European journal of haematology · 2020
Hematopoietic cell transplantation for Diamond Blackfan anemia: A report from the Pediatric Group of the Brazilian Bone Marrow Transplantation Society.
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Darrigo Luiz Guilherme, Loth Gisele, Kuwahara Cilmara, Vieira Ana, Colturato Vergilio, Rodrigues Ana Luiza, Arcuri Leonardo, Fernandes Juliana, Macedo Antonio, Tavares Rita, Gomes Alessandra, Ribeiro Lisandro, Seber Adriana, Zecchin Victor, de Souza Mair, Calixto Rodolfo, Pasquini Ricardo, Flowers Mary, Rocha Vanderson, Bonfim Carmem
Аннотация
OBJECTIVES: The aim of this study was to analyze the outcomes of children with Diamond-Blackfan anemia (DBA) treated in Brazil with hematopoietic cell transplantation (HCT).
METHODS: We performed a retrospective analysis of 44 pediatrics patients transplanted between 1990 and 2018. The median age of patients was 5 years, and 57% were male. Twenty-five received their first HCT from an HLA-matched sibling donor (MSD), 12 from a HLA matched unrelated bone marrow donor (MUD 10/10, n = 12) and 7 other HLA mismatched donors (MMD).
RESULTS: After a median follow-up of 4 years, estimate 5-year overall survival (OS) for the entire cohort was 70%, 80% for MSD group, 73% for MUD, and 29% for MMD. Thirty-eight out of the 44 evaluable patients engrafted successfully. Primary and secondary graft failure was observed in five and three patients, respectively. Rates of grade II-IV and III-IV acute graft-versus-host disease (aGVHD) were 25% and 18%, respectively. Nine patients developed chronic GVHD (cGVHD).
CONCLUSION: Overall survival rates observed after HLA matched donors transplant for DBA were comparable to those reported from higher-income countries and international registries.
METHODS: We performed a retrospective analysis of 44 pediatrics patients transplanted between 1990 and 2018. The median age of patients was 5 years, and 57% were male. Twenty-five received their first HCT from an HLA-matched sibling donor (MSD), 12 from a HLA matched unrelated bone marrow donor (MUD 10/10, n = 12) and 7 other HLA mismatched donors (MMD).
RESULTS: After a median follow-up of 4 years, estimate 5-year overall survival (OS) for the entire cohort was 70%, 80% for MSD group, 73% for MUD, and 29% for MMD. Thirty-eight out of the 44 evaluable patients engrafted successfully. Primary and secondary graft failure was observed in five and three patients, respectively. Rates of grade II-IV and III-IV acute graft-versus-host disease (aGVHD) were 25% and 18%, respectively. Nine patients developed chronic GVHD (cGVHD).
CONCLUSION: Overall survival rates observed after HLA matched donors transplant for DBA were comparable to those reported from higher-income countries and international registries.
Предметные рубрики MeSH
Anemia, Diamond-BlackfanBone Marrow TransplantationBrazilChildChild, PreschoolDisease ManagementFemaleGraft vs Host DiseaseHLA AntigensHealth Care SurveysHematopoietic Stem Cell TransplantationHistocompatibility TestingHumansInfantMaleRetrospective StudiesSeverity of Illness IndexSiblingsUnrelated Donors