Hematopoietic Stem Cell Transplantation in Pediatric Diseases

Abstract

Since the first successful hematopoietic stem cell transplantation (HSCT) in 1968, HSCT is currently a well-accepted treatment option for not only hematological diseases but also for immunodeficiency syndromes, solid tumors, and metabolic, rheumatological, and neurological disorders. Stem cell therapies are mainly classified as hematopoietic and mesenchymal stem cell therapies. Similar to adults, the main field of hematopoietic stem cell therapy in pediatric age group is hematopoietic stem cell transplantation (HSCT). The conditioning and the graft-versus-host disease (GVHD) prophylactic regimens have improved in the recent years associated with advancement in understanding of transplant biology; and these regimens are increasingly being tailored according to underlying disease, donor type, and stem cell source. The improvements in the supportive care of these patients including those for infectious complications and sinusoidal obstruction syndromes (SOS) have decreased the related morbidities and mortalities. Furthermore, our knowledge about the regenerative potential of hematopoietic stem cells is expanding.