Myelofibrosis is a chronic life-threatening disorder and is difficult to cure. Various treatment options are available for managing the symptoms of this condition, but the permanent cut of this disease is not available to all the patients. Stem cell transplantation is believed to the permanent cure of myelofibrosis if undergone successfully.
Is Myelofibrosis Curable?
Myelofibrosis is the condition in which there is an increased production of abnormal cells, the megakaryocytes. These megakaryocytes release the inflammatory mediators that result in the formation of fibrous tissue in the bone marrow. The disorder develops due to the abnormal functioning of the stem cells of the bone marrow. The stem cells are the cells present in the bone marrow which are responsible for the formation of blood cells. For most people, there is no cure for this disease. The treatment only alleviates the symptoms and increase the life expectancy and quality of life. However, for some people, the possible cure of myelofibrosis is the stem cell transplantation.
Stem cell transplantation is the process through which new stem cells are transplanted in the bone marrow of the patients and removing the abnormal stem cells. However, this treatment comes with a lot of complications which sometimes may be life-threatening. (1) Following are the complications associated with the stem cell transplantation:
Chemotherapy Side Effects- Side effects of chemotherapy are not tolerated by many individuals, especially the elderly. Chemotherapy is required to kill the previous abnormal stem cells to make a place for new stem cells. The killing of these cancer cells may also include the use of radiotherapy.
Life-Threatening Immune Reactions- The new stem cells Amy attack the healthy tissues of the body leading to permanent damage to the organ. Other complications include the cataract, damage to blood vessels and increased risk of cancer.
Infections- Chemotherapy kills all the stem cells of bone marrow virtually making the body devoid of immune cells. This results in an increased risk of infection in the patients. Further, after the stem cells are transplanted, the doctors lower the immune system of the body leading to further risk of infection.
Thus, the permanent cure of myelofibrosis comes with complications and these complications can even take away life. Two types of stem cells transplants are available. In the autologous transplant, the patient’s own stem cells are transplanted after treating the patient with chemotherapy and sometimes radiotherapy. In the allogeneic transplantation, stem cells of another person are transplanted in the patient.
What Are The Treatment Options For Myelofibrosis?
Treatment of myelofibrosis aims to manage the symptoms of the disease and helps to improve the quality of life. The treatment is strategized on the basis of the age of the disease and the stage of myelofibrosis. In the advanced stage of the disease, palliative care is provided to the patient. Following are the various treatment options available for myelofibrosis:
Cytoreductive Therapies- Cytoreductive therapies are those therapies which help in reducing the production of cells. These drugs reduce the activity of bone marrow. Hydroxyurea is a cytoreductive drug.
Androgen Therapy- Androgen therapy is provided to reduce the symptoms of anemia. These drugs increase the production or RBC.
JAK Inhibitors- JAK inhibitors such as ruxolitinib are indicated to alleviate the symptoms of myelofibrosis. These drugs work in both presences as well as the absence of the JAK mutation.
Radiotherapy- Radiation therapy is sometimes used to kill the cancerous cells. (1)
Immunomodulators- These drugs help in improving the immune system of the body as the WBC are not sufficient to prevent the body from potential infection.
Erythropoietin- Erythropoietin is used to increase the synthesis of RBC so as to manage anemia. (2)
Stem cell transplantation is the only permanent cure for myelofibrosis. But the treatment leads to severe complications due to which death may occur. Complications include chemotherapy side effects, risk of infection and Immune system complications. Stem cells for transplantation may be taken from own body or form other person’s body.