Thalassemia

Overview

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Thalassemia is a group of inherited blood disorders that affect the body’s ability to produce hemoglobin, the protein in red blood cells responsible for carrying oxygen. It results in the production of abnormal hemoglobin, leading to anemia (low red blood cell levels) and other health complications.

It results in the production of abnormal hemoglobin, leading to anemia (low red blood cell levels) and other health complications.

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Symptoms

Symptoms Vary Depending on the Type and Severity of Thalassemia

  • Minor or Trait Thalassemia
  • Usually asymptomatic or mild anemia
  • May feel fatigued or weak
  • Moderate to Severe Thalassemia
  • Fatigue, weakness, or tiredness
  • Pale or yellowish skin (jaundice)
  • Delayed growth and development in children
  • Dark urine
  • Facial bone deformities (especially prominent cheekbones or an enlarged forehead)
  • Abdominal swelling (due to an enlarged spleen or liver)
  • Heart problems (e.g., heart failure, arrhythmia)
Complications
  • Iron Overload: Frequent blood transfusions can lead to excess iron in the body, damaging organs like the heart, liver, and pancreas.
  • Bone Deformities: Bone marrow expansion can cause changes in bone structure, especially in the face and skull.
  • Enlarged Spleen (splenomegaly): The spleen works harder to remove defective red blood cells, causing it to enlarge, which can increase anemia.
  • Infection: A damaged spleen increases the risk of infections.
  • Heart Disease: Severe anemia can lead to heart failure or arrhythmias.
  • Delayed Growth: Children may have delayed growth and puberty due to anemia and poor nutrient absorption.
Causes

Thalassemia is Caused by Mutations in the Genes That Control the Production Of Hemoglobin. It is Inherited in an Autosomal Recessive Pattern, Meaning Both Parents Must Carry the Defective Gene to Pass It on to their child. There are two main Type

  • Alpha Thalassemia: A defect in the genes responsible for producing the alpha hemoglobin chain.
  • Beta Thalassemia: A defect in the genes responsible for producing the beta hemoglobin chain.
Prevention
  • Genetic Counseling: Couples with a family history of thalassemia should undergo genetic counseling before conceiving to understand the risk of passing on the condition.
  • Prenatal Testing: Testing during pregnancy (e.g., chorionic villus sampling or amniocentesis) can help determine whether a fetus has thalassemia.
  • There is no way to prevent thalassemia in individuals who already have the genetic mutation, but early diagnosis and treatment can help manage symptoms and complications.
Risk Factors
  • Family History: Having parents who are carriers of thalassemia or who have the condition.
  • Ethnicity: Thalassemia is more common in people of Mediterranean, Middle Eastern, South Asian, and African descent.
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How is it Treated?

Treatment Depends on the Type and Severity of Thalassemia

  • Mild Thalassemia (Thalassemia Minor)
  • May not require treatment but requires monitoring.
  • Moderate to Severe Thalassemia (Thalassemia Major)
  • Regular Blood Transfusions: Typically required every few weeks to maintain normal hemoglobin levels.
  • Iron Chelation Therapy: Used to remove excess iron from the body due to frequent blood transfusions (common medications include deferoxamine, deferiprone, and deferasirox).
  • Folic Acid Supplements: May be prescribed to help in the production of red blood cells.
  • Bone Marrow or Stem Cell Transplant: The only potential cure, especially in younger patients. This procedure replaces defective bone marrow with healthy cells from a donor.
  • Spleen Removal (Splenectomy): In some cases, the spleen may be removed if it becomes overly enlarged and contributes to anemia."
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How is it Diagnosed?

Thalassemia is a hereditary blood disorder marked by defective hemoglobin production, leading to anemia of varying severity. Diagnosis begins with a thorough clinical evaluation, focusing on signs such as pallor, fatigue, jaundice, hepatosplenomegaly, and growth delays in children.

Initial laboratory investigations include a complete blood count (CBC), which often reveals microcytic, hypochromic anemia with low mean corpuscular volume (MCV) and mean corpuscular hemoglobin (MCH). A peripheral blood smear typically shows target cells, anisopoikilocytosis, and nucleated red blood cells. Serum iron and ferritin levels are usually normal or elevated, which helps differentiate thalassemia from irondeficiency anemia.

Hemoglobin electrophoresis is the key diagnostic test. It identifies abnormal hemoglobin variants (like HbA2 or HbF), confirming beta-thalassemia trait or major. In alphathalassemia, electrophoresis may be normal, especially in silent carriers or trait; in such cases, DNA analysis for alpha-globin gene deletions is necessary.

Family history and ethnic background can support suspicion, as thalassemia is more prevalent in individuals from the Mediterranean, Middle East, South Asia, and Africa. Genetic counseling and prenatal diagnosis using chorionic villus sampling or amniocentesis are crucial in families with known mutations.

In conclusion, the diagnosis of thalassemia relies on clinical features, hematologic indices, hemoglobin electrophoresis, and genetic testing to determine the type and severity of the disease.

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FAQs

Alpha thalassemia involves mutations in the genes that produce alpha globin chains, while beta thalassemia involves mutations in the beta globin chain genes.

There is no widespread cure for thalassemia, but bone marrow or stem cell transplants can potentially cure the disease in some cases.

With proper treatment, including blood transfusions and iron chelation therapy, many people with thalassemia can live relatively normal lives, though they may experience complications related to the condition.

Untreated thalassemia can lead to severe anemia, organ damage, and a variety of complications including heart failure, growth problems, and bone deformities.

Yes, prenatal testing such as amniocentesis and chorionic villus sampling can detect thalassemia in the fetus.

Patients should avoid iron-rich foods if they are at risk of iron overload and avoid vitamin C supplements that increase iron absorption unless directed by a doctor.

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