Outcome Of Hematopoietic Stem Cell Transplantation For Wiskott-Aldrich Syndrome

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[Hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome].

BACKGROUND Wiskott-Aldrich syndrome (WAS) is an X linked congenital disease that presents as eczema, thrombocytopenia and immune deficiency. The only curative procedure for this illness is hematopoietic stem cell transplant (HSCT), preferably from a healthy HLA identical sibling donor. Cord blood is becoming an excellent alternative as stem cell source from unrelated donors. AIM To report our...

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Brief report Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation

Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the WiskottAldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrant the exploration of new therapeutic strategies. Transplantation studies of a murine model of WAS deficiency have been limited by the occurrence of a radiationinduced fatal exacerbation of a preexisting...

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Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation.

Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the Wiskott-Aldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrant the exploration of new therapeutic strategies. Transplantation studies of a murine model of WAS deficiency have been limited by the occurrence of a radiation-induced fatal exacerbation of a pre-exist...

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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...

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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...

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ژورنال

عنوان ژورنال: Biology of Blood and Marrow Transplantation

سال: 2014

ISSN: 1083-8791

DOI: 10.1016/j.bbmt.2013.12.389