Topic
Savior sibling
About: Savior sibling is a research topic. Over the lifetime, 336 publications have been published within this topic receiving 6831 citations.
Papers published on a yearly basis
Papers
TL;DR: Children recover from severe aplastic anemia and stem cell transplantations more readily with fewer problems and complications than adults and diminished resistance to infectious agents, primarily viruses and other opportunistic organisms are the primary risk that children who are recovering from these transplantations face.
Abstract: The hematopoietic system of the young child acquires, through time, the ability to cope with exposure to a number of environmental toxins and infectious agents. Occasionally, severe aplastic anemia occurs secondary to exposure to some of these toxins or infectious agents. The occurrence of severe aplastic anemia provides an opportunity to study the maturation of the hematopoietic system because often the immune system is partially intact. Hematopoietic stem cell transplants permit the study of the complete reconstitution of the hematopoietic and immunologic system. Stem cell transplants are often used to treat severe aplastic anemia or, alternatively, may be part of the treatment for an underlying malignant disease or a genetic disease. Sources of stem cells and the age of the recipient and donor have an impact on the success of the stem cell transplant. A stem cell transplantation provides a window of opportunity to study and observe the normal maturation of the immune system and the sensitivity. Very clearly, children recover from severe aplastic anemia and stem cell transplantations more readily with fewer problems and complications than adults. The environmental risks that a child who received a stem cell transplantation faces are related primarily to the deficiencies of the hematopoietic system and immune system during the recovery phase. Therefore, diminished resistance to infectious agents, primarily viruses and other opportunistic organisms, are the primary risk that children who are recovering from these transplantations face. There are few data on the susceptibility of these children to the toxic effects of other environmental toxicants during the recovery period, which may take years before complete recovery.
372 citations
TL;DR: This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.
Abstract: Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.
351 citations
TL;DR: The conversion of sickle-cell anemia to Sickle- cell trait by marrow transplantation in a child with both sicken- cell anemia and acute myeloblastic leukemia is described and the possibility of using marrow transplants is raised.
Abstract: SICKLE-CELL anemia affects 1 in 600 of the U.S. black population and accounts for 80,000 deaths annually throughout the world.1 , 2 Current therapy is designed to prevent sickle-cell crises, but supportive care remains the only treatment available once they occur. Attempts to alter the expression of hemoglobin genes in β-thalassemia and sickle-cell anemia with the use of azacytidine have produced only transient effects.3 This report describes the conversion of sickle-cell anemia to sickle-cell trait by marrow transplantation (done for treatment of leukemia) in a child with both sickle-cell anemia and acute myeloblastic leukemia. It raises the possibility of using marrow transplantation . . .
313 citations
TL;DR: This review identifies the types of tumors for which patients with Fanconi's anemia are at risk and indicates that older patients have an ever‐increasing risk for development of solid tumors.
Abstract: Patients with Fanconi's anemia (FA) are at a high risk for development of malignancies. It is well-known that leukemia occurs in approximately 10% of cases, with increasing risk with age. Less commonly recognized is the risk for myelodysplastic syndromes (approximately 5%); the relationship between myelodysplasia and evolution to leukemia remains speculative. What also needs to be emphasized is that older patients have an ever-increasing risk for development of solid tumors, with at least 5% reported to have liver tumors (male:female ratio, 2:1) and an equal number of other cancers (female:male ratio, 3:1, even after exclusion of gynecologic malignancies). Hematologists have tended to focus on aplastic anemia and leukemia. As FA patients live longer, more of the other malignancies will occur, perhaps related to cord blood or bone marrow transplant, or treatment with cytokines. This review identifies the types of tumors for which patients with Fanconi's anemia are at risk.
264 citations
TL;DR: In this review, the authors summarize the most current knowledge on the pathophysiology, clinical manifestations, and management of this potentially life‐threatening transplantation complication.
Abstract: Hematopoietic stem cell transplantation has evolved as a central treatment modality in the management of different hematologic malignancies. Despite adequate posttransplantation immunosuppressive therapy, acute graft-versus-host disease (GVHD) remains a major cause of morbidity and mortality in the hematopoietic stem cell transplantation setting, even in patients who receive human leukemic antigen (HLA) identical sibling grafts. Up to 30% of the recipients of stem cells or bone marrow transplantation from HLA-identical related donors and most patients who receive cells from other sources (matched-unrelated, non-HLA-identical siblings, cord blood) will develop > Grade 2 acute GVHD despite immunosuppressive prophylaxis. Thus, GVHD continues to be a major limitation to successful hematopoietic stem cell transplantation. In this review, the authors summarize the most current knowledge on the pathophysiology, clinical manifestations, and management of this potentially life-threatening transplantation complication.
226 citations
Related Topics (5)
Performance Metrics
| Year | Papers |
|---|---|
| 2025 | 10 |
| 2024 | 27 |
| 2023 | 16 |
| 2022 | 12 |
| 2021 | 3 |
| 2020 | 3 |