Advanced Thalassemia Treatment In India With High Success Rate
Do you know that lifelong medications and blood transfusions are not the only options for thalassemia? Are you spending sleepless nights thinking about your condition?
Repeated blood transfusions are not the solution to the problem; the cure is temporary. Medicines do not give lifelong protections, and your condition cannot be managed with medications.
If you are looking for a permanent solution, you are at the right place.
A bone marrow transplant can now cure your thalassemia permanently. A bone marrow transplant can regenerate new blood cells and replace the mutated cells.
Without wasting further time, let us learn about thalassemia treatment in India.
What is Thalassemia?
Thalassemia is an inherited disease that happens because of a lack of proper hemoglobin protein in the blood. Hemoglobin carries oxygen throughout the body.
The blood cells containing defective hemoglobin cannot fully carry oxygen to the different parts of the body, resulting in anemia, fatigue, and bone deformities.
There are certain causes of thalassemia. Let us learn about them.
What are the causes of Thalassemia?
When a child receives abnormal hemoglobin genes from the parents, thalassemia can happen. If you receive defective genes from one of your parents, you may develop only a mild type of thalassemia.
If both parents are responsible for your thalassemia, you may get the severe form. Thalassemia is not infectious and cannot be transmitted like a cold or flu.
Now, let’s move on to learn about different types of thalassemia.
What are the types of Thalassemia?
There are mainly two types of thalassemia: alpha and beta-thalassemia.
Hemoglobin is a protein in our body that is produced according to the instructions received from the genes. Proteins contain amino acids, which are chain-like structures, and hemoglobin consists of alpha and beta chains.
The genes consist of sequences of building blocks called nucleotides. If there is a wrong sequence of nucleotides (also called a mutation), defective protein formation occurs. Mutations in alpha-chain and beta-chain genes lead to structural changes in the hemoglobin protein and make it unfit for carrying oxygen.
Alpha thalassemia occurs when the mutated haemoglobin protein contains four genes responsible for producing the alpha chain. Beta thalassemia occurs when there are defects in two copies of the hemoglobin protein that produces the beta chain.
Now, let’s know how thalassemia is diagnosed.
What is the diagnostic test for Thalassemia?
If the doctor suspects that you have thalassemia, he may recommend a blood test. The blood test will tell about the shape of the blood cells and the hemoglobin content in the blood.
Electrophoresis (a technique to separate different types of proteins in the blood) of hemoglobin is done further to detect the type of thalassemia you have.
Genetic testing of the thalassemia of the unborn baby is also possible during the pregnancy of the mother.
Amniocentesis or prenatal testing is done when fluid is collected from the region surrounding the baby in the mother’s womb and checked for chromosomal abnormalities.
What are the symptoms of Thalassemia?
Symptoms of thalassemia include:
- Yellow or pale skin
- Bones deformity
- Enlarged spleen
- Weakness and fatigue
- Dark urine coloration
- Poor appetite
- Stunted growth in children
Thalassemia symptoms generally start showing up after a year of birth.
You may like to get insights into the complications caused by thalassemia.
What are the complications caused by Thalassemia?
Thalassemia may develop complications in the body or the complications may be due to repeated blood transfusion.
- Excessive iron overload: Excessive iron can accumulate in the tissues and cause organ damage. The liver, kidney, and heart may get affected.
- Enlarged Spleen: Spleen has to work harder to remove the defective blood cells from the body, and as a result, it may get affected.
- Bones deformities: The bones of the face may get distorted and puberty may be delayed. Thalassemia causes expansion of the bone of the skull and the face making them deformed and abnormal.
- Heart Problems: Congestive heart failure and abnormal heart rhythms are some of the complications of thalassemia
How Thalassemia is managed?
As a part of the routine, thalassemia is managed by blood transfusion and chelation therapy, but these are not permanent solutions. Let’s learn how they help manage the condition.
The kind of therapy you undergo depends on the degree of the thalassemia.
