Traditional treatments for Myelofibrosis (MF) aim to address the symptoms, treat related anemia, and reduce your spleen. Complementary therapies may also help.

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.

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.

That said, treatments are available to manage the condition. These treatments depend on how your condition progresses. For example, it can lead to anemia, which is a low number of blood cells, as well as thrombocytopenia, which is a decrease in platelets that aid in clotting.

Your doctor may recommend one or more medications to help treat symptoms or complications of MF.

What is the first line of treatment for myelofibrosis?

Medications used to treat MF commonly include corticosteroids, such as prednisone, which reduces inflammation, and JAK2 inhibitors, such as ruxolitinib (Jakafi) and fedratinib (Inrebic). Mutations in the JAK2 gene have been associated with developing MF.

In addition, some people will receive chemotherapy, such as the drug hydroxyurea (Hydrea, Droxia), which helps stop cancer cells from growing and can help reduce symptoms like night sweats and weight loss.

Note that not everyone will respond the same way to JAK2 inhibitors. For example, fedratinib (Inrebic) was approved by the FDA in 2019 to treat adults with intermediate-2 and high risk primary or secondary MF. It’s a highly selective JAK2 kinase inhibitor for people who don’t respond to treatment with ruxolitinib.

Side effects:

MF treatments can cause side effects. They include:

  • Corticosteroids: The side effects include high blood pressure, fluid retention, weight gain, and mood and memory problems. Long-term side effects and risks of corticosteroids include osteoporosis, bone fractures, high blood sugar, insomnia, and increased risk of infections.
  • JAK2 inhibitors: Common side effects 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: Common side effects of chemotherapy include fatigue, hair loss, nausea, vomiting, weight changes, and mood shifts.

Since anemia is a major complication of MF, some treatments will specifically target MF-related anemia.

This includes blood transfusions, which can increase your red blood cell count and lessen symptoms such as fatigue and easy bruising.

Medications can help improve blood cell counts. These include erythropoietin-stimulating agents (ESAs), androgens, and immunomodulators

Removing the spleen

MF can lead to an enlarged spleen, which can be painful and lead to anemia.

Medications help reduce the size of the spleen, but in some cases, your doctor may recommend surgery to remove it. This procedure is called a splenectomy.

Potential side effects:

Side effects of the treatments for MF-related anemia include:

  • Androgen therapies: These can cause liver damage, facial hair growth in females assigned at birth (FAABs), and growth of prostate cancer in males assigned at birth (MAAB).
  • Immunomodulators: 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.
  • Splenectomy: The removal of the spleen increases the chance of infections and bleeding complications, including blood clots, which can lead to a potentially fatal stroke or pulmonary embolism. Because of the higher chance of infection, you may need to undergo special vaccinations to prevent certain illnesses from developing after the procedure.

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.

An allogeneic 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.

Stem cell transplant side-effects

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 treatment, joining a clinical trial can give you access to newer therapies. Dozens of clinical trials are recruiting or actively evaluating myelofibrosis treatments.

It can also cause an enlarged spleen, abnormal growth of blood cells outside of the bone marrow, and damage to the blood vessels. In approximately 15-20% of cases, it develops into a more serious cancer called acute myeloid leukemia (AML).

Myelofibrosis is a chronic disease that requires medical intervention. No homeopathic or natural cures are proven for 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:

  • iron
  • 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 with your doctor before trying any supplement.

Predicting the outlook and survival of myelofibrosis can be difficult. Many people have had MF for many years without experiencing any symptoms.

Generally, survival varies depending on the type of MF, whether it’s low risk, intermediate-risk, or high risk. The median survival rate is about 5-7 years post-diagnosis.

What is the success rate of myelofibrosis treatment?

The only treatment option that can potentially cure MF is a stem cell transplant.

That said, certain treatments may significantly improve your outlook. A 2022 study of 844 patients found that over about a decade, the median survival increased from 4 to 5 years. Among those treated with ruxolitinib, the median survival rate was found to be 7 years.

Many clinical trials continue to study potential MF treatments.

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 with your doctor about your symptoms, and always let them know if you’re considering taking a new medication or supplement.