Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita

Shahinaz M. Gadalla, Carmem Sales-Bonfim, Jeanette Carreras, Blanche P. Alter, Joseph H. Antin, Mouhab Ayas, Prasad Bodhi, Jeffrey Davis, Stella M. Davies, Eric Deconinck, H. Joachim Deeg, Reggie E. Duerst, Anders Fasth, Ardeshir Ghavamzadeh, Neelam Giri, Frederick D. Goldman, E. Anders Kolb, Robert Krance, Joanne Kurtzberg, Wing H. LeungAlok Srivastava, Reuven Or, Carol M. Richman, Philip S. Rosenberg, Jose Sanchez de Toledo Codina, Shalini Shenoy, Gerard Socié, Jakub Tolar, Kirsten M. Williams, Mary Eapen, Sharon A. Savage

Research output: Contribution to journalArticlepeer-review

69 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1238-1243
Number of pages6
JournalBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Issue number8
StatePublished - Aug 2013
Externally publishedYes


  • Allogeneic transplantation
  • Dyskeratosis congenita
  • Long-term survival
  • Pulmonary toxicity

ASJC Scopus subject areas

  • Transplantation
  • Hematology


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