Outcomes of Allogeneic Hematopoietic cell transplantation in patients with Dyskeratosis Congenita

Biology of Blood and Marrow Transplantation

Volumn 19, Issue 8, 2013, Pages 1238-1243

  Gadalla, Shahinaz M ^a   Sales Bonfim, Carmem ^b   Carreras, Jeanette ^c   Alter, Blanche P ^a   Antin, Joseph H ^d   Ayas, Mouhab ^e   Bodhi, Prasad ^f   Davis, Jeffrey ^g   Davies, Stella M ^h   Deconinck, Eric ⁱ   Deeg, H Joachim ^j   Duerst, Reggie E ^k   Fasth, Anders ^l   Ghavamzadeh, Ardeshir ^m   Giri, Neelam ^a   Goldman, Frederick D ⁿ   Kolb, E Anders ^o   Krance, Robert ^p   Kurtzberg, Joanne ^q   Leung, Wing H ^r   Srivastava, Alok ^s   Or, Reuven ^t   Richman, Carol M ^u   Rosenberg, Philip S ^a   Toledo Codina, Jose Sanchez de ^v   Shenoy, Shalini ^w   Socié, Gerard ^x   Tolar, Jakub ^y   Williams, Kirsten M ^a,z   Eapen, Mary ^c   Savage, Sharon A ^a   more..

^a NATIONAL CANCER INSTITUTE   (United States)

^b FEDERAL UNIVERSITY OF PARANÁ   (Brazil)

^c MEDICAL COLLEGE OF WISCONSIN   (United States)

^d DANA FARBER CANCER INSTITUTE   (United States)

^e KING FAISAL SPECIALIST HOSPITAL AND RESEARCH CENTER   (Saudi Arabia)

^f CASE WESTERN RESERVE UNIVERSITY   (United States)

^g BC CHILDREN S HOSPITAL   (Canada)

^h CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

ⁱ UNIVERSITY HOSPITAL JEAN MINJOZ   (France)

^j Fred Hutchinson Cancer Research Center   (United States)

^k CHILDREN'S MEMORIAL HOSPITAL   (United States)

^l UNIVERSITY OF GOTHENBURG   (Sweden)

^m TEHRAN UNIVERSITY OF MEDICAL SCIENCES   (Iran)

ⁿ UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^o NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^p CENTER FOR CELL AND GENE THERAPY   (United States)

^q DUKE UNIVERSITY MEDICAL CENTER   (United States)

^r ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^s CHRISTIAN MEDICAL COLLEGE   (India)

^t HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^u UNIVERSITY OF CALIFORNIA   (United States)

^v HOSPITAL MATERNO INFANTIL VALL D HEBRON   (Spain)

^w ST LOUIS CHILDREN S HOSPITAL   (United States)

^x SAINT LOUIS HOSPITAL   (France)

^y UNIVERSITY OF MINNESOTA MEDICAL CENTER   (United States)

^z GEORGE WASHINGTON UNIVERSITY MEDICAL CENTER   (United States)

more..

Author keywords

Allogeneic transplantation; Dyskeratosis congenita; Long term survival; Pulmonary toxicity

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; MELPHALAN;

ADOLESCENT; ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CASE REPORT; CAUSE OF DEATH; CHILD; COMORBIDITY; DYSKERATOSIS CONGENITA; FEMALE; FOLLOW UP; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HLA MATCHING; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LUNG INSUFFICIENCY; MALE; MORTALITY; NEUTROPHIL; OVERALL SURVIVAL; PRESCHOOL CHILD; RETROSPECTIVE STUDY; SCHOOL CHILD; SKIN CANCER; THROMBOCYTE FUNCTION; WHOLE BODY RADIATION;

ALLOGENEIC TRANSPLANTATION; DYSKERATOSIS CONGENITA; LONG-TERM SURVIVAL; PULMONARY TOXICITY;

ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; DYSKERATOSIS CONGENITA; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; RETROSPECTIVE STUDIES; SURVIVAL ANALYSIS; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84880448795     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2013.05.021     Document Type: Article

References (24)

