Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita.

Journal Article (Journal Article)

We describe outcomes after allogeneic transplantation in 34 patients with dyskeratosis congenita who underwent transplantation between 1981 and 2009. The median age at transplantation was 13 years (range, 2 to 35). Approximately 50% of transplantations were from related donors. Bone marrow was the predominant source of stem cells (24 of 34). The day-28 probability of neutrophil recovery was 73% and the day-100 platelet recovery was 72%. The day-100 probability of grade II to IV acute GVHD and the 3-year probability of chronic graft-versus-host disease were 24% and 37%, respectively. The 10-year probability of survival was 30%; 14 patients were alive at last follow-up. Ten deaths occurred within 4 months from transplantation because of graft failure (n = 6) or other transplantation-related complications; 9 of these patients had undergone transplantation from mismatched related or from unrelated donors. Another 10 deaths occurred after 4 months; 6 of them occurred more than 5 years after transplantation, and 4 of these were attributed to pulmonary failure. Transplantation regimen intensity and transplantations from mismatched related or unrelated donors were associated with early mortality. Transplantation of grafts from HLA-matched siblings with cyclophosphamide-containing nonradiation regimens was associated with early low toxicity. Late mortality was attributed mainly to pulmonary complications and likely related to the underlying disease.

Full Text

Duke Authors

Cited Authors

  • Gadalla, SM; Sales-Bonfim, C; Carreras, J; Alter, BP; Antin, JH; Ayas, M; Bodhi, P; Davis, J; Davies, SM; Deconinck, E; Deeg, HJ; Duerst, RE; Fasth, A; Ghavamzadeh, A; Giri, N; Goldman, FD; Kolb, EA; Krance, R; Kurtzberg, J; Leung, WH; Srivastava, A; Or, R; Richman, CM; Rosenberg, PS; Toledo Codina, JSD; Shenoy, S; Socié, G; Tolar, J; Williams, KM; Eapen, M; Savage, SA

Published Date

  • August 2013

Published In

Volume / Issue

  • 19 / 8

Start / End Page

  • 1238 - 1243

PubMed ID

  • 23751955

Pubmed Central ID

  • PMC3736557

Electronic International Standard Serial Number (EISSN)

  • 1523-6536

Digital Object Identifier (DOI)

  • 10.1016/j.bbmt.2013.05.021


  • eng

Conference Location

  • United States