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What Is The Best Treatment For Thalassemia?

Thalassemia refers to an inherited type of blood disorder affecting the ability of a person’s body to produce red blood cells and hemoglobin. The patients thus have limited numbers of red blood cells and relatively less percentage of hemoglobin. In addition, a few patients may have very small red blood cells. Impacts of the disease are mild, moderate, severe and even life threatening.

What Is The Best Treatment For Thalassemia?

What Is The Best Treatment For Thalassemia?

Indeed, the main question that comes in the mind of every individual is that how one should choose the best treatment for thalassemia. For this, doctors have said that the best treatment depends solely on the type and extent of the disease you have. Accordingly, you may undergo with any one or a combination of the following major recommendable best treatment for thalassemia:

Blood Transfusion Method

Blood transfusion method is useful in replenishing different levels of hemoglobin and red blood cells. Patients suffering from thalassemia major require approximately 8 to 12 transfusions in one year. On the other side, if you have the problem in its relatively less severe form, you need maximum 8 transfusions yearly. However, if you experience infection, illness or stress, you may require 1 or 2 additional numbers of blood transfusions.

Excessive Iron Removal or Iron Chelation

Iron chelation or excessive iron removal involves the procedure to remove excess iron content from the thalassemia patient’s bloodstream. This is because; blood transfusions may sometimes result in overload of iron, which further causes damage to lungs, heart, liver and various other organs. Hence, doctors prescribe a medicine called deferoxamine injected beneath the skin or deferasirox to intake orally.

Folic Acid Supplements

Most of the thalassemia patients undergo with iron chelation and blood transfusion treatments may even require additional folic acid supplements. These are helpful in the development of red blood cells effectively.

Stem Cell Transplant or Bone Marrow Transplant

Stem cells or bone marrow cells are able to produce platelets, hemoglobin, white and red blood cells. Because of this, if a person suffers from severe form of alpha or beta thalassemia, doctors recommend for bone marrow transplantation from a compatible bone marrow or stem cell donor. In most of the cases, doctors prefer siblings as the donor and perform the respective transplantation treatment.

Surgical Procedure To Cure The Problem

Even though bone marrow transplantation or stem cell transplantation is preferable in some of the severe cases, it cause bone abnormalities in thalassemia patients. Because of this, doctors have to perform a surgical procedure to resolve it.

Gene Therapy and Genetic Techniques

Scientists are consistently researching for genetic techniques to treat and cure thalassemia. Possibilities in this case include insertion of a normal beta-globin gene in the bone marrow of a patient. Alternatively, doctors suggest for specific drugs to reactivate the respective genes responsible to produce fetal hemoglobin.

Types of Thalassemia

We know that two different beta-globin and four different alpha-globin chains of protein are responsible to form hemoglobin protein component in our blood. Based on this, experts have classified two major types of Thalassemia i.e. alpha thalassemia and beta thalassemia.

Alpha Type of Thalassemia

In case of alpha thalassemia, hemoglobin component present in our blood fails to produce alpha protein in enough amounts. To create alpha-globin chains, we require four genes i.e. two present on each of the chromosomes. A baby gets the required number of gene from each of its parent. In case, any one or more than one among them is missing, baby will suffer from alpha thalassemia. Severity in this regard depends on exact numbers of mutated or faulty genes.

Beta Type of Thalassemia

The formation of beta-globin chain involves two globin genes i.e., one from each of the parents. In case, any one or both of the genes are of faulty, you will suffer from beta thalassemia. Similar to the case of alpha one, here also severity depends on the exact numbers of mutated genes.


  1. Weatherall DJ. The inherited diseases of hemoglobin are an emerging global health burden. Blood. 2010;115(22):4331-4336. doi: 10.1182/blood-2010-01-251348. (https://pubmed.ncbi.nlm.nih.gov/20233974/)
  2. Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. doi: 10.1186/1750-1172-5-11. (https://pubmed.ncbi.nlm.nih.gov/20492708/)
  3. Cao A, Galanello R. Beta-thalassemia. Genet Med. 2010;12(2):61-76. doi: 10.1097/GIM.0b013e3181cd68ed. (https://pubmed.ncbi.nlm.nih.gov/20179624/)
  4. Weatherall DJ. The thalassemias: disorders of globin synthesis. Cold Spring Harb Perspect Med. 2012;2(5):a011700. doi: 10.1101/cshperspect.a011700. (https://pubmed.ncbi.nlm.nih.gov/22553492/)
  5. Viprakasit V, Ekwattanakit S, Riolueang S, et al. Advances in understanding the pathogenesis of non-transfusion-dependent thalassemia. Blood Rev. 2016;30(5):329-336. doi: 10.1016/j.blre.2016.03.003. (https://pubmed.ncbi.nlm.nih.gov/27085958/)

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Team PainAssist
Written, Edited or Reviewed By: Team PainAssist, Pain Assist Inc. This article does not provide medical advice. See disclaimer
Last Modified On:July 25, 2023

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