Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched familial donor.
We compared the outcomes of immunosuppressive treatment (IST) with those of alternative donor hematopoietic stem cell transplantation (HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 42 patients with SAA who received frontline IST (N=19) or frontline HSCT with an alternative donor (N=23) between 1998 and 2012 were analyzed retrospectively. Six patients responded in the frontline IST group, whereas 11 underwent salvage HSCT after IST failure. Twenty-one of 23 patients who underwent frontline HSCT survived without treatment failure. The estimated failure-free survival rate of the frontline HSCT group was higher than that of the frontline IST group (91.3% vs 30.7% respectively, P<0.001). Six of 11 patients who underwent salvage HSCT experienced event-free survival (EFS). The estimated EFS of the frontline HSCT group was higher than that of the salvage HSCT group (91.3% vs 50.9% respectively, P=0.015). The outcome of alternative donor HSCT was better than commonly reported rates, especially in patients who underwent frontline HSCT. These results suggest that frontline alternative donor HSCT may be a better treatment option than IST for children and adolescents with SAA who lack a human leukocyte Ag-matched familial donor.
Department of Pediatrics, Chung-Ang University Hospital, Seoul, Korea.
Y B
YB
Choi
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
E S
ES
Yi
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
J W
JW
Lee
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
K W
KW
Sung
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
H H
HH
Koo
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.|Department of Medical Device Management and Research, SAIHST, Sungkyunkwan University, Seoul, Korea.
K H
KH
Yoo
D000293:Adolescent; D000367:Age Factors; D064591:Allografts; D000741:Anemia, Aplastic / Q000401:mortality* / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006680:HLA Antigens; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007165:Immunosuppression Therapy; D007223:Infant; D008297:Male; D015996:Survival Rate; D061349:Unrelated Donors
D006680:HLA Antigens
J Pediatr. 2008 Dec;153(6):814-9
18672253
Blood. 2002 Aug 1;100(3):799-803
12130489
Br J Haematol. 2015 May;169(4):565-73
25683884
Bone Marrow Transplant. 2005 Dec;36(11):947-50
16205733
Bone Marrow Transplant. 2015 May;50(5):685-9
25730184
Biol Blood Marrow Transplant. 2013 Mar;19(3):338-43
23228588
Semin Hematol. 2010 Jan;47(1):37-45
20109610
Bone Marrow Transplant. 2012 Sep;47(9):1186-90
22246086
Haematologica. 2011 Jan;96(1):134-41
21071499
Biol Blood Marrow Transplant. 1999;5(4):243-52
10465104
Pediatr Blood Cancer. 2008 Jan;50(1):52-7
17941069
Haematologica. 2014 Dec;99(12):1784-91
25193958
Br J Haematol. 2012 May;157(3):339-46
22372373
Br J Haematol. 2003 Dec;123(5):782-801
14632769
Br J Haematol. 2000 Oct;111(1):321-8
11091219
Blood. 2013 Aug 8;122(6):1072-8
23692854
Haematologica. 2007 Jan;92(1):11-8
17229630
Bone Marrow Transplant. 2012 Dec;47(12):1507-12
22635243
Blood. 2002 Aug 1;100(3):786-90
12130487
Bone Marrow Transplant. 2008 Nov;42(9):589-95
18695669
Blood. 2008 Feb 1;111(3):1054-9
17989314
Br J Haematol. 2009 Oct;147(1):43-70
19673883
N Engl J Med. 2011 Aug 4;365(5):430-8
21812672
Haematologica. 2010 Jun;95(6):976-82
20494932
Blood. 2012 Jun 7;119(23):5391-6
22544699
Biol Blood Marrow Transplant. 2008 Sep;14(9):1057-63
18721769
Haematologica. 2011 Jun;96(6):814-9
21422115
Br J Haematol. 1996 Jul;94(1):65-72
8757510