There is a method of increasing the number of red blood cells to transport oxygen in the body and this is the treatment for anemia. This can be done through a blood transfusion, a very safe, and routine procedure in which you are given blood through a small plastic tube which is inserted into a blood vessel.
Some patients with thalassemia, especially those with thalassemia major, require frequent blood transfusions because the hemoglobin levels produced by the body are very low. Those who have thalassemia intermedia do not have severe symptoms like those with thalassemia major. But they still need blood transfusion, especially in the case of an illness or an infection.
Thalassemia minor or trait patients are not usually dependent on blood transfusions because they either have no anemia or have only mild anemia.
Folic acid also known as B vitamin is often recommended for you if you suffer from anemia. This nutrient is beneficial in the formation of red blood cells. It is often done along with other treatments.
The procedure that is used to eliminate the excess iron brought about by frequent blood transfusion is called chelation therapy.
It is important because excess iron in the blood can harm the body’s organs.
Drugs that are administered in the treatment of the condition are referred to as chelating agents.
There are 3 chelating agents currently available.
One is administered via infusion, whereby the drug is delivered through a tube into the patient’s flesh through a needle and at a rate of 8-12 hours; this is done 5-6 times a week.
Another drug, a tablet or liquid, is given three times a day and is used along with the previous drug to minimize the number of the mentioned infusions.
The third drug is taken once a day in the form of a tablet which can be consumed or dissolved in a liquid.
Every medicine has its positive and negative impacts. Your healthcare provider will help you find out which one will suit you or your child the most.
Best Thalassemia Treatment in India
Bone marrow transplant and gene therapy are the two approaches available in the country and they offer a permanent cure for thalassemia. Let’s learn about them in detail.
Bone marrow transplant is the curative and the best thalassemia treatment in India
The stem cells in the bone marrow will give rise to new blood cells which are healthy. There are two types of bone marrow transplants. One is autologous and another is allogenic.
An autologous transplant involves the collection of bone marrow cells from the hip bones of the patient before transplanting them through central venous catheters ( a tube connecting the collarbone with the central vein of the heart).
HLA (Human Leukocyte Antigen) typing is done before allogeneic transplantation. HLA is a specific protein that is present in the white blood cells of your body and if the HLA matches with the sibling or the patient then allogeneic transplantation is done.
Bone marrow transplantation can completely cure thalassemia. It may take 4-6 months for your body to completely recover from thalassemia. There are a few side effects and complications of a bone marrow transplant.
Conditioning is a process in which defective blood cells are killed using chemotherapy before bone marrow transplantation. This may have side effects like chills, fever, bleeding, fatigue, and skin problems.
Complications of bone marrow transplant include graft versus host disease (GVD). In GVD, the newly transferred bone marrow cells may attack your body cells causing various health problems. Medicines are prescribed by the doctor to suppress this action by the newly grafted cells.
The MHRA has approved a new treatment for people with transfusion-dependent β-thalassemia aged 12 and above after a thorough evaluation of the product’s safety, quality, and efficacy.
A drug has been approved for use following the revolutionary CRISPR technique for which the inventors received the Nobel Prize in 2020.
β-thalassemia belongs to the group of genetic disorders that result from alterations in the hemoglobin genes which is the protein used by red blood cells to transport oxygen. β-thalassemia is mostly seen among people of Mediterranean, South Asia, Southeast Asia, and Middle East origin.
The drug is intended to operate through the correction of the gene in your bone marrow stem cell so that the body can synthesize hemoglobin. For this, stem cells are harvested from the bone marrow, genetically altered in a lab, and then transplanted back into your blood, resulting in a lifelong cure.
What is the typical approach of doctors to treat Thalassemia?
According to the severity of thalassemia, beta-thalassemia can be of two types – thalassemia major and thalassemia intermedia.
Thalassemia intermedia can be treated with less number of blood transfusions and iron chelation therapy, whereas the major type of beta thalassemia requires at least 12 times transfusions in a year along with iron chelation therapy
For both types of thalassemia, the permanent cure is a bone marrow transplant – either autologous or allogenic.