Brazilian Consensus Guidelines for Hematopoietic Stem Cell Transplantation - Inborn errors of metabolismo
Keywords:Inborn errors of metabolism, Pediatric patients, Stem cell transplantation
Hematopoietic stem cell transplantation (HSCT) has the potential to cure a significant proportion of patients with malignant and nonmalignant diseases. The main rationale for HSCT in inborn errors of metabolism (IEM) is based on correcting the decreases enzymes by the donor cells within and outside the intravascular compartment.
In this article, Brazilian Group for Pediatric Bone Marrow Transplantation of the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy (SBTMO) provides a review of HSCT indications in IEM.
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