Stem cell transplantation for primary immunodeficiency diseases

stem cell transplantation for treatment of primary immunodeficiency disorders

primary immunodeficiencies constitute a group of highly complex congenital disorders most of which are characterized by a very poor prognosis. allogeneic hematopoietic stem cell transplantation (hsct) has become an established curative treatment approach in many of these disorders, which may be permanently corrected. in this presentation basic and practical aspects of hsct are presented, with a...

Stem Cell Transplantation for Primary Immunodeficiency

Stem cell transplantation for treatment of primary immunodeficiency disorders.

Primary Immunodeficiencies constitute a group of highly complex congenital disorders most of which are characterized by a very poor prognosis. Allogeneic hematopoietic stem cell transplantation (HSCT) has become an established curative treatment approach in many of these disorders, which may be permanently corrected. In this presentation basic and practical aspects of HSCT are presented, with a...

Hematopoietic Stem Cell Transplantation for Thalassemia

Thalassemia is an autosomal recessive disorder associated with defective synthesis of the α- or β-chain of hemoglobin. For β-thalassemia major patients, therapeutic options are either monthly red cell transfusions and chelation therapy or allogeneic stem cell transplant. Stem cell transplant is the only curative approach and success is inversely correlated with the degree of iron overload and h...

Stem cell transplantation for rheumatic autoimmune diseases

Immunoablative therapy and hematopoietic stem cell transplantation (HSCT) is an intensive treatment modality aimed at 'resetting' the dysregulated immune system of a patient with immunoablative therapy and allow outgrowth of a nonautogressive immune system from reinfused hematopoietic stem cells, either from the patient (autologous HSCT) or a healthy donor (allogeneic HSCT). HSCT has been shown...