title: Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia.
abstract: OBJECTIVE
  Allogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed.
  
  
  METHODS
  We analyzed data on 113 patients with FA with cytogenetic abnormalities (n = 54), MDS (n = 45), or acute leukemia (n = 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007.
  
  
  RESULTS
  Neutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival (≤ v > 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P = .001). In transplantations from HLA-matched related donors (n = 82), younger patients (≤ v > 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P < .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P = .03) had superior 5-year survival.
  
  
  CONCLUSIONS
  Our analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival.
authors.affiliation: King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia. mouhab@kfshrc.edu.sa
authors.forename: Mouhab
authors.initials: M
authors.lastname: Ayas
authors.forename: Wael
authors.initials: W
authors.lastname: Saber
authors.forename: Stella M
authors.initials: SM
authors.lastname: Davies
authors.forename: Richard E
authors.initials: RE
authors.lastname: Harris
authors.forename: Gregory A
authors.initials: GA
authors.lastname: Hale
authors.forename: Gerard
authors.initials: G
authors.lastname: Socie
authors.forename: Jennifer
authors.initials: J
authors.lastname: LeRademacher
authors.forename: Monica
authors.initials: M
authors.lastname: Thakar
authors.forename: H Joachim J
authors.initials: HJ
authors.lastname: Deeg
authors.forename: Amal
authors.initials: A
authors.lastname: Al-Seraihy
authors.forename: Minoo
authors.initials: M
authors.lastname: Battiwalla
authors.forename: Bruce M
authors.initials: BM
authors.lastname: Camitta
authors.forename: Richard
authors.initials: R
authors.lastname: Olsson
authors.forename: Rajinder S
authors.initials: RS
authors.lastname: Bajwa
authors.forename: Carmem M
authors.initials: CM
authors.lastname: Bonfim
authors.forename: Ricardo
authors.initials: R
authors.lastname: Pasquini
authors.forename: Margaret L
authors.initials: ML
authors.lastname: Macmillan
authors.forename: Biju
authors.initials: B
authors.lastname: George
authors.forename: Edward A
authors.initials: EA
authors.lastname: Copelan
authors.forename: Baldeep
authors.initials: B
authors.lastname: Wirk
authors.forename: Abdullah
authors.initials: A
authors.lastname: Al Jefri
authors.forename: Anders L
authors.initials: AL
authors.lastname: Fasth
authors.forename: Eva C
authors.initials: EC
authors.lastname: Guinan
authors.forename: Biljana N
authors.initials: BN
authors.lastname: Horn
authors.forename: Victor A
authors.initials: VA
authors.lastname: Lewis
authors.forename: Shimon
authors.initials: S
authors.lastname: Slavin
authors.forename: Polina
authors.initials: P
authors.lastname: Stepensky
authors.forename: Marc
authors.initials: M
authors.lastname: Bierings
authors.forename: Robert Peter
authors.initials: RP
authors.lastname: Gale
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D020732:Cytogenetic Analysis; D005199:Fanconi Anemia / Q000235:genetics / Q000601:surgery* / Q000628:therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000235:genetics / Q000601:surgery*; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000097:blood / Q000235:genetics / Q000601:surgery*; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
references.citation: Bone Marrow Transplant. 2008 Jan;41(2):127-32
references.pmid: 18084332
references.citation: Blood. 1996 Jan 1;87(1):386-92
references.pmid: 8547667
references.citation: N Engl J Med. 2006 Oct 5;355(14):1456-65
references.pmid: 17021321
references.citation: Br J Haematol. 2004 Jul;126(2):176-91
references.pmid: 15238138
references.citation: Blood. 2009 Mar 26;113(13):3110-8
references.pmid: 19059878
references.citation: Biol Blood Marrow Transplant. 2007 Sep;13(9):1073-82
references.pmid: 17697970
references.citation: J Clin Oncol. 2002 May 15;20(10):2429-40
references.pmid: 12011120
references.citation: Cancer. 2008 Dec 15;113(12):3315-22
references.pmid: 18831513
