Shalini Shenoy1, Jaap J Boelens. 1. aDepartment of Pediatrics, Washington University School of Medicine, St. Louis, Missouri, USA bUniversity Medical Center Utrecht, Pediatric Blood and Marrow Transplantation Program cLaboratory Translational Immunology, University Medical Center Utrecht, Utrecht, the Netherlands.
Abstract
PURPOSE OF REVIEW: The role of hematopoietic cell transplantation in non-malignant disorders has increased exponentially with the recognition that multiple diseases can be controlled or cured if engrafted with donor-derived cells. This review provides an overview of advances made in alternative donor transplants for nonmalignant disorders. RECENT FINDINGS: Stem cell sources, novel transplant methods, and sophisticated supportive care have simultaneously made giant strides toward improving the safety and efficacy of hematopoietic cell transplantation. This has led to the utilization of marrow, cord, peripheral blood stem cell and haploidentical stem cell sources, and novel reduced toxicity or reduced intensity conditioning regimens to transplant non-malignant disorders such as immune dysfunctions, marrow failure syndromes, metabolic disorders and hemoglobinopathies. Transplant complications such as graft rejection, infections, and graft versus host disease are better combated in this modern era of medicine, achieving better survival with decreased late effects. These aspects of transplant for non-malignant disorders are discussed. SUMMARY: This review presents the progress made in the realm of hematopoietic cell transplantation for non-malignant disorders. It advocates the consideration of alternative donor transplants in the absence of human leukocyte antigen matched siblings when indicated by disease severity. The ultimate goal is to provide curative transplant options for more patients that can benefit from this intervention, prior to detrimental outcomes.
PURPOSE OF REVIEW: The role of hematopoietic cell transplantation in non-malignant disorders has increased exponentially with the recognition that multiple diseases can be controlled or cured if engrafted with donor-derived cells. This review provides an overview of advances made in alternative donor transplants for nonmalignant disorders. RECENT FINDINGS: Stem cell sources, novel transplant methods, and sophisticated supportive care have simultaneously made giant strides toward improving the safety and efficacy of hematopoietic cell transplantation. This has led to the utilization of marrow, cord, peripheral blood stem cell and haploidentical stem cell sources, and novel reduced toxicity or reduced intensity conditioning regimens to transplant non-malignant disorders such as immune dysfunctions, marrow failure syndromes, metabolic disorders and hemoglobinopathies. Transplant complications such as graft rejection, infections, and graft versus host disease are better combated in this modern era of medicine, achieving better survival with decreased late effects. These aspects of transplant for non-malignant disorders are discussed. SUMMARY: This review presents the progress made in the realm of hematopoietic cell transplantation for non-malignant disorders. It advocates the consideration of alternative donor transplants in the absence of human leukocyte antigen matched siblings when indicated by disease severity. The ultimate goal is to provide curative transplant options for more patients that can benefit from this intervention, prior to detrimental outcomes.
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