1. Savage S.A., Bertuch A.A. The genetics and clinical manifestations of telomere biology disorders. Genet Med 2010, 12:753-764.
2. de la Fuente J., Dokal I. Dyskeratosis congenita: advances in the understanding of the telomerase defect and the role of stem cell transplantation. Pediatr Transplant 2007, 11:584-594.
3. Giri N., Lee R., Faro A., et al. Lung transplantation for pulmonary fibrosis in dykeratosis congenita: case report and systematic literature review. BMC Blood Disord 2011, 11:3.
4. Alter B.P., Baerlocher G.M., Savage S.A., et al. Very short telomere length by flow fluorescence in situ hybridization identifies patients with dyskeratosis congenita. Blood 2007, 110:1439-1447.
5. Walne A.J., Bhagat T., Kirwan M., et al. Mutation in the telomere capping complex in bone marrow failure. Haematologica 2013, 98:334-338.
6. Ballew B.J., Yaeger M., Jacobs K., et al. Germline mutations of regulator of telomere elongation helicase1, RTEL1 in dyskeratosis congenita. Hum Genet 2013, 132:473-480.
7. Savage S.A., Alter B.P. The role of telomere biology in bone marrow failure and other disorders. Mech Ageing Dev 2008, 129:35-47.
8. Al-Rahawan M.M., Giri N., Alter B.P. Intensive immunosuppression therapy for aplastic anemia associated with dyskeratosis congenita. Int J Hematol 2006, 83:275-276.
9. Dietz A.C., Orchard P.J., Baker K.S., et al. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant 2011, 46:98-104.
10. Nishio N., Takahashi Y., Ohashi H., et al. Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita. Pediatr Transplant 2011, 15:161-166.
11. Vuong L.G., Hemmati P.G., Neuburger S., et al. Reduced-intensity conditioning using fludarabine and antithymocyte globulin alone allows stable engraftment in a patient with dyskeratosis congenita. Acta Haematol 2010, 124:200-203.
12. Ayas M., Nassar A., Hamideh A.A., et al. Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenital-related BM failure. Bone Marrow Transplant 2013, (DOI:10:1038/bmt2013.35).
13. Przepiorka D., Weisdorf D., Martin P., et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995, 15:825-828.
14. Flowers M.E., Kansu E., Sullivan K.M. Pathophysiology and treatment of graft-versus-host disease. Hematol Oncol Clin North Am 1999, 13:1091-1112. viii-ix.
15. Kaplan E.L., Meier P. Nonparametric estimation from incomplete observations. JAmer Statist Assn 1958, 53:457-481.
16. Gooley T.A., Leisenring W., Crowley J., Storer B.E. Estimation of failure probabilities in the presence of competing risks: new representation of old estimators. Statistics in Medicine 1999, 18:695-706.
17. Rocha V., Devergie A., Socie G., et al. Unusual complications after bone marrow transplantation for dyskeratosis congenita. Br J Haematol 1998, 103:243-248.
18. Alter B.P., Giri N., Savage S.A., Rosenberg P.S. Cancer in dyskeratosis congenita. Blood 2009, 113:6549-6557.
19. Shaw P.H., Haut P.R., Olszewski M., Kletzel M. Hematopoietic stem-cell transplantation using unrelated cord-blood versus matched sibling marrow in pediatric bone marrow failure syndrome: one center's experience. Pediatr Transplant 1999, 3:315-321.
20. Fogarty P.F., Yamaguchi H., Wiestner A., et al. Late presentation of dyskeratosis congenita as apparently acquired aplastic anaemia due to mutations in telomerase RNA. Lancet 2003, 362:1628-1630.
21. Ju Z., Jiang H., Jaworski M., et al. Telomere dysfunction induces environmental alterations limiting hematopoietic stem cell function and engraftment. Nat Med 2007, 13:742-747.
22. Awaya N., Baerlocher G.M., Manley T.J., et al. Telomere shortening in hematopoietic stem cell transplantation: a potential mechanism for late graft failure?. Biol Blood Marrow Transplant 2002, 8:597-600.
23. Rizzo J.D., Curtis R.E., Socie G., et al. Solid cancers after allogeneic hematopoietic cell transplantation. Blood 2009, 113:1175-1183.
24. MacMillan M.L., Wagner J.E. Haematopoeitic cell transplantation for Fanconi anaemia - when and how?. Br J Haematol 2010, 149:14-21.