Neonatal screening improves sickle cell disease clinical outcome in Belgium
Neonatal screening improves sickle cell disease clinical outcome in Belgium
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DOI：
10.1177/0969141317701166
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发表时间：
2018-06-01
影响因子：
2.9
通讯作者：
Gulbis, Beatrice
中科院分区：
文献类型：
--
作者：
Phu-Quoc Le;Ferster, Alina;Gulbis, Beatrice
目的：比较镰状细胞病患者的诊断结果，通过新生儿筛查与那些谁wasn 't.Methods：在比利时的一项观察性多中心研究，167筛选和93未筛选镰状细胞病患者进行了分析，共1116和958患者年的后续行动，分别。两组患者均采用倾向评分分析进行比较，患者在三个协变量（性别、基因型和中非血统）上匹配。结果：筛选组无菌血症生存率的Kaplan-Meier估计值显著高于未筛选组（94.47%; [95%CI，88.64-97.36%] vs 83.78% [95%CI，72.27-90.42%]），p = 0.032。在无急性胸部综合征、急性贫血、脑并发症、严重感染和血管闭塞危象的情况下，两组的生存率无显著差异。与未筛查组相比，筛查组的住院率和每100患者年的住院天数显著降低（0.27 vs. 0.63和1.25 vs. 2.82，p = 0.0006和
Objectives: To compare the outcomes of sickle cell disease patients diagnosed through neonatal screening with those who were not.Methods: In an observational multicenter study in Belgium, 167 screened and 93 unscreened sickle cell disease patients were analyzed for a total of 1116 and 958 patient-years of follow-up, respectively. Both groups were compared with propensity score analysis, with patients matched on three covariates (gender, genotype, and central Africa origin). Bonferroni correction was applied for all comparisons.Results: Kaplan-Meier estimates of survival without bacteremia were significantly higher in the screened group than the unscreened group (94.47%; [95% CI, 88.64-97.36%] versus 83.78% [95% CI, 72.27-90.42%]), p = 0.032. