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 journalArticle

60 Citations (Scopus)

Abstract

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
Volume19
Issue number8
DOIs
StatePublished - Aug 2013

Fingerprint

Dyskeratosis Congenita
Cell Transplantation
Transplantation
Unrelated Donors
Transplants
Lung
Mortality
Homologous Transplantation
Graft vs Host Disease
Cyclophosphamide
Siblings
Neutrophils
Stem Cells
Blood Platelets

Keywords

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

ASJC Scopus subject areas

  • Transplantation
  • Hematology

Cite this

Gadalla, S. M., Sales-Bonfim, C., Carreras, J., Alter, B. P., Antin, J. H., Ayas, M., ... Savage, S. A. (2013). Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita. Biology of Blood and Marrow Transplantation, 19(8), 1238-1243. https://doi.org/10.1016/j.bbmt.2013.05.021

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

In: Biology of Blood and Marrow Transplantation, Vol. 19, No. 8, 08.2013, p. 1238-1243.

Research output: Contribution to journalArticle

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 2013, 'Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita', Biology of Blood and Marrow Transplantation, vol. 19, no. 8, pp. 1238-1243. https://doi.org/10.1016/j.bbmt.2013.05.021
Gadalla, Shahinaz M. ; Sales-Bonfim, Carmem ; Carreras, Jeanette ; Alter, Blanche P. ; Antin, Joseph H. ; Ayas, Mouhab ; Bodhi, Prasad ; Davis, Jeffrey ; Davies, Stella M. ; Deconinck, Eric ; Deeg, H. Joachim ; Duerst, Reggie E. ; Fasth, Anders ; Ghavamzadeh, Ardeshir ; Giri, Neelam ; Goldman, Frederick D. ; Kolb, E. Anders ; Krance, Robert ; Kurtzberg, Joanne ; Leung, Wing H. ; Srivastava, Alok ; Or, Reuven ; Richman, Carol M ; Rosenberg, Philip S. ; Toledo Codina, Jose Sanchez de ; Shenoy, Shalini ; Socié, Gerard ; Tolar, Jakub ; Williams, Kirsten M. ; Eapen, Mary ; Savage, Sharon A. / Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita. In: Biology of Blood and Marrow Transplantation. 2013 ; Vol. 19, No. 8. pp. 1238-1243.
@article{614761efa37a4a04af916f93a8fe591b,
title = "Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita",
abstract = "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.",
keywords = "Allogeneic transplantation, Dyskeratosis congenita, Long-term survival, Pulmonary toxicity",
author = "Gadalla, {Shahinaz M.} and Carmem Sales-Bonfim and Jeanette Carreras and Alter, {Blanche P.} and Antin, {Joseph H.} and Mouhab Ayas and Prasad Bodhi and Jeffrey Davis and Davies, {Stella M.} and Eric Deconinck and Deeg, {H. Joachim} and Duerst, {Reggie E.} and Anders Fasth and Ardeshir Ghavamzadeh and Neelam Giri and Goldman, {Frederick D.} and Kolb, {E. Anders} and Robert Krance and Joanne Kurtzberg and Leung, {Wing H.} and Alok Srivastava and Reuven Or and Richman, {Carol M} and Rosenberg, {Philip S.} and {Toledo Codina}, {Jose Sanchez de} and Shalini Shenoy and Gerard Soci{\'e} and Jakub Tolar and Williams, {Kirsten M.} and Mary Eapen and Savage, {Sharon A.}",
year = "2013",
month = "8",
doi = "10.1016/j.bbmt.2013.05.021",
language = "English (US)",
volume = "19",
pages = "1238--1243",
journal = "Biology of Blood and Marrow Transplantation",
issn = "1083-8791",
publisher = "Elsevier Inc.",
number = "8",

}

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

UR - http://www.scopus.com/inward/record.url?scp=84880448795&partnerID=8YFLogxK

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

VL - 19

SP - 1238

EP - 1243

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

IS - 8

ER -