Disease-specific hematopoietic cell transplantation: Nonmyeloablative conditioning regimen for dyskeratosis congenita

Bone Marrow Transplantation

Volumn 46, Issue 1, 2011, Pages 98-104

  Dietz, A C ^a   Orchard, P J ^a   Baker, K S ^b   Giller, R H ^c   Savage, S A ^d   Alter, B P ^d   Tolar, J ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b Fred Hutchinson Cancer Research Center   (United States)

^c UNIVERSITY OF COLORADO   (United States)

^d NATIONAL CANCER INSTITUTE   (United States)

Author keywords

dyskeratosis congenita; hematopoietic cell transplantation; reduced intensity conditioning regimen

Indexed keywords

ACICLOVIR; ALEMTUZUMAB; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; GANCICLOVIR; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PREDNISONE; THYMOCYTE ANTIBODY; VALGANCICLOVIR;

ADULT; ARTICLE; AUTOLOGOUS STEM CELL TRANSPLANTATION; CHIMERA; CLINICAL ARTICLE; CLINICAL TRIAL; CYTOMEGALOVIRUS INFECTION; DRUG DOSE REDUCTION; DRUG WITHDRAWAL; DYSKERATOSIS CONGENITA; FEMALE; FOLLOW UP; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HERPES ZOSTER; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LIVING DONOR; LUNG FIBROSIS; MALE; MULTIPLE CYCLE TREATMENT; NONMYELOABLATIVE CONDITIONING; PATIENT CARE; PRIORITY JOURNAL; SINGLE DRUG DOSE; SURVIVAL TIME; UMBILICAL CORD BLOOD; WHOLE BODY RADIATION;

ADOLESCENT; ANTIBODIES, MONOCLONAL; ANTIBODIES, NEOPLASM; ANTINEOPLASTIC AGENTS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; BONE MARROW TRANSPLANTATION; CHILD, PRESCHOOL; COMBINED MODALITY THERAPY; CORD BLOOD STEM CELL TRANSPLANTATION; CYCLOPHOSPHAMIDE; DYSKERATOSIS CONGENITA; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PULMONARY FIBROSIS; TRANSPLANTATION CONDITIONING; VIDARABINE; WHOLE-BODY IRRADIATION; YOUNG ADULT;

EID: 78651373073     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2010.65     Document Type: Article

References (43)

