Acute graft-versus-host disease (aGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT) in children. Although 30% to 50% of children respond to corticosteroids…
Children who survive hematopoietic stem cell transplantation (HSCT) are at risk for an inordinate number of long-term side effects. Late effects can be secondary to the underlying diagnosis for which…
Five-year survival rates for childhood cancer now exceed 80% and with the significant progress made by the transplant community in developing less toxic conditioning regimens and in the treatment of…
Autologous and allogeneic hematopoietic stem cell transplantation (HSCT) can be used in the management of patients with autoimmune disorders. Experience gained in adults has helped to better define the conditioning…
Advances in immunology have led to a breathtaking expansion of recognized primary immunodeficiency diseases (PID) with over 120 disease-related genes identified. In North America alone more than 1000 children have…
Despite improvements in the management of thalassemia major and sickle cell disease, treatment complications are frequent and life expectancy remains diminished for these patients. Hematopoietic stem cell transplantation (HSCT) is…
The inherited bone marrow failure (BMF) syndromes are characterized by impaired hematopoiesis and cancer predisposition. Most inherited BMF syndromes are also associated with a range of congenital anomalies. Progress in…
Osteopetrosis is the generic name for a group of diseases caused by deficient formation or function of osteoclasts, inherited in either autosomal recessive or dominant fashion. Osteopetrosis varies in severity…
Immunotherapy in the context of hematopoietic stem cell transplantation has been dominated for many years by T-cell- and dendritic-cell-based treatment modalities. During the last decade, insight into the biology of…
Inherited metabolic disorders (IMD) or inborn errors of metabolism are a diverse group of diseases arising from genetic defects in lysosomal enzymes or peroxisomal function. These diseases are characterized by…