Thalassemia can be cured with stem cell transplantation. Stem cells are taken from the bone marrow or blood from the donor and transplanted in the patient although, an important curative therapy, this therapy has a limited access to patients due to various reasons.
Stem cells are the cells through which all the cells develop. Stem cells present in the bone marrow gives rise to various types of cells. Various types of cells emerging from the hematopoietic stem cells includes red blood cells which are used to deliver the oxygen to various tissues, white blood cells responsible for maintaining the immune system of the body and the platelets which are used for clotting and wound healing. There are two types of stem cell transplantation for thalassemia:
Bone Marrow Transplantation: Bone marrow is the place situated in the body of the long bones and is responsible for the production of various types of cells through stem cells. If the stem cells, which are to be transplanted in the body of the patient, are taken from the bone marrow, the process is known as bone marrow transplant.
Blood Stem Cell Transplant: When the cells, which are to be transplanted, are taken from the blood of the donor, the process is known as blood stem cell transplant.
Allogenic hematopoietic stem cell transplantation is an important treatment option for the management of thalassemia. But due to various factors, it does not adequately serve the purpose of curing patients suffering from thalassemia. Following are the hurdles which reduces the availability of this approach:
Matching Donor: Approximately half of the patients waiting for stem cell transplantation does not have access to this process due to lack of suitable donor. Suitable donor is a prerequisite to avoid severe immunological reaction which may occur inside the patient.
Less Service Providers: Stem cell transplantation is a highly specialized service and very few hospitals offer this service. The transplantation service hospitals are inadequate, reducing the number of patients getting benefit from this therapy.
High Cost: High cost is also an important factor as the cost is so high that most of the patients are not able to afford the therapy.
Due to the above factors, only 10% of the patients are able to undergo the stem cell transplant therapy for thalassemia.
Various steps are followed while doing stem transplant:
Damaging The Old Bone Marrow: As the original bone marrow becomes abnormal, thus it has to be destroyed so as to transplant a new bone marrow. The drug generally used to damage the old bone marrow is busulfan.
Immunosuppressive Action: As the new bone marrow or the new stem cells are the cells from the other person, thus the immune system of the patient will initiate immunogenic reaction against the transplanted cells thereby killing those cells. Thus, to make the stem cells survive and grow, immunosuppressant drugs are given to the patient. The drug generally used in cyclophosphamide.
Transplanting Stem Cells: The stem cells from the body of the donor are transplanted to the patient.
The treatment should be initiated as soon as possible as the thalassemia managed for longer time through frequent blood transfusions causes iron overload. Further, the resulting severe anemia may cause organ failure. The organ damage may also be caused by iron accumulation. The transfusion-transmission infection also occurs which is also the cause of fatality. If the therapy is provided in the early stage, it may lead to good prognosis and 90% of the patients able to survive. The cure rate of 70%-80% is also found in high risk patients. The high risk patients are those patients which are over 7 years of age and have related complications such as hepatomegaly.
There is a huge mismatch between demand and supply in terms of the therapy as only few people are able to access this therapy and various measures should be adopted to narrow this gap.
Patient suffering from thalassemia can be cured with stem cell transplant. But various reasons such as unavailability of matching donor, inadequate service provider and high cost has limited the access for this therapy.
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