Myelofibrosis is a disorder characterized by scarring and fibrosis of bone marrow that disrupts the production of the blood cells.1 Each patient with this disorder represents a different set of symptoms and may require different treatment options. A single treatment is not helpful to manage all the symptoms. Some patients are not aware of their condition due to the absence of symptoms for many years and may not need treatment at that time. Some patients may require immediate treatment. Bone marrow or stem cell transplantation can cure most many cases with great success rate. However, if the disease has progressed to more deterioration of the organs like spleen and liver, then bone marrow transplantation cannot help.
Can A Bone Marrow Transplant Cure Myelofibrosis?
Bone marrow transplantation is a treatment procedure in which damaged or faulty bone marrow is replaced by healthy bone marrow from a donor.2 Hematopoietic stem cell transplant is the most effective cure for myelofibrosis. The patient is given chemotherapy or radiation therapy in order to remove defective bone marrow. It is beneficial to improve the outcomes of this procedure in myelofibrosis. It is most successful in primary myelofibrosis where another diseased condition of blood is absent.
The donor who has the highest chance of providing benefits for the patient is immediate sibling of the patient. His stem cells or bone marrow matches best with the patient. It limits the number of people who can enjoy the benefits of this technique at the age of 60 years or above.
Bone marrow transplantation is selected for cases of myelofibrosis depending on the signs and symptoms of the patients. The candidate who is selected for this procedure should healthy to face the changes developed in the body after the procedure. If it is selected in cases where the disease is at low risk and survival rate of the patient is 15.4 years after the diagnosis of the disease, it also has side effects that reduce the benefits of the procedure. Some studies state that survival rate after this technique reduces in low risk or intermediate -1 risk disease.
Bone marrow transplantation has the most beneficial effects in intermediate -2 and high-risk disease. It improves five-year survival rate. It is preferred before the age of 60-67 years as the age increases, there is a subsequent increase in the transplant-related complications. Thus, potential candidates for bone marrow transplant are limited due to the age factor. The complications of this procedure are fever, body pain, urticarial hives, etc.
Some patients who have the high-risk disease are less likely to enjoy the benefits of this procedure if they have abnormally enlarged spleen or who have undergone blood transfusions more than 20. The survival rate in such cases drops down after the procedure. So, this procedure is not recommended for such patients. In such cases, chemotherapy is preferred. Ruxolitinib is a most beneficial remedy for such cases. It does not provide a cure but it can relieve many symptoms of the disease.
The outcomes of bone marrow transplantation are good in favorable cases such as primary myelofibrosis with intermediate 1, intermediate 2 or high-risk patients as discussed above. The survival rate depends upon the impact of the disease on spleen or liver. The healthy the spleen is, the prognosis is much better in the patients after transplantation. About 40-60 % of the patients can survive three to four years more after bone marrow transplantation. If splenectomy is done before this procedure, the survival rate is reported to increase in male patients. but its exact cause is not clear till today. It is also reported that patients who have myelofibrosis after polycythemia vera or essential thrombocythemia live more after transplantation than patients with primary myelofibrosis.
Bone marrow transplantation is the only curative method for myelofibrosis. It is effective in primary myelofibrosis in spleen and liver are not severely damaged. It improves the survival rate of the patients up to 3 to 5 years after the procedure.