Asunto(s)

Humanos , Masculino , Femenino , Filosofía , Moral , Ética , Alemania

RESUMEN

OBJECTIVES: To evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in a series of patients with inherited complete IFN-gamma receptor 1 (IFNgammaR1) deficiency. STUDY DESIGN: We report 8 patients who received altogether 11 HSCT from family donors, including 10 HLA-identical (5 siblings and 5 relatives) and 1 HLA-haplo-identical donors. Five grafts were T-cell depleted, and conditioning regimens varied in intensity. RESULTS: Four patients died within 8 months after HSCT. Two of these deaths were due to specific complications related to mycobacterial infection. There was no or very low (2%) donor cell engraftment in 2 survivors. Only 2 patients are in full remission of mycobacterial disease 5 years after HSCT. These are the only patients who received non-T-cell-depleted grafts from an HLA-identical sibling after a fully myeloablative conditioning regimen. CONCLUSIONS: HSCT can lead to prolonged remission of mycobacterial disease in children with complete IFNgammaR1 deficiency. However, optimal control of mycobacterial infection before HSCT and the use of a non-T-cell-depleted transplant from an HLA-identical sibling after a fully myeloablative conditioning regimen are recommended.

Asunto(s)

Trasplante de Células Madre Hematopoyéticas , Infecciones por Mycobacterium/complicaciones , Receptores de Interferón/deficiencia , Acondicionamiento Pretrasplante/métodos , Niño , Preescolar , Femenino , Enfermedad Injerto contra Huésped , Humanos , Lactante , Masculino , Estudios Multicéntricos como Asunto , Mutación , Infecciones por Mycobacterium/genética , Infecciones por Mycobacterium/mortalidad , Receptores de Interferón/genética , Resultado del Tratamiento , Receptor de Interferón gamma

Asunto(s)

Psicoanálisis

Asunto(s)

Psicoanálisis