1. Armanios M. Syndromes of telomere shortening. Annu Rev Genomics Hum Genet 2009; 10: 45-61.
2. Walne A, Dokal I. Advances in the understanding of dyskeratosis congenita. Br J Haematol 2009; 145: 164-172.
3. Savage S, Alter B. Dyskeratosis congenita. Hematol Oncol Clin North Am 2009; 23: 215-231.
4. Palm W, de Lange T. How shelterin protects mammalian telomeres. Annu Rev Genet 2008; 42: 301-334.
5. Savage S, Giri N, Baerlocher G, Orr N, Lansdorp P, Alter B. TINF2, a component of the shelterin telomere protection complex, is mutated in dyskeratosis congenita. Am J Hum Genet 2008; 82: 501-509.
6. MacMillan ML, Blazar BR, DeFor T, Ma L, Tolar J, Zierhut H et al. Alternate donor HCT for fanconi anemia (FA): Results of a total body irradiation (TBI) dose de-escalation study. ASH Ann Meet Abst 2008; 112: 298-299.
7. Savage S, Dokal I, Armanios M, Aubert G, Cowen E, Domingo D et al. Dyskeratosis congenita: The first NIH clinical research workshop. Pediatr Blood Cancer 2009; 53: 520-523.
8. Wagner J, Eapen M, MacMillan M, Harris R, Pasquini R, Boulad F et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood 2007; 109: 2256-2262.
9. 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.
10. Alter B, Giri N, Savage S, Rosenberg P. Cancer in dyskeratosis congenita. Blood 2009; 113: 6549-6557.
11. Kirwan M, Dokal I. Dyskeratosis congenita, stem cells and telomeres. Biochim Biophys Acta 2009; 1792: 371-379.
12. Amarasinghe K, Dalley C, Dokal I, Laurie A, Gupta V, Marsh J. Late death after unrelated-BMT for dyskeratosis congenita following conditioning with alemtuzumab, fludarabine and melphalan. Bone Marrow Transplant 2007; 40: 913-914.
13. Yabe M, Yabe H, Hattori K, Morimoto T, Hinohara T, Takakura I et al. Fatal interstitial pulmonary disease in a patient with dyskeratosis congenita after allogeneic bone marrow transplantation. Bone Marrow Transplant 1997; 19: 389-392.
14. Brazzola P, Duval M, Fournet J, Gauvin F, Dalle J, Champagne J et al. Fatal diffuse capillaritis after hematopoietic stem-cell transplantation for dyskeratosis congenita despite low-intensity conditioning regimen. Bone Marrow Transplant 2005; 36: 1103-1105 (author reply 1105).
15. Alter B, Baerlocher G, Savage S, Chanock S, Weksler B, Willner J et al. Very short telomere length by flow fluorescence in situ hybridization identifies patients with dyskeratosis congenita. Blood 2007; 110: 1439-1447.
16. Scharf S, Smith A, Hansen J, McFarland C, Erlich H. Quantitative determination of bone marrow transplant engraftment using fluorescent polymerase chain reaction primers for human identity markers. Blood 1995; 85: 1954-1963.
17. Kaplan E, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc 1958; 53: 457-481.
18. Straathof K, Rao K, Eyrich M, Hale G, Bird P, Berrie E et al. Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: A phase 1/2 study. Lancet 2009; 374: 912-920.
19. Dror Y, Freedman M, Leaker M, Verbeek J, Armstrong C, Saunders F et al. Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita. Bone Marrow Transplant 2003; 31: 847-850.
20. Cossu F, Vulliamy T, Marrone A, Badiali M, Cao A, Dokal I. A novel DKC1 mutation, severe combined immunodeficiency (T+B-NK- SCID) and bone marrow transplantation in an infant with Hoyeraal-Hreidarsson syndrome. Br J Haematol 2002; 119: 765-768.
21. Coman D, Herbert A, McGill J, Lockwood L, Hallahan A. Unrelated cord blood transplantation in a girl with Hoyeraal- Hreidarsson syndrome. Bone Marrow Transplant 2008; 42: 293-294.
22. Ostronoff F, Ostronoff M, Calixto R, Florêncio R, Domingues M, Souto Maior A et al. Fludarabine, cyclophosphamide, and antithymocyte globulin for a patient with dyskeratosis congenita and severe bone marrow failure. Biol Blood Marrow Transplant 2007; 13: 366-368.
23. Ghavamzadeh A, Alimoghadam K, Nasseri P, Jahani M, Khodabandeh A, Ghahremani G. Correction of bone marrow failure in dyskeratosis congenita by bone marrow transplantation. Bone Marrow Transplant 1999; 23: 299-301.
24. Ayas M, Al-Musa A, Al-Jefri A, Al-Seraihi A, Al-Mahr M, Rifai S et al. Allogeneic stem cell transplantation in a patient with dyskeratosis congenita after conditioning with low-dose cyclophosphamide and anti-thymocyte globulin. Pediatr Blood Cancer 2007; 49: 103-104.
25. Güngör T, Corbacioglu S, Storb R, Seger R. Nonmyeloablative allogeneic hematopoietic stem cell transplantation for treatment of Dyskeratosis congenita. Bone Marrow Transplant 2003; 31: 407-410. (Pubitemid 36410689)
26. Nobili B, Rossi G, De Stefano P, Zecca M, Giorgiani G, Perrotta S et al. Successful umbilical cord blood transplantation in a child with dyskeratosis congenita after a fludarabinebased reduced-intensity conditioning regimen. Br J Haematol 2002; 119: 573-574.
27. Kang S, Kirkpatrick J, Halperin E. Low-dose radiation for posttransplant lymphoproliferative disorder. Am J Clin Oncol 2003; 26: 210-214.
28. Ruggeri A, de Latour R, Rocha V, Larghero J, Robin M, Rodrigues C et al. Double cord blood transplantation in patients with high risk bone marrow failure syndromes. Br J Haematol 2008; 143: 404-408.
29. Langston A, Sanders J, Deeg H, Crawford S, Anasetti C, Sullivan K et al. Allogeneic marrow transplantation for aplastic anaemia associated with dyskeratosis congenita. Br J Haematol 1996; 92: 758-765.
30. Chan K, Li C, Worth L, Chik K, Jeha S, Shing M et al. A fludarabine-based conditioning regimen for severe aplastic anemia. Bone Marrow Transplant 2001; 27: 125-128.
31. Sampath S, Schultheiss T, Wong J. Dose response and factors related to interstitial pneumonitis after bone marrow transplant. Int J Radiat Oncol Biol Phys 2005; 63: 876-884.
32. Grochow L, Jones R, Brundrett R, Braine H, Chen T, Saral R et al. Pharmacokinetics of busulfan: Correlation with venoocclusive disease in patients undergoing bone marrow transplantation. Cancer Chemother Pharmacol 1989; 25: 55-61.
33. Samuels B, Bitran J. High-dose intravenous melphalan: A review. J Clin Oncol 1995; 13: 1786-1799.
34. Akasheh M, Freytes C, Vesole D. Melphalan-associated pulmonary toxicity following high-dose therapy with autologous hematopoietic stem cell transplantation. Bone Marrow Transplant 2000; 26: 1107-1109.
35. Rocha V, Devergie A, Socié G, Ribaud P, Espérou H, Parquet N et al. Unusual complications after bone marrow transplantation for dyskeratosis congenita. Br J Haematol 1998; 103: 243-248.
36. Weaver T, Kirk A. Alemtuzumab. Transplantation 2007; 84: 1545-1547.
37. Siegal D, Xu W, Sutherland R, Kamel-Reid S, Kuruvilla J, Lipton J et al. Graft-versus-host disease following marrow transplantation for aplastic anemia: Different impact of two GVHD prevention strategies. Bone Marrow Transplant 2008; 42: 51-56.
38. Landgren O, Gilbert E, Rizzo J, Socié G, Banks P, Sobocinski K et al. Risk factors for lymphoproliferative disorders after allogeneic hematopoietic cell transplantation. Blood 2009; 113: 4992-5001.
39. Myers G, Krance R, Weiss H, Kuehnle I, Demmler G, Heslop H et al. Adenovirus infection rates in pediatric recipients of alternate donor allogeneic bone marrow transplants receiving either antithymocyte globulin (ATG) or alemtuzumab (Campath). Bone Marrow Transplant 2005; 36: 1001-1008.
40. Mathew C. Fanconi anaemia genes and susceptibility to cancer. Oncogene 2006; 25: 5875-5884.
41. Alter B. Cancer in Fanconi anemia, 1927-2001. Cancer 2003; 97: 425-440.
42. Calado R, Young N. Telomere maintenance and human bone marrow failure. Blood 2008; 111: 4446-4455.
43. Fogarty P, Yamaguchi H, Wiestner A, Baerlocher G, Sloand E, Zeng W et al. Late presentation of dyskeratosis congenita as apparently acquired aplastic anaemia due to mutations in telomerase RNA. Lancet 2003; 362: 1628-1630.