Purpose of Technology
Allogeneic hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD) involves transplantation of allogeneic hematopoietic stem cells from a human leukocyte antigen (HLA)-matched donor. SCD is a genetic disorder characterized by the presence of abnormal hemoglobin known as hemoglobin S. Patients with SCD have abnormally shaped red blood cells, anemia, and often develop severe pain, organ dysfunction, and premature death. HSCT is the only treatment for SCD with a curative intent.
Does allogeneic HSCT improve disease symptoms and prolong survival in pediatric and young adult patients with SCD?
Is allogeneic HSCT equivalent or superior to standard SCD treatments, including hydroxyurea or chronic transfusions in pediatric and young adult patients?
Is allogeneic HSCT for SCD in pediatric and young adult patients safe?
Have definitive patient selection criteria been established for allogeneic HSCT for SCD in pediatric and young adult patients?
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