How is thalassemia (congenital hemolysis) treated?

The article was consulted with Master, Doctor Nguyen Thi An - Pediatrician - Neonatologist - Department of Pediatrics - Neonatology - Vinmec Ha Long International General Hospital.

People with congenital hemolytic disease can still have a normal life, get married and have children like everyone else. However, these patients must adhere to a long-term, lifelong treatment with continuous blood transfusions and iron chelation.

According to current medical advances, in the world there are two methods to cure the disease. The first is a human hematopoietic stem cell transplant that perfectly matches the patient. This method has been implemented in many countries around the world, including Vietnam. The second is gene therapy. However, this method is currently only being carried out in a step-by-step study on volunteer patients.

According to an expert of the World Thalassemia Federation, the worrying situation about congenital hemolytic disease is that if left untreated, the disease causes death very early in childhood, inadequate treatment can lead to death. died between the ages of 15 and 20. However, the cost of treating congenital hemolytic disease is very expensive.
Therefore, patient management and disease prevention are urgent issues in order to reduce the birth rate of children carrying the gene and having congenital hemolytic disease.

Currently, the main treatment methods for hemolytic anemia are blood transfusion and iron chelation. Patients have to go to the hospital for blood transfusion periodically, on average once a month.
Continuous blood transfusion: Depending on the severity of the disease, the doctor will announce the necessary time for a blood transfusion. Periodic chelation: Your doctor will give you medications or injections to chelate iron periodically to reduce complications. For patients with congenital G6PD deficiency, it is necessary to avoid using drugs or foods with oxidizing agents such as antimalarials, quinolones, peas...
In addition to the two main treatment methods, blood transfusion and iron chelation, at present, Vinmec Times City International Hospital has deployed and successfully implemented stem cell transplantation to cure congenital hemolysis. This is a method that helps to treat the root cause and cure the disease for the patient.
One of the prerequisites for a successful transplant is the post-transplant care conditions, which require state-of-the-art and modern equipment. To avoid the risk of infection right after the transplant, the baby care conditions in the isolation area of ​​Vinmec International General Hospital are also strictly followed: All blood products are irradiated and filtered. Even your baby's food must be irradiated with ultraviolet light to kill all bacteria. During the post-transplant period, pediatric patients received prophylactic treatment of fungi, bacteria and viruses, combined with maintenance immunosuppressive drugs.
Professor - Dr. Nguyen Thanh Liem - Head of High-Tech Unit for Autism and Cerebral Palsy Treatment and Director of Vinmec Institute of Stem Cell and Gene Technology said: "There are about 10 million people in Vietnam currently. people with Thalassemia and carry the gene for this disease. In addition to the efforts of regulatory agencies and experts in counseling, screening, disease detection, and patient management, the success of Thalassemia stem cell transplantation at Vinmec is entirely attributable to the success of the stem cell transplant method. could open up the prospect of a radical cure for this disease.”
Currently in Vietnam, a number of cord blood banks have begun to develop. At Vinmec, there are more than 1,300 cord blood samples stored and preserved according to international standards; At the same time, the stem cell transplant area is equipped with a modern sterile room system to create optimal conditions for successful transplants.