Risk-adjusted monitoring of veno-occlusive disease following Bayesian individualization of busulfan dosage for bone marrow transplantation in paediatrics
Risk-adjusted monitoring of veno-occlusive disease following Bayesian individualization of busulfan dosage for bone marrow transplantation in paediatrics
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DOI：
10.1002/pds.1504
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发表时间：
2008-02-01
影响因子：
2.6
通讯作者：
Nathalie, Bleyzac
中科院分区：
文献类型：
--
作者：
Brice, Kitio;Valerie, Bertholle;Nathalie, Bleyzac
为了评估贝叶斯个体化白消安（BU）给药方案的性能，监测了接受异基因骨髓移植（BMT）的儿科患者的静脉闭塞病（VOD）发生率。回顾性分析2000年1月至2006年2月间连续5年接受BU预处理方案的异基因骨髓移植患者。VOD是一个主要的结果变量。采用评分系统评估每位患者的VOD预处理风险，评分系统包括移植类型、受体CMV阳性状态和移植前提供的全肠外营养（TPN）。采用风险调整的累积和方法，通过基于对数似然比为每例患者分配风险评分，比较观察结果与预测结果。将这些累积评分与“信号传导”的预设控制限连续作图，其中结果与预期显著不同（比值比加倍或减半）。口服白消安预处理方案后接受骨髓移植66例，中位年龄3.9岁，男性占63.6%。VOD的中位预处理风险为0.34，范围为0.23至0.84。观察到的VOD率为16.7%（n = 11），比风险评分估计的预期数量少60.7%（17）。按顺序绘制每例患者的风险调整评分。该图早期采用负斜率，在27例和66例患者后两次越过控制下限，表明与预期结果相比结果有所改善。口服白消安剂量方案的贝叶斯个体化有助于降低接受异基因BMT的儿童的VOD率。版权所有（C）2007约翰威利父子有限公司
In order to assess the performance of Bayesian individualization of busulfan (BU) dosage regimens, veno-occlusive disease (VOD) rate was monitored for paediatric patients undergoing allogeneic bone marrow transplantation (BMT). Consecutive patients undergoing allogeneic BMT with BU as conditioning regimen during 5-years period (January 2000-February 2006) were reviewed. VOD was a major outcome variable. Preconditioning risk of VOD was estimated for each patient using a scoring system that included type of transplant, recipient CMV-positive status and total parenteral nutrition (TPN) provided pretransplantation. A risk-adjusted cumulative sum method was used to compare observed versus predicted outcome by assigning a risk score, based on log-likelihood ratios, to each patient. These cumulative scores were sequentially plotted with preset control limits for 'signalling' where results were substantially different than expected (doubling or halving of odds ratio). Sixty-six children received BMT after oral busulfan-based conditioning regimen with median age 3.9 years, 63.6% of male. Median preconditioning risk of VOD was 0.34 ranging from 0.23 to 0.84. Observed VOD rate was 16.7% (n = 11) which was 60.7% (17) fewer than the expected number estimated by the risk score. The resulting risk-adjusted score for each patient was plotted sequentially. This plot adopted early a negative slope, crossing the lower control limit twice, after 27 and 66 patients, indicating improved results compared to those expected. Bayesian individualization of oral busulfan dosage regimens is useful to reduce the VOD rate in children undergoing allogeneic BMT. Copyright (C) 2007 John Wiley & Sons, Ltd.