Introduction: Myelofibrosis Treatments
In this article, we will discuss some of the most common Myelofibrosis treatments and what to expect from them. Myelofibrosis is a rare type of blood cancer that affects the bone marrow, the soft tissue inside the bones where blood cells are made. In myelofibrosis, abnormal blood cells multiply and cause scar tissue in the bone marrow, disrupting the normal production of healthy blood cells. This can lead to various problems, such as anaemia (low red blood cell count), thrombocytopenia (low platelet count), splenomegaly (enlarged spleen), and increased risk of infections and bleeding.
Myelofibrosis can occur independently (primary myelofibrosis) or due to another blood disorder (secondary myelofibrosis). It can affect people of any age, but it is more common in people over 50. The exact cause of myelofibrosis is unknown, but it is linked to mutations in certain genes that control blood cell development.
There is no cure for myelofibrosis, but there are treatments that can help manage the symptoms and complications of the disease. The treatment options depend on several factors, such as the severity of the disease, the age and overall health of the patient, and the availability of a suitable stem cell donor.
Stem Cell Transplant
A stem cell transplant is the only Myelofibrosis treatment that can cure or significantly prolong the survival of patients with myelofibrosis. It involves replacing the abnormal stem cells in the bone marrow with healthy stem cells from another person (a donor) with a compatible tissue type. The donor stem cells can come from the blood or the bone marrow.
Before the transplant, the patient receives high doses of chemotherapy and/or radiation therapy to destroy the abnormal stem cells and make room for new ones. This is called conditioning therapy. The patient may also receive medications to suppress their immune system and prevent rejection of the donor cells. This is called immunosuppression therapy in Myelofibrosis treatment.
After the conditioning therapy, the patient receives the donor stem cells through a vein (intravenously). The stem cells travel to the bone marrow and start making new blood cells. This is called engraftment. The patient may need to stay in the hospital for several weeks or months until their blood counts recover and their immune system stabilises.
A stem cell transplant is a complex and risky procedure with serious side effects and complications, such as infections, bleeding, graft-versus-host disease (GVHD), organ damage, infertility, and disease relapse. Therefore, it is not suitable for everyone, and it is usually reserved for younger patients with severe or progressive diseases who have a good chance of finding a matched donor.
Targeted therapy in Myelofibrosis treatment uses drugs that target specific abnormalities or pathways involved in cancer cell growth and survival. Targeted therapy can help reduce the number of abnormal blood cells, shrink an enlarged spleen, improve blood counts, and relieve symptoms such as fatigue, night sweats, itching, and bone pain.
The most commonly used targeted therapy for myelofibrosis is ruxolitinib (Jakafi), which blocks a protein called JAK2 that is overactive in most patients with myelofibrosis. Ruxolitinib can be taken orally (by mouth) twice a day. It can take several weeks or months to see its full effect.
Ruxolitinib can cause side effects such as low blood cell counts, infections, bleeding, weight gain, headache, dizziness, nausea, diarrhoea, and an increased risk of skin cancer. It can also worsen anaemia or thrombocytopenia in some patients. Therefore, patients who take ruxolitinib need regular blood tests and follow-up visits with their doctor.
Other targeted therapies in Myelofibrosis treatment that are being studied or used include fedratinib (Inrebic), momelotinib (Siklos), pacritinib (Pacritinix), navitoclax (Venclexta), imetelstat (GRN163L), and interferon alfa (Intron A, Roferon A). These drugs have different mechanisms of action and may have different benefits and side effects. Some of them are only available through clinical trials.
Another Myelofibrosis treatment is Chemotherapy a treatment that uses drugs that kill or stop the growth of cancer cells. Chemotherapy can help reduce the size of an enlarged spleen, improve blood counts, and relieve symptoms. However, chemotherapy cannot cure myelofibrosis; it can also damage normal cells, causing side effects such as hair loss, nausea, vomiting, mouth sores, infections, bleeding, and fatigue.
The most commonly used chemotherapy for Myelofibrosis treatment is hydroxyurea (Hydrea), which can be taken orally or injected. Hydroxyurea works by interfering with the DNA synthesis of rapidly dividing cells. It can lower the number of abnormal blood cells, platelets, and normal red blood cells and white blood cells. Therefore, patients who take hydroxyurea need regular blood tests and follow-up visits with their doctor.
Other chemotherapy drugs that are sometimes used for Myelofibrosis treatment include busulfan (Myleran), melphalan (Alkeran), cladribine (Leustatin), and azacitidine (Vidaza). These drugs have different mechanisms of action and may have different benefits and side effects. Some of them are only available through clinical trials.
Splenectomy is a surgical procedure that removes the spleen. The spleen is an organ that filters the blood and removes damaged or old blood cells. In Myelofibrosis treatment, the spleen can enlarge and cause symptoms such as abdominal pain, fullness, discomfort, early satiety (feeling full after eating a small amount of food), and weight loss.
Splenectomy can help relieve these symptoms and improve patient’s quality of life with myelofibrosis. However, splenectomy does not cure myelofibrosis, and it can have complications such as bleeding, infection, injury to nearby organs, and an increased risk of blood clots. Therefore, splenectomy is usually reserved for patients with severe or refractory splenomegaly that do not respond to other treatments.
Patients who undergo splenectomy need to take antibiotics and vaccinations to prevent infections. They must also take medications to prevent blood clots and monitor their blood counts regularly.
In Myelofibrosis treatment supportive care is a treatment that aims to improve the symptoms and quality of life of patients with myelofibrosis without directly targeting the disease. Supportive care can include:
- Blood transfusions: Transfusing red blood cells or platelets can help treat anemia or thrombocytopenia and prevent complications such as fatigue, shortness of breath, bleeding, or bruising.
- Growth factors: Injecting substances that stimulate the production of blood cells can help treat anemia or thrombocytopenia. Examples include erythropoietin (EPO), darbepoetin alfa (Aranesp), romiplostim (Nplate), eltrombopag (Promacta), and luspatercept (Reblozyl).
- Medications: Taking drugs that can help relieve symptoms such as pain, itching, inflammation, or infections. Examples include analgesics (painkillers), antihistamines (for itching), corticosteroids (for inflammation), antibiotics (for infections), or anticoagulants (for blood clots).
- Dietary supplements: Taking vitamins, minerals, or herbs that can help improve the general health and well-being of patients with myelofibrosis. Examples include iron, folic acid, vitamin B12, vitamin C, vitamin D, calcium, magnesium, zinc, selenium, omega-3 fatty acids, turmeric, ginger, garlic, green tea, or milk thistle.
- Lifestyle changes: Making changes in the daily habits that can help improve the physical and mental health of patients with myelofibrosis. Examples include eating a balanced diet, drinking enough water, getting enough sleep, exercising regularly, quitting smoking, limiting alcohol intake, managing stress, practicing relaxation techniques, joining a support group, or seeking counseling.
Myelofibrosis is a rare type of blood cancer that affects the bone marrow and causes scar tissue to form in it. This disrupts the normal production of healthy blood cells and causes various problems such as anaemia, thrombocytopenia, splenomegaly, and increased risk of infections and bleeding.
There is no cure for myelofibrosis, but there are Myelofibrosis treatments that can help manage the symptoms and complications of the disease. The treatment options depend on several factors, such as the severity of the disease, the age and overall health of the patient, and the availability of a suitable stem cell donor.