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J Pediatr Hematol Oncol ; 39(7): e394-e398, 2017 10.
Artigo em Inglês | MEDLINE | ID: mdl-28538506

RESUMO

Although bone marrow failure in patients with dyskeratosis congenita (DKC) can be successfully treated with allogeneic hematopoietic cell transplantation (allo-HCT) using a reduced intensity conditioning (RIC) regimen, the outcome of nonhematological disorders in patients with DKC treated with allo-HCT using RIC has not been fully elucidated. Here, we describe the clinical course of nonhematological disorders after allo-HCT with RIC in 3 consecutive patients with DKC. Allo-HCT with RIC was feasible in all cases; however, patient 1 developed lethal pulmonary disease and patient 2 experienced progression of hepatic fibrosis. Careful follow-up of patient-specific complications is required after allo-HCT in patients with DKC.


Assuntos
Disceratose Congênita/terapia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Adolescente , Aloenxertos , Pré-Escolar , Progressão da Doença , Disceratose Congênita/complicações , Humanos , Cirrose Hepática/etiologia , Pneumopatias/etiologia , Masculino , Condicionamento Pré-Transplante
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