From blood transfusions to gene therapy

Thalassemia is an inherited blood disorder which affects the body’s ability to produce normal amounts of haemoglobin, a key elements in the red blood cells. Haemoglobin enables the red blood cells to carry oxygen to every parts of the body to nbourish the body’s each cells. In thalassemia patients, the bone marrow not able to produce adequate healthy red blood cells, leading to anaemia.

What are the main treatment options for thalassemia?

· Treatment modes are dependent on the type and seriousness.

· Blood transfusions are the primary mode of treatment for moderate to severe disease.

· Patients receiving regular blood transfusions have the risk of complications like iron overload, alloimmunization (a harmful immune reaction), infections, Cardiac failure, Liver failure.

· Those receiving transfusions might require simultaneous treatment to remove excess iron before it causes organ damage, known as chelation therapy.

How is thalassemia treated?

# Blood transfusion: Most commonly used method of treating thalassemia is to supply adequate blood to the body with more red blood cells to carry oxygen. Requirements of transfusion varying patient to patient, and its gradually increasing as the increasing age.

# Iron chelation: Blood transfusions come with the danger of causing iron overload. Iron chelation is a process of removal of excess iron from the body, since too much iron could damage organs. Those receiving frequent transfusions will need iron chelation therapy which can be taken as a tablet and also as injections form.

# Folic acid supplements: Often, thalassemia patients are prescribed vitamin B supplements, also known as folic acid, to help treat anaemia. Folic acid helps red blood cells to develop. Treatment with folic acid is usually given simultaneously with other therapies.

# Stem cell transplant (bone marrow transplant): Such transplant from a compatible donor could provide a cure of the thalassemia. Its always better to do stem cell transplant with siblings who are 10/10 HKA match. During transplant procedure healthy stem cells collected form the donor and after condition of the patient, the stem cells infused to the patient. The transplanted cells usually start making healthy blood cells within 15 - 20 days.

# Gene therapy: gene therapy is also a promising treatment modalities, now coming, in near future it will also helps the thalassemia major patient, to lead good quality of life.