Paediatric Bone Marrow Transplant

Inherited blood disorders such as Thalassemia and Sickle Cell Disease (SCD) significantly impact the production and function of haemoglobin, the protein in red blood cells responsible for oxygen transport. Thalassemia affects the body's ability to produce haemoglobin. There are two main types: alpha and beta-thalassemia. The severity of symptoms varies depending on the type and number of gene mutations inherited. Individuals may experience mild to severe anaemia, leading to symptoms like fatigue, weakness, and shortness of breath. Management includes regular blood transfusions and chelation therapy to remove excess iron from the body. SCD is a group of inherited disorders that cause abnormal haemoglobin. The most common form is Sickle Cell Anaemia. In SCD, red blood cells become sickle-shaped, stiff, and sticky, leading to a shortage of healthy red blood cells. These sickle cells can block blood flow in small vessels, causing severe pain and complications such as infections, acute chest syndrome, and stroke. Both Thalassemia and SCD can be cured with Bone Marrow Transplant to improve quality of life and longevity.
Inborn errors of metabolism (IEM) are a group of rare genetic disorders caused by defects in specific enzymes that affect the body's ability to convert food to energy. These enzyme deficiencies can lead to the accumulation of toxic substances or the deficiency of critical compounds, disrupting normal bodily functions. A few of them are Lysosomal storage disorders, Galactosemia, Maple syrup urine disease (MSUD), Phenylketonuria (PKU). Symptoms vary widely but may include developmental delays, weight loss, neurological issues, feeding difficulties, failure to thrive. Diagnosis typically involves newborn screening, genetic testing, and metabolic assays. Treatment depends on the specific disorder and may include dietary modifications, enzyme replacement therapy, and medications to manage symptoms and prevent complications.
Aplastic anaemia is a rare but serious disorder that disrupts the bone marrow's ability to produce enough blood cells. This malfunctioning bone marrow leads to deficiencies in red blood cells (causing fatigue), white blood cells (increasing infection risk), and platelets (raising the risk of bleeding). Aplastic anaemia has no universal cure, but treatments like blood transfusions and medications can help manage symptoms. In some cases, a stem cell transplant may offer a potential cure.
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Leukaemia is a type of blood cancer that affects the white blood cells. It primarily starts in the bone marrow, the site where blood cells are produced. The most common types of leukaemia acute lymphoblastic leukaemia (ALL), acute myeloid leukaemia (AML), and chronic myeloid leukaemia (CML). It is crucial to consult a doctor for accurate diagnosis and treatment options specific to the type you might have.
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Our superspecialist doctors provide the highest quality of care through a team-based, doctor-led model. Trained at some of the world's most renowned institutions, our highly experienced doctors are distinguished experts in their respective specialities. Our doctors work full-time and exclusively across Medanta hospitals. In addition to offering superspecialised care in their own field, the Medanta organisational structure enables every doctor to help create a culture of collaboration and multispecialty care integration.
Our superspecialist doctors provide the highest quality of care through a team-based, doctor-led model. Trained at some of the world's most renowned i..... Continue Reading