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학술연구/단체지원/교육 등 연구자 활동을 지속하도록 DBpia가 지원하고 있어요.
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연구자들이 자신의 연구와 전문성을 널리 알리고, 새로운 협력의 기회를 만들 수 있는 네트워킹 공간이에요.
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- 자료유형
- 학술저널
- 저자정보
- 발행연도
- 2020.1
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Background: This study aimed to assess the outcome of stem cell transplantation (SCT), including overall survival (OS), failure-free survival (FFS) and graft-versus-host disease (GvHD)-free/failure-free survival (GFFS), and to analyze prognostic factors in children with aplastic anemia (AA). Methods: From 1991 to 2018, 43 allogeneic SCT recipients were enrolled in the study to investigate the demographic characteristics, survival outcomes and prognostic factors. Results: With the median follow-up of 7.1 years, the estimated 10-year OS, FFS, GFFS were 86.0%, 60.5%, and 51.2%, respectively. Matched related donors (MRD, n = 28) showed better 10-year OS than unrelated donors (n = 15) (96.4% vs. 66.7%; P = 0.006). Engraftment failure was seen in 13 patients (30.2%). Donor-type aplasia was seen in 13.8% (4/29) after fludarabine (Flu)-based conditioning (Flu-group), while in 42.6% (6/14) after cyclophosphamide (Cy)-based regimen (Cy-group) (P = 0.035). Six patients died. The 10- year OS in Cy-group was 92.9% (n = 14, all MRD), while that of Flu-group was 82.1% (n = 29; P = 0.367). But Flu-group tended to have better FFS and GFFS than Cy-group, although Flu-group had less MRDs (41.4% vs. 100%; P = 0.019), and higher proportion of previous immunosuppressive treatment (IST; 62% vs. 21.4%, P = 0.012). In MRD transplants, OS was similar between Flu-group (100%, n = 14) and Cy-group (92.9%, n = 14), while FFS (100.0% vs. 42.9%; P = 0.001) and GFFS (85.7% vs. 35.7%; P = 0.006) were significantly better in Flu- group. Stem cell sources, irradiation in the conditioning, and method of GvHD prophylaxis did not significantly influence the outcome. Conclusion: This study reviewed SCT outcomes for pediatric AA with changes of transplant strategies over the last 25 years. The FFS and GFFS were higher in Flu-group than in Cy- group, especially in matched related transplantation. Graft failure including donor-type aplasia remains troublesome even with Flu-based conditioning. Further refinement of transplant strategies to ensure better quality-of-life should be pursued.
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참고문헌 (37)
참고문헌 신청
Young NS / 2006 / Current concepts in the pathophysiology and treatment of aplastic anemia / Blood 108(8):2509~2519
Marsh JC / 2012 / Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party / Blood 119(23):5391~5396
Young NS / 2000 / Hematopoietic cell destruction by immune mechanisms in acquired aplastic anemia / Semin Hematol 37(1):3~14
Risitano AM / 2004 / In-vivo dominant immune responses in aplastic anaemia : molecular tracking of putatively pathogenetic T-cell clones by TCR β-CDR3 sequencing / Lancet 364(9431):355~364
Maciejewski JP / 2005 / Aplastic anemia : management of adult patients / Hematology Am Soc Hematol Educ Program 2005(1):110~117
Marsh JC / 2012 / Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party / Blood 119(23):5391~5396
Young NS / 2000 / Hematopoietic cell destruction by immune mechanisms in acquired aplastic anemia / Semin Hematol 37(1):3~14
Risitano AM / 2004 / In-vivo dominant immune responses in aplastic anaemia : molecular tracking of putatively pathogenetic T-cell clones by TCR β-CDR3 sequencing / Lancet 364(9431):355~364
Maciejewski JP / 2005 / Aplastic anemia : management of adult patients / Hematology Am Soc Hematol Educ Program 2005(1):110~117
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