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why stem cell transplants is more effective in children in treating sickle cell Anemia?

why stem cell transplants is more effective in children in treating sickle cell Anemia?

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Expert Solution

Sickle cell anemia/disease (SCA/SCD) is a common blood genetic disorder. It is caused by a haemoglobin gene mutation were two abnormal copies of the gene is transferred from one generation to next generation. The mode of inheritance pattern is autosomal recessive. It is necessary as mentioned that both parents should transfer /pass the mutated copy of the gene to their child to get affected. Sickle cell Anemia leads to many other complications that affect the regular life such as breathing difficulties, edema in hands and feet, pain in abdomen, chest and joints in severe cases it may lead to acute chest syndrome, organ damage, gall stones, one-sided paralysis, blindness.

Pathophysiology of Sickle cell disease:

Loss of elasticity in the red blood cells (RBCs) is the main cause of sickle cell disease. Under normal conditions the cells flow through capillaries without any disturbances, in sickle cell condition due to low oxygen tension RBCs form sickle shape and loss the elasticity which leads to rigidity of the blood cells and unable to pass through the capillaries. This in turn leads to vessel occlusion finally haemolysis (rupture of RBCs).

  1. Treatment Strategy:

The management of the disease includes preventing infections, folic acid supplementation and medication to relieve the pain.

a) Role of Stem cells in Sickle cell disease:

Stem cell transplant is a process that replaces unhealthy cells in the blood to healthy cells. There are many names provided for the same procedure of transplantation methods such as a) hematopoietic sten cell transplantation (HSCT) or b) bone marrow transplantation (BMT), or c) cord blood transplantation. It is stated that all blood and immune cells begin their journey from stem cells in marrow. These stem cells can be collected by two ways 1) from marrow (surgical method) and 2) from peripheral blood (non-surgical method). Currently Hematopoietic stem cell transplantation remains the best way of therapy for SCD, however there are few limitations that needs to be monitored before transplantation such as the lack of human lecukocyte antigen matched donors during HSCT transplantation.

b) Procedure to be followed for proper management of the disease for effective HSCT transplantation : (Workflow-image)

c) Effectiveness of stem cell transplants in children with sickle cell anemia

Stem cell transplantation is more effective in children with proper HLA-matched stem cell transplantation. HSCT provides an excellent success rate especially in children’s with long term protection from clinical manifestations associated vaso-occlusion with SCD. It is also observed that severe patients (children) with SCD undergone transplantation (HSCT) with HLA-identical sibling showed a good long term survival (more than 5 year) exceeds about 90% when compared with the older patients with best supportive care. In research it is evident that in young patients the stem cell transplantation is taken well than in elder patients with good prognosis. Allergenic stem-cell transplantation is best in young patients especially with symptomatic sickle cell disease. In terms of management of the disease in children supportive therapy providing antibiotics and treating the complications at right time, educating the families, with continue medical care will definitely improve the survival rate of the child to 95% and reaching to adulthood.


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