow fibrosis, extramedullary haematopoiesis, anaemia, hepatosplenomegaly, constitutional symptoms, leukaemic progression, and shortened survival.
Introduction
Myelofibrosis (MF) belongs to the category of myeloproliferative neoplasms (MPNs) and may present as a primary disorder (primary myelofibrosis [PMF]) or evolve from polycythaemia vera (PV) or essential thrombocythaemia (ET) to post-PV or post-ET MF.
Anaemia
The management of anaemia can be one of the most challenging aspects of treating patients with MF ( Table 2 ). Blood transfusion is the standard therapy for symptomatically anaemic patients, and the transfusion target should be assessed individually.
Splenomegaly
Cytoreductive agents have been the treatment of choice for most MF patients with symptomatic splenomegaly ( Table 3 ).
Iron Overload
Nearly 40% of patients with MF are anaemic at the time of diagnosis, including 25% who are already transfusion dependent, 77, 78 and more than 60% will develop clinically significant anaemia during the course of follow-up.
Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation is still the only intervention that has been shown to be a potential cure for MF or a means of prolonging the survival of these patients. Data from the most recent studies suggest that the expected 3-year progression-free survival rate is in the range of 40% to 50%. 102
Conclusions
Traditional MF treatments are primarily palliative and have proved to be inadequate to address the considerable morbidity and mortality associated with this disabling disease.
What is the main aim of treatment for MF?
The main aim of treatment is to control any symptoms you have. If you’ve been diagnosed with MF and don’t have any symptoms, you may not need to start treatment for a while. The treatment you have will for MF depends on a variety of factors, including: your overall fitness.
What to do if you don't have symptoms?
If you don't need to start treatment, you'll be monitored with regular check-ups and blood tests. This is known as 'watch and wait'. While this might seem strange, there’s no evidence to show that treating people who don't have any symptoms has any impact on their outcome.
How often do you need blood transfusions for anemia?
If you have severe anaemia you’ll need regular blood transfusions, usually every one to three months. These can be carried out during a single day and you would need to stay in hospital overnight. You might also have platelet transfusions if you have low platelets and you’re having unusual bleeding or bruising.
What is the best treatment for high platelet count?
This is a mild form of chemotherapy and works by directly preventing the production of red blood cells.
Why do you need Danazol?
You may be given danazol to help improve anaemia (low number of red blood cell s ). The length of time you take the drug for depends on how well it works and any side effects you get from it. A stem cell transplant aims to give patients healthy stem cells, which then produce normal blood cells.
Can ruxolitinib be taken as a tablet?
It can reduce the size of your spleen, improve symptoms and improve your overall prognosis (the forecast for your future). It is taken as tablets, twice a day, for as long as it is working. Possible side effects of ruxolitinib include anaemia, a low platelet count, ...
Is hydrocarbamide safe for AML?
Hydroxycarbamide is a very safe treatment. However, there’s a theoretical risk that it may increase the risk of MF transforming into acute myeloid leukaemia (AML) if it’s used as a long-term treatment.
What is the best treatment for primary myelofibrosis?
Updated on October 21, 2020. The only curative therapy for primary myelofibrosis (PMF) is stem cell transplant , however, this therapy is recommended for high- and intermediate-risk patients only. Even in this group, age and other medical conditions can increase the risks associated with transplantation significantly making it a less ...
What is the medication for myelodysplastic syndrome?
These medications are currently used to treat myelodysplastic syndrome. Studies looking at the role of azacitidine and decitabine are in early phases. The other medications are histone deacetlyase (HDAC) inhibitors like givinostat and panobinostat.
What is the best treatment for PMF?
Momelotinib is another JAK2 inhibitor being studied for treatment of PMF. Early studies noted that 45% of people who received momelotinib had reduction in spleen size. About half of the people studied had improvement in their anemia and more than 50% were able to stop transfusion therapy.
Is lenalidomide a PMF?
Thalidomide and lenalidomide have already been studied as treatment options in PMF. Although they both show benefit, their use is often limited by side effects. Pomalidomide was developed as a less toxic option. Some patients have improvement in anemia but no effect was seen in spleen size.
Can you have a stem cell transplant donor with PMF?
Additionally, not all people with high- and intermediate-risk PMF will have a suitable stem cell transplant donor (matched sibling or matched unrelated donor). It is recommended that people with low-risk PMF receive treatment aimed at reducing symptoms associated with the disease.
Is Everolimus an immunosuppressant?
Everolimus is a medication classified as an mTOR kinase inhibitor and immunosuppressant. It is FDA (Food and Drug Administration) approved for treatment of several cancers (breast, renal cell carcinoma, neuroendocrine tumors, etc.) and to prevent organ rejection in people who have received organ transplantation (liver or kidney). Everolimus is taken orally. Early studies indicate that it can decrease symptoms, spleen size, anemia, platelet count and white blood cell count.
Is Momelotinib a phase 3 drug?
Momelotinib will be compared to ruxolitinib in a phase 3 studies to determine its role in treatment for PMF. In August 2019, the FDA approved fedratinib for the treatment of adults with intermediate-2 or high-risk MF. 1 .
How to get rid of MF?
Make sure to take care of yourself. Eating right and getting mild exercise like walking, swimming, or yoga will help give you energy. It can also help take your mind off the stress involved in having MF.
What is MF in medical terms?
Myelofibrosis (MF) is a type of bone marrow cancer. This condition affects how your body produces blood cells. MF is also a progressive disease that affects each person differently. Some people will have severe symptoms that progress quickly.
What causes pain in the MF?
One of the most common symptoms and complications of MF is pain. Causes vary, and can include: gout, which can lead to bone and joint pain. anemia, which also results in fatigue. side effect of a treatment. If you’re in a lot of pain, talk with your doctor about medications or other ways to keep it under control.
Does MF have side effects?
Side effects of treatment for MF. Treatment side effects depend on many different factors. Not everyone will have the same side effects. Reactions depend on such variables as your age, treatment, and medication dosage. Your side effects may also relate to other health conditions you have or have had in the past.
Is MF a life changing disease?
MF is a chronic, life-altering disease. Coping with the diagnosis and treatment can be difficult, but your doctor and healthcare team can help. It’s important to communicate with them openly. This can help you feel comfortable with the care you’re receiving.
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