references.citation: Biol Blood Marrow Transplant. 2008 Apr;14(4):445-8
references.pmid: 18342787
references.citation: Mutat Res. 2009 Jul 31;668(1-2):141-9
references.pmid: 19508850
references.citation: Haematologica. 2008 Apr;93(4):511-7
references.pmid: 18322251
references.citation: Biol Blood Marrow Transplant. 2008 Oct;14(10):1141-1147
references.pmid: 18804044
references.citation: Haematologica. 2008 Apr;93(4):486-8
references.pmid: 18379006
references.citation: Biol Blood Marrow Transplant. 2007 Dec;13(12):1455-60
references.pmid: 18022575
references.citation: Blood. 2011 Apr 14;117(15):e161-70
references.pmid: 21325596
references.citation: Bone Marrow Transplant. 2008 Aug;42 Suppl 1:S45-S48
references.pmid: 18724300
references.citation: Semin Hematol. 2006 Jul;43(3):147-56
references.pmid: 16822457
references.citation: Pediatr Blood Cancer. 2009 May;52(5):683-5
references.pmid: 19156855
references.citation: Br J Haematol. 2005 Jul;130(1):99-106
references.pmid: 15982351
references.citation: Blood. 2002 Aug 15;100(4):1201-7
references.pmid: 12149198
references.citation: Stat Med. 1999 Mar 30;18(6):695-706
references.pmid: 10204198
references.citation: Leukemia. 1996 Nov;10(11):1700-4
references.pmid: 8892670
references.citation: Bone Marrow Transplant. 1995 Oct;16(4):621-4
references.pmid: 8528182
references.citation: Biol Blood Marrow Transplant. 2006 Jul;12(7):712-8
references.pmid: 16785060
references.citation: Bone Marrow Transplant. 2008 Feb;41(3):261-5
references.pmid: 17982499
references.citation: Am J Med. 1980 Aug;69(2):204-17
references.pmid: 6996481
references.citation: Br J Haematol. 1997 Jun;97(4):855-64
references.pmid: 9217189
references.citation: Cancer. 2006 Apr 15;106(8):1794-803
references.pmid: 16532500
references.citation: Blood. 2003 Feb 1;101(3):822-6
references.pmid: 12393424
references.citation: Br J Haematol. 1993 Nov;85(3):627-30
references.pmid: 8136289
references.citation: Cancer Genet Cytogenet. 2010 Dec;203(2):180-6
references.pmid: 21156231
references.citation: Blood. 2000 Feb 15;95(4):1188-94
references.pmid: 10666189
references.citation: Br J Haematol. 2010 Apr;149(1):14-21
references.pmid: 20136826
references.citation: Blood. 2003 May 15;101(10):3872-4
references.pmid: 12511406
references.citation: Blood. 2012 Sep 20;120(12):2454-65
references.pmid: 22740453
references.citation: Bone Marrow Transplant. 1997 Dec;20(12):1109-10
references.pmid: 9466288
references.citation: Biol Blood Marrow Transplant. 2011 Sep;17(9):1282-8
references.pmid: 21220033
references.citation: Pediatr Blood Cancer. 2006 May 1;46(5):630-6
references.pmid: 16078221
references.citation: Blood. 2003 Feb 15;101(4):1249-56
references.pmid: 12393516
references.citation: Bone Marrow Transplant. 1994 Jul;14(1):151-3
references.pmid: 7951104
references.citation: Haematologica. 2010 Aug;95(8):1236-40
references.pmid: 20675743
references.citation: Genes Dev. 2005 Dec 15;19(24):2925-40
references.pmid: 16357213
references.citation: Br J Haematol. 1998 Oct;103(1):249-55
references.pmid: 9792317
references.citation: Bone Marrow Transplant. 2005 Mar;35(5):463-6
references.pmid: 15654354
references.citation: Am J Clin Pathol. 2010 Jan;133(1):92-100
references.pmid: 20023263
references.citation: Blood. 2007 Mar 1;109(5):2256-62
references.pmid: 17038525
references.citation: Cancer Genet Cytogenet. 1991 Jan;51(1):1-12
references.pmid: 1984836
references.citation: Blood. 2004 Jul 15;104(2):579-85
references.pmid: 15039286
references.citation: Biol Blood Marrow Transplant. 2008 Jul;14(7):748-58
references.pmid: 18541193
references.citation: Cancer Genet Cytogenet. 2000 Mar;117(2):125-31
references.pmid: 10704682
references.citation: Bone Marrow Transplant. 2005 Feb;35(4):341-3
references.pmid: 15640819
references.citation: Br J Haematol. 2007 Feb;136(4):633-40
references.pmid: 17367413
references.citation: Haematologica. 2007 Oct;92(10):1381-8
references.pmid: 18024375
references.citation: Blood. 1997 Mar 15;89(6):2079-88
references.pmid: 9058730
references.citation: Blood. 1994 Sep 1;84(5):1650-5
references.pmid: 8068955