Applicability of and potential barriers preventing allogeneic stem cell transplant in sickle cell patients treated outside a sickle cell program

American Journal of Hematology, EarlyView.
Source: American Journal of Hematology - Category: Hematology Source Type: research

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Authors: Leonard A, Tisdale J Abstract INTRODUCTION: Sickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide, and is a life limiting disease with limited therapeutic options to reduce disease severity. Despite being a monogenic disorder, the clinical phenotypes of SCD are variable, with few reliable predictors of disease severity easily identifying patients where the benefits of curative therapy outweigh the risks. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option, though significant advances in gene therapy raise the promise for additional curative meth...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Authors: Demirci S, Tisdale JF Abstract Derivation of functional and mature red blood cells (RBCs) with adult globin expression from renewable source such as induced pluripotent stem cells (iPSCs) is of importance from the clinical point of view. Definitive RBC generation can only be succeeded through production of true hematopoietic stem cells (HSCs). There has been a great effort to obtain definitive engraftable HSCs from iPSCs but the results were mostly unsatisfactory due to low, short-term and linage-biased engraftment in mouse models. Moreover, ex vivo differentiation approaches ended up with RBCs with mostly...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Publication date: Available online 30 May 2018 Source:Cytotherapy Author(s): Selami Demirci, Naoya Uchida, John F. Tisdale Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensively and...
Source: Cytotherapy - Category: Cytology Source Type: research
Sickle cell disease is the most prevalent monogenic disorder worldwide and curative therapies are limited to hematopoietic stem cell transplant to the few with matched donors. Gene therapy has curative potential, whereby autologous hematopoietic stem cells are genetically modified and transplanted, which would not be limited by matched donors, resulting in 1-time, life-long correction devoid of immune side effects. Significant progress has been made to clinically translate gene therapy for sickle cell disease using lentivirus vectors carrying antisickling genes. This review focuses on the current state of the field, factor...
Source: Pediatric Clinics of North America - Category: Pediatrics Authors: Source Type: research
This article focuses on supportive and preventive care improvements and the benefits of hydroxyurea. Indications for erythrocyte transfusion, hematopoietic stem cell transplant, and gene therapy trials are also summarized.
Source: Pediatric Clinics of North America - Category: Pediatrics Authors: Source Type: research
Pediatric Blood&Cancer, EarlyView.
Source: Pediatric Blood and Cancer - Category: Cancer & Oncology Authors: Source Type: research
Peripheral hematopoietic stem cell mobilization utilizing growth factors in donors with sickle cell trait is safe and effective, Published online: 09 May 2018; doi:10.1038/s41409-018-0208-3Peripheral hematopoietic stem cell mobilization utilizing growth factors in donors with sickle cell trait is safe and effective
Source: Bone Marrow Transplantation - Category: Hematology Authors: Source Type: research
Allogeneic hematopoietic stem cell transplantation (aHSCT) has been administered as a therapeutic modality for a number of genetic disorders, immune deficiency syndromes, and hematologic diseases and malignancies. These conditions include reconstitution of the lymphoid system in patients with congenital immunodeficiency diseases (eg, SCID), as a vehicle for gene therapy for individuals with enzyme deficiencies (eg, Gaucher's disease), as a tolerance protocol for patients in need of tissue transplants (eg, diabetes, kidney), and as rescue and treatment for patients with sickle cell disease, thalassemias, and hematologic can...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Allogeneic hematopoietic stem cell transplantation (aHSCT) has been administered as a therapeutic modality for a number of genetic disorders, immune deficiency syndromes, and hematologic diseases and malignancies. These conditions include reconstitution of the lymphoid system in patients with congenital immunodeficiency diseases (eg, SCID), as a vehicle for gene therapy for individuals with enzyme deficiencies (eg, Gaucher's disease), as a tolerance protocol for patients in need of tissue transplants (eg, diabetes, kidney), and as rescue and treatment for patients with sickle cell disease, thalassemias, and hematologic can...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Hematopoietic stem cell transplantation for adult sickle cell disease in the era of universal donor availibility, Published online: 04 May 2018; doi:10.1038/s41409-018-0193-6Hematopoietic stem cell transplantation for adult sickle cell disease in the era of universal donor availibility
Source: Bone Marrow Transplantation - Category: Hematology Authors: Source Type: research
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