Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients with Dyskeratosis Congenita

被引：92

作者：

Gadalla, Shahinaz M. ^{[1
]}

Sales-Bonfim, Carmem ^{[2
]}

Carreras, Jeanette ^{[3
]}

Alter, Blanche P. ^{[1
]}

Antin, Joseph H. ^{[4
]}

Ayas, Mouhab ^{[5
]}

Bodhi, Prasad ^{[6
]}

Davis, Jeffrey ^{[7
]}

Davies, Stella M. ^{[8
]}

Deconinck, Eric ^{[9
]}

Deeg, H. Joachim ^{[10
]}

Duerst, Reggie E. ^{[11
]}

Fasth, Anders ^{[12
]}

Ghavamzadeh, Ardeshir ^{[13
]}

Giri, Neelam ^{[1
]}

Goldman, Frederick D. ^{[14
]}

Kolb, E. Anders ^{[15
]}

Krance, Robert ^{[16
]}

Kurtzberg, Joanne ^{[17
]}

Leung, Wing H. ^{[18
]}

Srivastava, Alok ^{[19
]}

Or, Reuven ^{[20
]}

Richman, Carol M. ^{[21
]}

Rosenberg, Philip S. ^{[1
]}

de Toledo Codina, Jose Sanchez ^{[22
]}

Shenoy, Shalini ^{[23
]}

Socie, Gerard ^{[24
]}

Tolar, Jakub ^{[25
]}

Williams, Kirsten M. ^{[26
,27
]}

Eapen, Mary ^{[3
]}

Savage, Sharon A. ^{[1
]}

机构：

[1] NCI, Clin Genet Branch, Div Canc Epidemiol & Genet, Bethesda, MD 20892 USA

[2] Univ Fed Parana, Hosp Clin Curitiba, BR-80060000 Curitiba, Parana, Brazil

[3] Med Coll Wisconsin, Ctr Int Blood & Marrow Transplant Res, Dept Med, Milwaukee, WI 53226 USA

[4] Dana Farber Canc Inst, Boston, MA 02115 USA

[5] King Faisal Specialist Hosp & Res Ctr, Dept Pediat Hematol Oncol, Riyadh 11211, Saudi Arabia

[6] Rainbow Babies & Childrens Hosp, Cleveland, OH 44106 USA

[7] British Columbia Childrens Hosp, Dept Paediat, BMT Serv, Vancouver, BC V6H 3V4, Canada

[8] Cincinnati Childrens Hosp, Cincinnati, OH USA

[9] Hop Jean Minjoz, Serv Hematol D, F-25030 Besancon, France

[10] Fred Hutchinson Canc Res Ctr, Seattle, WA 98104 USA

[11] Childrens Mem Hosp, Chicago, IL 60614 USA

[12] Univ Gothenburg, Dept Pediat, Gothenburg, Sweden

[13] Univ Tehran Med Sci, Hematol Oncol & Stem Cell Transplantat Res Ctr, Tehran, Iran

[14] Univ Alabama Birmingham, Birmingham, AL USA

[15] Alfred I Dupont Hosp Children, Wilmington, DE USA

[16] Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX 77030 USA

[17] Duke Univ, Med Ctr, Durham, NC USA

[18] St Jude Childrens Res Hosp, Div Bone Marrow Transplantat, Memphis, TN 38105 USA

[19] Christian Med Coll & Hosp, Dept Haematol, Vellore, Tamil Nadu, India

[20] Hadassah Univ Hosp, Dept Bone Marrow Transplantat, IL-91120 Jerusalem, Israel

[21] Univ Calif Davis, Davis Canc Ctr, Div Hematol Oncol, Sacramento, CA 95817 USA

[22] Hosp Infantil Vall dHebron, Paediat Oncol Haematol & SCT Dept, Barcelona, Spain

[23] Washington Univ, St Louis Childrens Hosp, St Louis, MO 63110 USA

[24] Hosp St Louis, Paris, France

[25] Univ Minnesota, Med Ctr, Minneapolis, MN 55455 USA

[26] George Washington Univ, Med Ctr, Childrens Natl Med Ctr, Ctr Canc & Blood Disorders,Div Blood & Marro Tran, Washington, DC 20037 USA

[27] NCI, Expt Transplantat & Immunol Branch, Bethesda, MD 20892 USA

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2013年 / 19卷 / 08期

关键词：

Dyskeratosis congenita; Allogeneic transplantation; Long-term survival; Pulmonary toxicity; BONE-MARROW FAILURE; TELOMERE BIOLOGY; APLASTIC-ANEMIA; MUTATIONS; ENGRAFTMENT; DISORDERS; DISEASE;

D O I：

10.1016/j.bbmt.2013.05.021

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 transplaneation 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. (C) 2013 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

引用

页码：1238 / 1243

页数：6

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