Boelens J J, Wynn R F, O'Meara A, Veys P, Bertrand Y, Souillet G, Wraith J E, Fischer A, Cavazzana-Calvo M, Sykora K W, Sedlacek P, Rovelli A, Uiterwaal C S P M, Wulffraat N
Department of Immunology/BMT, Wilhelmina Children's Hospital, Utrecht Medical Center, Utrecht, The Netherlands.
Bone Marrow Transplant. 2007 Aug;40(3):225-33. doi: 10.1038/sj.bmt.1705718. Epub 2007 May 28.
Hurler's syndrome (HS), the most severe form of mucopolysaccharidosis type-I, causes progressive deterioration of the central nervous system and death in childhood. Allogeneic stem cell transplantation (SCT) before the age of 2 years halts disease progression. Graft failure limits the success of SCT. We analyzed data on HS patients transplanted in Europe to identify the risk factors for graft failure. We compared outcomes in 146 HS patients transplanted with various conditioning regimens and grafts. Patients were transplanted between 1994 and 2004 and registered to the European Blood and Marrow Transplantation database. Risk factor analysis was performed using logistic regression. 'Survival' and 'alive and engrafted'-rate after first SCT was 85 and 56%, respectively. In multivariable analysis, T-cell depletion (odds ratio (OR) 0.18; 95% confidence interval (CI) 0.04-0.71; P=0.02) and reduced-intensity conditioning (OR 0.08; 95% CI 0.02-0.39; P=0.002) were the risk factors for graft failure. Busulfan targeting protected against graft failure (OR 5.76; 95% CI 1.20-27.54; P=0.028). No difference was noted between cell sources used (bone marrow, peripheral blood stem cells or cord blood (CB)); however, significantly more patients who received CB transplants had full-donor chimerism (OR 9.31; 95% CI 1.06-82.03; P=0.044). These outcomes may impact the safety/efficacy of SCT for 'inborn-errors of metabolism' at large. CB increased the likelihood of sustained engraftment associated with normal enzyme levels and could therefore be considered as a preferential cell source in SCT for 'inborn errors of metabolism'.
黏多糖贮积症 I 型最严重的形式——胡勒氏综合征(HS),会导致中枢神经系统进行性恶化,并在儿童期死亡。2岁前进行异基因干细胞移植(SCT)可阻止疾病进展。移植物失败限制了SCT的成功率。我们分析了在欧洲接受移植的HS患者的数据,以确定移植物失败的危险因素。我们比较了146例接受不同预处理方案和移植物的HS患者的结局。患者于1994年至2004年间接受移植,并登记到欧洲血液与骨髓移植数据库。使用逻辑回归进行危险因素分析。首次SCT后的“生存率”和“存活并植入率”分别为85%和56%。在多变量分析中,T细胞去除(比值比(OR)0.18;95%置信区间(CI)0.04 - 0.71;P = 0.02)和减低强度预处理(OR 0.08;95% CI 0.02 - 0.39;P = 0.002)是移植物失败的危险因素。白消安靶向治疗可预防移植物失败(OR 5.76;95% CI 1.20 - 27.54;P = 0.028)。所用细胞来源(骨髓、外周血干细胞或脐带血(CB))之间未发现差异;然而,接受CB移植的患者中有更多比例具有完全供体嵌合现象(OR 9.31;95% CI 1.06 - 82.03;P = 0.044)。这些结果可能会对总体上SCT治疗“先天性代谢缺陷”的安全性/有效性产生影响。CB增加了与正常酶水平相关的持续植入的可能性,因此在SCT治疗“先天性代谢缺陷”时可被视为优先选择的细胞来源。
N Engl J Med. 2004-5-6
Biol Blood Marrow Transplant. 2009-5
Pediatr Hematol Oncol. 2014-11
Bone Marrow Transplant. 2008-2
J Inflamm (Lond). 2005-7-26
N Engl J Med. 2005-5-19
Biol Blood Marrow Transplant. 2005-5
Blood. 2005-5-15
Hematology Am Soc Hematol Educ Program. 2004