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. Many people with MF also have an enlarged spleen.
Traditional treatments aim to address the symptoms of MF and reduce the size of your spleen. Complementary therapies may ease some of your symptoms and improve your quality of life.
Here’s a closer look at the available treatments for MF.
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.
The procedure has significant and potentially life threatening risks. It’s usually only recommended for younger people without other preexisting health conditions.
Your doctor may recommend one or more medications to help treat symptoms or complications of MF. 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
- androgen therapies, such as danazol
- immunomodulators, including thalidomide (Thalomid), lenalidomide (Revlimid), and pomalidomide (Pomalyst)
- chemotherapy, including hydroxyurea
- JAK2 inhibitors, such as ruxolitinib (Jakafi) and fedratinib (Inrebic)
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. Mutations in the JAK2 gene have been associated with developing MF.
Fedratinib (Inrebic) was approved by the FDA in 2019 to treat adults with intermediate-2 and high-risk primary or secondary MF. Fedratinib is a highly selective JAK2 kinase inhibitor. It’s for people who don’t respond to treatment with ruxolitinib.
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.
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. Your doctor will need to assess your individual risk to determine if you’re a good candidate for the procedure.
Before a stem cell transplant, you’ll receive chemotherapy or radiation. This gets rid of remaining cancer cells and increases the odds that your immune system will accept donor cells.
Your healthcare practitioner then transfers bone marrow cells from a donor. The donor’s healthy stem cells replace the damaged stem cells in your bone marrow and produce healthy blood cells.
Stem cell transplants carry significant and potentially life threatening risks. 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. It may be better for older individuals.
Blood cells are normally produced by bone marrow. Sometimes, in those with MF, the liver and spleen produce blood cells. This can cause the liver and spleen to grow larger than normal.
An enlarged spleen can be painful. Medications help reduce the size of the spleen. If medications aren’t enough, your doctor may recommend surgery to remove your spleen. This procedure is called a splenectomy.
All MF treatments can cause side effects. Your doctor will carefully weigh the risks and benefits of potential treatments before recommending an approach.
It’s important to talk to your doctor about any treatment side effects you experience. Your doctor may want to change your dosage or switch you to a new medication.
The side effects you may experience depend on your MF treatment.
Androgentherapies can cause liver damage, facial hair growth in women, and growth of prostate cancer in men.
Side effects of corticosteroids depend on the medication and dosage. They may include high blood pressure, fluid retention, weight gain, and mood and memory problems.
Long-term risks of corticosteroids include osteoporosis, bone fractures, high blood sugar, and increased risk of infections.
These drugs can increase the number of white blood cells and platelets. This can lead to symptoms such as constipation and a prickly sensation in your hands and feet. They may also cause serious birth defects during pregnancy.
Your doctor will carefully monitor your blood cell count and may prescribe these drugs in combination with a low-dose steroid to minimize risks.
Common side effects of JAK2 inhibitors include a decreased level of platelets and anemia. They may also cause diarrhea, headache, dizziness, nausea, vomiting, headache, and bruising.
Fedratinib can, in rare cases, cause serious and potentially fatal brain damage known as encephalopathy.
Chemotherapy targets rapidly dividing cells, which include hair cells, nail cells, and cells in the digestive and reproductive tract. Common side effects of chemotherapy include:
- hair loss
- skin and nail changes
- nausea, vomiting, and appetite loss
- weight changes
- mood changes
- fertility problems
Removal of the spleen increases risk of infections and bleeding complications, including blood clots. Blood clots can lead to a potentially fatal stroke or pulmonary embolism.
Stem cell transplants
Bone marrow transplants may cause a life threatening side effect known as graft-versus-host disease (GVHD), when the donor’s immune cells attack your healthy cells.
Doctors try to stop this from happening with preventive treatments including removing T cells from the donor graft and using medications to suppress the T cells in the graft.
GVHD can affect your skin, gastrointestinal tract, or liver in the first 100 days after the transplant. You may experience symptoms including skin rashes and blistering, nausea, vomiting, abdominal cramps, loss of appetite, diarrhea, and jaundice.
Chronic GVHD can involve one or several organs and is the leading cause of death after a stem cell transplant. Symptoms can affect the mouth, skin, nails, hair, gastrointestinal tract, lungs, liver, muscles, joints, or genitalia.
Your doctor may recommend taking corticosteroids like prednisone or a topical steroid cream. They may also prescribe ruxolitinib for acute symptoms.
Clinical trials continue to look for new MF treatments. Researchers are testing new JAK2 inhibitors and exploring whether combining ruxolitinib with other medications could improve outcomes for people with 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. The telomerase inhibitor imetelstatis being studied to improve bone marrow fibrosis and function and blood cell counts in people with MF.
If you don’t respond well to a treatment, joining a clinical trial can give you access to newer therapies. Dozens of clinical trials are recruiting or actively evaluating myelofibrosis treatments.
Myelofibrosis is a chronic disease that requires medical intervention. No homeopathic or natural cures are proven myelofibrosis treatments. Always ask your doctor before taking any herbs or supplements.
Certain nutrients that support red blood cell production may reduce the risk and the symptoms of anemia. They won’t treat the underlying disease. Ask your doctor if you should take any of the following supplements:
- folic acid
- vitamin B-12
Eating a balanced diet and getting regular exercise can help reduce stress and keep your body functioning at a more optimal level.
NUTRIENT trial researchers hope that a Mediterranean diet can lower inflammation in the body to reduce the risk of blood clots, abnormal blood counts, and spleen changes in people with myelofibrosis. The Mediterranean diet is rich in fresh, anti-inflammatory foods including olive oil, nuts, legumes, vegetables, fruits, fish, and whole grain products.
One lab study suggested the traditional Chinese herbal remedy known as danshen or red sage (Salvia miltiorrhiza bunge) may theoretically impact the signaling pathways for myelofibrosis. The herb hasn’t been studied in humans, and it hasn’t been evaluated by the FDA for safety and efficacy. Always talk to your doctor before trying any supplement.
Two drugs have already made it through early stage clinical testing and are now in phase III clinical trials. Pacritinib is an oral kinase inhibitor with specificity for JAK2 and IRAK1. Momelotinib is a JAK1, JAK2, and ACVR1 inhibitor that will be compared to ruxolitinib in a phase III study.
Interferon-alfa has already been used to treat people with MF. It’s been shown to potentially reduce the production of blood cells by the bone marrow. More research is necessary to determine its long-term safety and efficacy.
Imetelstat is a telomerase inhibitor in phase II testing for intermediate-2 or high-risk MF individuals for whom JAK inhibitors didn’t work. The agent has shown promising results, though larger clinical trials are needed.
Predicting the outlook and survival with myelofibrosis can be difficult. Many people have MF for many years without experiencing any symptoms.
Survival varies depending on the type of MF, whether it’s low-risk, intermediate-risk, or high-risk.
One study found that people with low-risk MF were as likely to live for the 5 years following diagnosis as the general population, after which survival decreased. It found that people with high-risk MF lived for up to seven years after diagnosis.
The only treatment option that can potentially cure MF is a stem cell transplant. Some
A number of MF treatments are effective at addressing symptoms and improving your quality of life.
Medications including immunomodulators, JAK2 inhibitors, corticosteroids, and androgen therapies help manage symptoms. You may also require chemotherapy, blood transfusions, or a splenectomy.
Talk to your doctor about your symptoms and always let them know if you’re considering taking a new medication or supplement.