Hematopoietic stem cell transplantation for primary immunodeficiency disease

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...

Pediatric Hematopoietic Stem Cell Transplantation

The introduction and evolution of hematopoietic stem cell transplantation (HSCT) could be traced back to 1950s, to the studies on interactions among irradiation, covering spleen and bone marrow from it and injection of bone marrow cells. Today, HSCT is considered a well-established, effective and promising means of therapy for various malignant and non-malignant medical conditions, both in chil...

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 primary immunodeficiencies.

Allogeneic hematopoietic stem cell transplantation has been shown to be curative for well-described as well as newly discovered immunodeficiencies. However, it is difficulty to define a universal transplant regimen given the rarity of these disorders and the varied pathophysiology these disorders encompass. This article discusses those primary immunodeficiencies most commonly treated by hematop...

Stem Cell Transplantation for Primary Immunodeficiency