Treatment of umbilical cord blood transplantation in pediatric rare diseases

Abstract

Rare diseases are very rare, but usually have severe symptoms.Some rare diseases are life-threa-tening.Most rare diseases cannot be cured.A very small part of these diseases can be cured by hematopoietic stem cell transplantation (HSCT). Umbilical cord blood transplantation(UCBT) is more suitable for children for the weak T cell immunity, the lower request for human leukocyte antigen(HLA) identity type and the lower incidence of graft versus host disease(GVHD). This article reviewed the published data in the treatment of UCBT in primary immunodeficiency disease, inherited metabolic disease, inflammatory bowel disease and bone marrow failure syndrome, in order to improve the level of rare disease treatment by HSCT, especially for UCBT. Key words: Rare diseases; Umbilical cord blood transplantation; Immunodeficiency disease; Inherited metabo-lic diseases; Inflammatory bowel disease; Bone marrow failure