Allogeneic Hematopoietic Cell Transplantation for Dyskeratosis Congenita: A Report of 3 Cases

J Pediatr Hematol Oncol. 2017 Oct;39(7):e394-e398. doi: 10.1097/MPH.0000000000000844.

Abstract

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.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Allografts
  • Child, Preschool
  • Disease Progression
  • Dyskeratosis Congenita / complications
  • Dyskeratosis Congenita / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Liver Cirrhosis / etiology
  • Lung Diseases / etiology
  • Male
  • Transplantation Conditioning