BMT- Thalassemia

Overview

Thalassemia is described as an inherited blood disorder characterized by fewer red blood cells and lesser haemoglobin than normal. The condition causes anemia, leaving you very exhausted. Mild thalassemia may not require any treatment, but severe conditions of thalassemia may require regular blood transfusions.

Symptoms of severe thalassemia include weakness, extreme exhaustion, facial bone deformities, yellowish or pale skin, abdominal swelling, slow growth and dark urine. Other critical symptoms of severe thalassemia include:

• Slowed growth rate- Anemia causes slow growth in a child and it can cause delay in puberty.
• Bone deformities- The condition may cause abnormal expansion of your bone marrow, thus resulting into abnormal bone structure. The condition makes your bone brittle and thin, increasing chances of broken bones.
• Heart problems- Conditions like abnormal heart rhythms and congestive heart failure are associated with severe thalassemia.
• Enlarged spleen- Thalassemia destroys red blood cells causing the spleen to enlarge and function harder than normal. The spleen plays a significant role in your body by fighting unwanted material and infections.

Diagnosis of severe thalassemia is done with blood tests that reveal low count of red blood cells, smaller than normal red blood cells, pale red blood cells, lower level of haemoglobin, abnormal measure of iron count in blood, etc.

Treatment for severe thalassemia includes frequent blood transfusions and stem cell transplants. A patient requires frequent blood transfusions, possibly every week to get more iron content in blood. However, in the process, extra iron may cause damage to the organs, liver and the heart. It is necessary to take medications along with frequent blood transfusions.

Stem cells transplant (also called bone marrow transplant) is an effective treatment option for severe thalassemia. It can eliminate the need of lifelong drugs and blood transfusions to control iron overload.

Bone marrow transplant for severe thalassemia can be carried out successfully, provided there is a compatible family donor, the patient is below five years of age without a liver enlargement etc. However, older children with high ferritin levels, enlarged spleen or liver will have to undergo intensive treatment along with chelation drugs and hydroxyurea. The success rate of BMT for thalassemia is associated with 80-90% cure probability.

Before going through a stem cell transplant, these are the preparations to be taken:

Before the treatment

• The donor is treated and tested to ensure if there is a close-enough match for the patient. The stem cells collected from the donor are stored and frozen.
• Meantime, the patient is prepared for the treatment. He or she will have to undergo specific tests to ensure a good health condition appropriate for the transplantation. Blood tests for hepatitis virus, deficiency diseases and other infectious agents are done to evaluate the patient’s health condition and status.
• High doses of chemotherapy and radiotherapy are given to destroy diseased stem cells in the bone marrow of the patient.

How it is performed

• The procedure is performed on an outpatient basis. After the patient lies down, an intravenous channel is created in the arm through which the donor’s stem cells that were stored are injected in the patient’s blood.
• The procedure is painless and the patient is usually discharged on the same day.

Recovery

Recovery of BMT thalassemia depends upon a number of factors like physical condition of the person, the severity of side effects of the transfusion, etc. Immediately after the bone marrow transplant, the patient can get back to a normal life. The stem cells infused, travel into the blood stream and it takes about a week before new blood cells start to develop. It feels normal when the new blood cells grow, but if in case of any side effects, it should be consulted with a doctor right away.