We report graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) (a composite end point of survival without grade III-IV acute GVHD [aGVHD], systemic therapy–requiring chronic GVHD [cGVHD], or relapse) and cGVHD-free relapse-free survival (CRFS) among pediatric patients with acute leukemia (n = 1613) who underwent transplantation with 1 antigen–mismatched (7/8) bone marrow (BM; n = 172) or umbilical cord blood (UCB; n = 1441). Multivariate analysis was performed using Cox proportional hazards models. To account for multiple testing, P < .01 for the donor/graft variable was considered statistically significant. Clinical characteristics were similar between UCB and 7/8 BM recipients, because most had acute lymphoblastic leukemia (62%), 64% received total body irradiation–based conditioning, and 60% received anti-thymocyte globulin or alemtuzumab. Methotrexate-based GVHD prophylaxis was more common with 7/8 BM (79%) than with UCB (15%), in which mycophenolate mofetil was commonly used. The univariate estimates of GRFS and CRFS were 22% (95% confidence interval [CI], 16-29) and 27% (95% CI, 20-34), respectively, with 7/8 BM and 33% (95% CI, 31-36) and 38% (95% CI, 35-40), respectively, with UCB (P < .001). In multivariate analysis, 7/8 BM vs UCB had similar GRFS (hazard ratio [HR], 1.12; 95% CI, 0.87-1.45; P = .39), CRFS (HR, 1.06; 95% CI, 0.82-1.38; P = .66), overall survival (HR, 1.07; 95% CI, 0.80-1.44; P = .66), and relapse (HR, 1.44; 95% CI, 1.03-2.02; P = .03). However, the 7/8 BM group had a significantly higher risk for grade III-IV aGVHD (HR, 1.70; 95% CI, 1.16-2.48; P = .006) compared with the UCB group. UCB and 7/8 BM groups had similar outcomes, as measured by GRFS and CRFS. However, given the higher risk for grade III-IV aGVHD, UCB might be preferred for patients lacking matched donors.
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机译：我们报告了无移植物抗宿主病（GVHD）的无复发生存期（GRFS）（无III-IV级急性GVHD [aGVHD]，需要长期GVHD [cGVHD]或需要复发的全身治疗的复合终点） ）和接受1种抗原不匹配（7/8）骨髓（BM; n = 172）或脐带血（ UCB； n = 1441）。使用Cox比例风险模型进行多变量分析。考虑到多次测试，供体/移植物变量的P <0.01被认为具有统计学意义。 UCB和7/8 BM接受者之间的临床特征相似，因为大多数患有急性淋巴细胞白血病（62％），64％接受了基于全身辐射的调理，60％接受了抗胸腺细胞球蛋白或alemtuzumab。基于甲氨蝶呤的GVHD预防在7/8 BM（79％）上比在通常使用霉酚酸酯的UCB（15％）上更常见。 GRFS和CRFS的单变量估计分别为22％（95％置信区间[CI]，16-29）和27％（95％CI，20-34），其中7/8 BM和33％（95％CI） （31-36）和38％（95％CI，35-40），使用UCB（P <.001）。在多变量分析中，7/8 BM vs UCB具有相似的GRFS（危险比[HR]，1.12； 95％CI，0.87-1.45； P = 0.39），CRFS（HR，1.06； 95％CI，0.82-1.38； P = .66），总生存期（HR，1.07; 95％CI，0.80-1.44; P = .66）和复发（HR，1.44; 95％CI，1.03-2.02; P = .03）。然而，与UCB组相比，7/8 BM组患III-IV级aGVHD的风险显着更高（HR为1.70； 95％CI为1.16-2.48； P = 0.006）。根据GRFS和CRFS的测量，UCB和7/8 BM组的结果相似。但是，鉴于III-IV级aGVHD的风险较高，对于缺乏匹配供体的患者，UCB可能是首选。
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