TY - JOUR
T1 - Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita
AU - Gadalla, Shahinaz M.
AU - Sales-Bonfim, Carmem
AU - Carreras, Jeanette
AU - Alter, Blanche P.
AU - Antin, Joseph H.
AU - Ayas, Mouhab
AU - Bodhi, Prasad
AU - Davis, Jeffrey
AU - Davies, Stella M.
AU - Deconinck, Eric
AU - Deeg, H. Joachim
AU - Duerst, Reggie E.
AU - Fasth, Anders
AU - Ghavamzadeh, Ardeshir
AU - Giri, Neelam
AU - Goldman, Frederick D.
AU - Kolb, E. Anders
AU - Krance, Robert
AU - Kurtzberg, Joanne
AU - Leung, Wing H.
AU - Srivastava, Alok
AU - Or, Reuven
AU - Richman, Carol M.
AU - Rosenberg, Philip S.
AU - Toledo Codina, Jose Sanchez de
AU - Shenoy, Shalini
AU - Socié, Gerard
AU - Tolar, Jakub
AU - Williams, Kirsten M.
AU - Eapen, Mary
AU - Savage, Sharon A.
PY - 2013/8
Y1 - 2013/8
N2 - 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.
AB - 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.
KW - Allogeneic transplantation
KW - Dyskeratosis congenita
KW - Long-term survival
KW - Pulmonary toxicity
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UR - http://www.scopus.com/inward/citedby.url?scp=84880448795&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2013.05.021
DO - 10.1016/j.bbmt.2013.05.021
M3 - Article
C2 - 23751955
AN - SCOPUS:84880448795
SN - 1083-8791
VL - 19
SP - 1238
EP - 1243
JO - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
IS - 8
ER -