Haploidentical transplantation in pediatric non-malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH).

Torres Canizales, Juan; Ferreras, Cristina; Pascual, Antonia; Alonso, Laura; Regueiro, Alexandra; Plaza, Mercedes; Pérez Hurtado, José María; Benito, Ana; Couselo, José M; Fuster, José L; Díaz-Almirón, Mariana; Bueno, David; Mozo, Yasmina; Gómez López, Alicia; Vicario, José L; Balas, Antonio; Sisinni, Luisa; Díaz de Heredia, Cristina; Pérez-Martínez, Antonio

Torres Canizales, Juan; Ferreras, Cristina; Pascual, Antonia; Alonso, Laura; Regueiro, Alexandra; Plaza, Mercedes; Pérez Hurtado, José María; Benito, Ana; Couselo, José M; Fuster, José L; Díaz-Almirón, Mariana; Bueno, David; Mozo, Yasmina; Gómez López, Alicia; Vicario, José L; Balas, Antonio; Sisinni, Luisa; Díaz de Heredia, Cristina; Pérez-Martínez, Antonio.

Afiliación

Torres Canizales J; La Paz University Hospital, Madrid, Spain.
Ferreras C; La Paz Institute of Health Research, IdiPAZ, Paz University Hospital, Madrid, Spain.
Pascual A; La Paz University Hospital, Madrid, Spain.
Alonso L; La Paz Institute of Health Research, IdiPAZ, Paz University Hospital, Madrid, Spain.
Regueiro A; Hospital Carlos Haya, Málaga, Spain.
Plaza M; Hospital Vall d'Hebron, Barcelona, Spain.
Pérez Hurtado JM; University of Santiago Clinical Hospital, Santiago de Compostela, Spain.
Benito A; Virgen de la Arrixaca University Clinical Hospital, Biomedical Research Institute of Murcia (IMIB), Murcia, Spain.
Couselo JM; Hospital Virgen del Rocio, Sevilla, Spain.
Fuster JL; Hospital of Salamanca, Salamanca, Spain.
Díaz-Almirón M; University of Santiago Clinical Hospital, Santiago de Compostela, Spain.
Bueno D; Virgen de la Arrixaca University Clinical Hospital, Biomedical Research Institute of Murcia (IMIB), Murcia, Spain.
Mozo Y; La Paz University Hospital, Madrid, Spain.
Gómez López A; La Paz University Hospital, Madrid, Spain.
Vicario JL; La Paz University Hospital, Madrid, Spain.
Balas A; Faculty of Medicine, Autonomous University of Madrid, Madrid, Spain.
Sisinni L; Centro de Transfusión de la Comunidad de Madrid, Madrid, Spain.
Díaz de Heredia C; Centro de Transfusión de la Comunidad de Madrid, Madrid, Spain.
Pérez-Martínez A; La Paz University Hospital, Madrid, Spain.

Eur J Haematol ; 106(2): 196-204, 2021 Feb.

Article en En | MEDLINE | ID: mdl-33084101

RESUMEN

OBJECTIVE: Describe the GETH haploidentical stem cell transplantation (haplo-HSCT) activity in non-malignant disease (NMDs). METHODS: We retrospectively analyzed data from children with NMDs who underwent haplo-HSCT. RESULTS: From January 2001 to December 2016, 26 pediatric patients underwent 31 haplo-HSCT through ex vivo T cell-depleted (TCD) graft platforms or post-transplantation cyclophosphamide (PT-Cy) at 7 Spanish centers. Five cases employed unmanipulated PT-Cy haplo-HSCT, 16 employed highly purified CD34+ cells, and 10 employed ex vivo TCD grafts manipulated either with CD3+ CD19+ depletion, TCRαß+ CD19+ selection or naive CD45RA+ T-cell depletion. Peripheral blood stem cells were the sole source for patients following TCD haplo-HSCT, and bone marrow was the source for one PT-Cy haplo-HSCT. The most common indications for transplantation were primary immunodeficiency disorders (PIDs), severe aplastic anemia, osteopetrosis, and thalassemia. The 1-year cumulative incidence of graft failure was 27.4%. The 1-year III-IV acute graft-versus-host disease (GvHD) and 1-year chronic GvHD rates were 34.6% and 16.7%, respectively. The 2-year overall survival was 44.9% for PIDs, and the 2-year graft-versus-host disease-free and relapse-free survival rate was 37.6% for the other NMDs. The transplantation-related mortality at day 100 was 30.8%. CONCLUSION: Although these results are discouraging, improvements will come if procedures are centralized in centers of expertise.

Asunto(s)

Trasplante de Células Madre Hematopoyéticas/estadística & datos numéricos; Trasplante Haploidéntico/estadística & datos numéricos; Factores de Edad; Preescolar; Manejo de la Enfermedad; Femenino; Supervivencia de Injerto; Enfermedad Injerto contra Huésped/diagnóstico; Enfermedad Injerto contra Huésped/etiología; Enfermedad Injerto contra Huésped/prevención & control; Trasplante de Células Madre Hematopoyéticas/efectos adversos; Trasplante de Células Madre Hematopoyéticas/métodos; Humanos; Lactante; Infecciones/etiología; Infecciones/terapia; Masculino; Evaluación de Resultado en la Atención de Salud; Pediatría/métodos; Pautas de la Práctica en Medicina; Pronóstico; Estudios Retrospectivos; España; Quimera por Trasplante; Acondicionamiento Pretrasplante; Trasplante Haploidéntico/efectos adversos; Trasplante Haploidéntico/métodos

Palabras clave

T cell-depleted graft; haploidentical hematopoietic stem cell transplantation; high-dose post-transplantation cyclophosphamide; non-malignant diseases; pediatric

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Trasplante Haploidéntico Tipo de estudio: Diagnostic_studies / Etiology_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Child, preschool / Female / Humans / Infant / Male País/Región como asunto: Europa Idioma: En Revista: Eur J Haematol Asunto de la revista: HEMATOLOGIA Año: 2021 Tipo del documento: Article País de afiliación: España Pais de publicación: Reino Unido

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google