Hematopoietic stem cell transplantation for primary immunodeficiency disease.
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TL;DR: The role of pretransplant conditioning in patients with SCID disease in terms of its effects upon T- and B-cell immune reconstitution and late effects is still under debate and will require further study.
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Abstract: Hematopoietic stem cell transplantation is the definitive therapy for a variety of rare primary cellular immunodeficiency syndromes diagnosed in children. All primary immunodeficiencies benefit from early diagnosis and transplantation before the development of serious infections, which contribute to a significant increased risk of mortality following transplant. In the absence of a matched sibling, parental haplocompatible, matched unrelated donor and cord blood stem cells have all been utilized with varying degrees of success and immune reconstitution. The role of pretransplant conditioning in patients with SCID disease in terms of its effects upon T- and B-cell immune reconstitution and late effects is still under debate and will require further study.
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Citations
Themes reported by families as important when proceeding with pediatric hematopoietic stem cell transplantation.
Wendy Pelletier,Pamela S. Hinds,Melissa A. Alderfer,Diane L. Fairclough,Kristin Stegenga,Rebecca D. Pentz +5 more
TL;DR: Theme reported by family members as influencing their acceptance of HSCT as a necessary treatment intervention are explored in this study.
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Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study.
A.A. Hamidieh,Maryam Behfar,Zahra Pourpak,Sara Faghihi-Kashani,Mohammad Reza Fazlollahi,Ashraf sadat Hosseini,M. Movahedi,Mohammad Mozafari,Mostafa Moin,A. Ghavamzadeh +9 more
TL;DR: This study suggests that RIC regimen provides satisfactory rates of successful engraftment and full chimerism and patients with mixed Chimerism were stable in long-term follow-up and this chimerist status offered the potential to resolve symptoms of immunodeficiency.
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Evaluation of the effects of mesenchymal stem cells on neutrophils isolated from severe congenital neutropenia patients
TL;DR: It was demonstrated that MSC co-culture could increase neutrophil lifespan and phagocytic activity of the neutrophils isolated from SCN patients and healthy individuals, and MSCs could be considered as novel candidates for treatment ofSCN patients.
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Chronic Cholangiopathy Associated with Primary Immune Deficiencies Can Be Resolved by Effective Hematopoietic Stem Cell Transplantation.
Nedim Hadžić,Z Nademi,Maesha Deheragoda,Yoh Zen,Reem Elfeky,Austen Worth,Paul Veys,Giorgina Mieli-Vergani,E. Graham Davies +8 more
TL;DR: Effective HSCT has the potential to improve biochemical and histologic features of cholangiopathy in children with PIDs, presumably by clearance of chronic infection following establishment of immune competence, however, careful patient selection is critical as advanced liver injury is often associated with serious complications and mortality.
14
The biology of allogeneic hematopoietic cell resistance.
TL;DR: The factors that contribute to host resistance are reviewed, emerging concepts on the basic biology of resistance are described, and hematopoietic resistance as it relates specifically to patients with severe combined immunodeficiencies (SCID)- disorders are discussed.
14
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