Diego Medina Valencia, Natalia Builes, Alexis A Franco, Angela Trujillo, Laura Isabel Niño Quiroga, Mauricio Chaparro, Andrés Felipe Escobar-González, Eliana Manzi, Diana Muñoz-Caluce, Estefania Beltran, Angela Devia Zapata, Marcela Estupiñan
{"title":"配对兄弟姐妹和单倍体相同供体造血干细胞移植治疗儿童严重再生障碍性贫血的结果:一项回顾性多中心研究。","authors":"Diego Medina Valencia, Natalia Builes, Alexis A Franco, Angela Trujillo, Laura Isabel Niño Quiroga, Mauricio Chaparro, Andrés Felipe Escobar-González, Eliana Manzi, Diana Muñoz-Caluce, Estefania Beltran, Angela Devia Zapata, Marcela Estupiñan","doi":"10.4103/hemoncstem.HEMONCSTEM-D-24-00037","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Acquired aplastic anemia (AA) is a life-threatening hematologic disorder characterized by bone marrow failure. This study evaluates clinical outcomes of matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT) in pediatric patients with severe (SAA) and very severe aplastic anemia (VSAA) in Colombia.</p><p><strong>Methods: </strong>A retrospective multicenter study was conducted in four high-complexity centers in Colombia between 2011 and 2021, including 59 pediatric patients with SAA/VSAA who underwent allo-HSCT. Eligible patients were divided into two groups: one group underwent MSD-HSCT and the other haplo-HSCT, either as first-line treatment or after immunosuppressive therapy (IST) failure. Clinical outcomes, graft failure, graft-versus-host disease (GVHD), and overall survival (OS) were analyzed.</p><p><strong>Results: </strong>A total of 59 patients with SAA/VSAA undergoing allo-HSCT were included, 29 undergoing MSD-HSCT and 30 undergoing haplo-HSCT. The 2-year OS for the cohort was 81%, with 85% for MSD-HSCT and 75% for haplo-HSCT. Modified Baltimore-based conditioning regimens in haplo-HSCT showed an OS of 83%. Primary graft failure occurred in two haplo-HSCT patients. Grades III-IV acute GVHD and moderate-severe chronic GVHD were more common in haplo-HSCT. Infection was the leading cause of posttransplant mortality.</p><p><strong>Conclusion: </strong>Pediatric SAA patients undergoing MSD-HSCT had better survival outcomes and less incidence of aGVHD compared to haplo-HSCT. Haplo-HSCT with modified Baltimore conditioning offers a viable alternative, particularly when an MSD is unavailable. Prospective studies are needed to confirm these findings.</p>","PeriodicalId":516321,"journal":{"name":"Hematology/oncology and stem cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Outcomes of Matched Sibling and Haploidentical Donors Hematopoietic Stem Cell Transplantation for Pediatric Severe Aplastic Anemia: A Retrospective Multicenter Study.\",\"authors\":\"Diego Medina Valencia, Natalia Builes, Alexis A Franco, Angela Trujillo, Laura Isabel Niño Quiroga, Mauricio Chaparro, Andrés Felipe Escobar-González, Eliana Manzi, Diana Muñoz-Caluce, Estefania Beltran, Angela Devia Zapata, Marcela Estupiñan\",\"doi\":\"10.4103/hemoncstem.HEMONCSTEM-D-24-00037\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Acquired aplastic anemia (AA) is a life-threatening hematologic disorder characterized by bone marrow failure. This study evaluates clinical outcomes of matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT) in pediatric patients with severe (SAA) and very severe aplastic anemia (VSAA) in Colombia.</p><p><strong>Methods: </strong>A retrospective multicenter study was conducted in four high-complexity centers in Colombia between 2011 and 2021, including 59 pediatric patients with SAA/VSAA who underwent allo-HSCT. Eligible patients were divided into two groups: one group underwent MSD-HSCT and the other haplo-HSCT, either as first-line treatment or after immunosuppressive therapy (IST) failure. Clinical outcomes, graft failure, graft-versus-host disease (GVHD), and overall survival (OS) were analyzed.</p><p><strong>Results: </strong>A total of 59 patients with SAA/VSAA undergoing allo-HSCT were included, 29 undergoing MSD-HSCT and 30 undergoing haplo-HSCT. The 2-year OS for the cohort was 81%, with 85% for MSD-HSCT and 75% for haplo-HSCT. Modified Baltimore-based conditioning regimens in haplo-HSCT showed an OS of 83%. Primary graft failure occurred in two haplo-HSCT patients. Grades III-IV acute GVHD and moderate-severe chronic GVHD were more common in haplo-HSCT. Infection was the leading cause of posttransplant mortality.</p><p><strong>Conclusion: </strong>Pediatric SAA patients undergoing MSD-HSCT had better survival outcomes and less incidence of aGVHD compared to haplo-HSCT. Haplo-HSCT with modified Baltimore conditioning offers a viable alternative, particularly when an MSD is unavailable. Prospective studies are needed to confirm these findings.</p>\",\"PeriodicalId\":516321,\"journal\":{\"name\":\"Hematology/oncology and stem cell therapy\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-06-20\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Hematology/oncology and stem cell therapy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-24-00037\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Hematology/oncology and stem cell therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.4103/hemoncstem.HEMONCSTEM-D-24-00037","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Outcomes of Matched Sibling and Haploidentical Donors Hematopoietic Stem Cell Transplantation for Pediatric Severe Aplastic Anemia: A Retrospective Multicenter Study.
Background: Acquired aplastic anemia (AA) is a life-threatening hematologic disorder characterized by bone marrow failure. This study evaluates clinical outcomes of matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and haploidentical HSCT (haplo-HSCT) in pediatric patients with severe (SAA) and very severe aplastic anemia (VSAA) in Colombia.
Methods: A retrospective multicenter study was conducted in four high-complexity centers in Colombia between 2011 and 2021, including 59 pediatric patients with SAA/VSAA who underwent allo-HSCT. Eligible patients were divided into two groups: one group underwent MSD-HSCT and the other haplo-HSCT, either as first-line treatment or after immunosuppressive therapy (IST) failure. Clinical outcomes, graft failure, graft-versus-host disease (GVHD), and overall survival (OS) were analyzed.
Results: A total of 59 patients with SAA/VSAA undergoing allo-HSCT were included, 29 undergoing MSD-HSCT and 30 undergoing haplo-HSCT. The 2-year OS for the cohort was 81%, with 85% for MSD-HSCT and 75% for haplo-HSCT. Modified Baltimore-based conditioning regimens in haplo-HSCT showed an OS of 83%. Primary graft failure occurred in two haplo-HSCT patients. Grades III-IV acute GVHD and moderate-severe chronic GVHD were more common in haplo-HSCT. Infection was the leading cause of posttransplant mortality.
Conclusion: Pediatric SAA patients undergoing MSD-HSCT had better survival outcomes and less incidence of aGVHD compared to haplo-HSCT. Haplo-HSCT with modified Baltimore conditioning offers a viable alternative, particularly when an MSD is unavailable. Prospective studies are needed to confirm these findings.