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Article: Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita

TitleOutcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita
Authors
KeywordsLong-term survival
Allogeneic transplantation
Dyskeratosis congenita
Pulmonary toxicity
Issue Date2013
Citation
Biology of Blood and Marrow Transplantation, 2013, v. 19, n. 8, p. 1238-1243 How to Cite?
AbstractWe 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. © 2013 American Society for Blood and Marrow Transplantation.
Persistent Identifierhttp://hdl.handle.net/10722/294467
ISSN
2022 Impact Factor: 4.3
2020 SCImago Journal Rankings: 2.301
PubMed Central ID
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorGadalla, Shahinaz M.-
dc.contributor.authorSales-Bonfim, Carmem-
dc.contributor.authorCarreras, Jeanette-
dc.contributor.authorAlter, Blanche P.-
dc.contributor.authorAntin, Joseph H.-
dc.contributor.authorAyas, Mouhab-
dc.contributor.authorBodhi, Prasad-
dc.contributor.authorDavis, Jeffrey-
dc.contributor.authorDavies, Stella M.-
dc.contributor.authorDeconinck, Eric-
dc.contributor.authorDeeg, H. Joachim-
dc.contributor.authorDuerst, Reggie E.-
dc.contributor.authorFasth, Anders-
dc.contributor.authorGhavamzadeh, Ardeshir-
dc.contributor.authorGiri, Neelam-
dc.contributor.authorGoldman, Frederick D.-
dc.contributor.authorKolb, E. Anders-
dc.contributor.authorKrance, Robert-
dc.contributor.authorKurtzberg, Joanne-
dc.contributor.authorLeung, Wing H.-
dc.contributor.authorSrivastava, Alok-
dc.contributor.authorOr, Reuven-
dc.contributor.authorRichman, Carol M.-
dc.contributor.authorRosenberg, Philip S.-
dc.contributor.authorToledo Codina, Jose Sanchez de-
dc.contributor.authorShenoy, Shalini-
dc.contributor.authorSocié, Gerard-
dc.contributor.authorTolar, Jakub-
dc.contributor.authorWilliams, Kirsten M.-
dc.contributor.authorEapen, Mary-
dc.contributor.authorSavage, Sharon A.-
dc.date.accessioned2020-12-03T08:22:48Z-
dc.date.available2020-12-03T08:22:48Z-
dc.date.issued2013-
dc.identifier.citationBiology of Blood and Marrow Transplantation, 2013, v. 19, n. 8, p. 1238-1243-
dc.identifier.issn1083-8791-
dc.identifier.urihttp://hdl.handle.net/10722/294467-
dc.description.abstractWe 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. © 2013 American Society for Blood and Marrow Transplantation.-
dc.languageeng-
dc.relation.ispartofBiology of Blood and Marrow Transplantation-
dc.subjectLong-term survival-
dc.subjectAllogeneic transplantation-
dc.subjectDyskeratosis congenita-
dc.subjectPulmonary toxicity-
dc.titleOutcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita-
dc.typeArticle-
dc.description.naturelink_to_OA_fulltext-
dc.identifier.doi10.1016/j.bbmt.2013.05.021-
dc.identifier.pmid23751955-
dc.identifier.pmcidPMC3736557-
dc.identifier.scopuseid_2-s2.0-84880448795-
dc.identifier.volume19-
dc.identifier.issue8-
dc.identifier.spage1238-
dc.identifier.epage1243-
dc.identifier.eissn1523-6536-
dc.identifier.isiWOS:000322607000018-
dc.identifier.issnl1083-8791-

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