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Thalassemia major, also known as Alpha Thalassemia, is a blood disorder characterised by decreased levels of haemoglobin and red blood cells in the patient’s body. It is an inherited problem caused by unhealthy mutations in the DNA. Thalassemia major is marked by symptoms like fatigue, abdominal bloating, weakness and slow growth. Patients suffering from Thalassemia major are likely to have higher levels of iron in their body, which can damage their heart, liver and endocrine system.
Thalassemia is divided into 3 classes based on symptoms and adversity of the patient’s condition. Class 3 thalassemia is marked by iron overload leading to a drastic impact on the patient.
Patients suffering from thalassemia require regular blood transfusion and allogeneic bone marrow transplant is the only treatment. For this, a suitable, HLA matched donor is required. The patient’s siblings are usually a perfect match. The best age for transplant is 2 to 5 years.Children above 2 years of age can be donors for their siblings.  
If the patient does not have any siblings, doctors suggest a haploid identical bone marrow transplant by using a half-matched donor from the patient’s family.
Finding the right donor:
Finding a matching donor is very important for a successful allogeneic bone marrow transplant. These include:

  • HLA-matched relative (most often a sibling)
  • HLA-matched unrelated donor
  • HLA miss-matched family member
  • Unrelated umbilical cord blood

Stem cells used during the procedure are not only collected from the circulating blood but can also be collected from the umbilical cord. These are frozen at birth to be used later. One of the greatest advantages of this is that stem cells taken from the umbilical cord are less mature and can differentiate into different cell types thereby evading the need for a highly compatible tissue type.
Preparing for allogenic transplant
Before undergoing the transplant the patient has to undergo a conditioning regimen that includes procedures like chemotherapy which help to kill all the malignant cells. The transfusion is then started after two days. The stem cells are introduced inside the patient’s body intravenously.
These stem cells then travel to the bone marrow and trigger the formation of new blood cells.
Over the past few years, allogenic bone marrow transplant has helped to save numerous lives. It is better to get the transplant done in early life as this ensures a better outcome. More than 80 per cent of the children with thalassemia can be cured with the help of an allogeneic bone marrow transplant.
 

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