Stem Cell Transplantation for Treatment of Primary Immunodeficiency Disorders

Primary immunodeficiencies constitute a group of highly complex congenitaldisorders most of which are characterized by a very poor prognosis. allogeneichematopoietic stem cell transplantation (hsct) has become an established curativetreatment approach in many of these disorders, which may be permanently corrected.in this presentation basic and practical aspects of hsct are presented, with anemphasis on its application in lymphocyte disorders such as severe combinedimmunodeficiency (scid). optimal results and outcome of hsct are highlydependant on early and correct diagnosis of these rare disorders, and hsct shouldusually be applied early in the course of the disease in order to prevent irreversiblecomplications from infections. clinical results will be summarized based on recentanalysis performed in large patient cohorts, which have shown steady improvementsand have led to a marked change in the prognosis of patients with primaryimmunodeficiencies.