Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases
Inherited metabolic diseases (IMD) belonging to lysosomal storage disorders (Hurler syndrome, Krabbe disease, metachromatic leukodystrophy, and others) and peroxisomal disorders (adrenoleukodystrophy) cause progressive degeneration of the nervous system, neurocognitive deterioration, disabilities, and premature death. Umbilical cord blood transplantation (UCBT) leads to development of donor-derived cells in the brain and other organs resulting in stabilization of disease and prevention of further damage. This chapter describes preclinical studies, treatment options, comparative analysis of outcomes for cord blood and bone marrow transplant, current controversies, and future directions. Umbilical cord blood (UCB) provides rapid and increased access to transplantation with favorable outcomes. Data from our center as well as other centers will be reviewed to show that the morbidity, quality of life, and survival outcomes are significantly better if transplant is performed early in the course of the disease.
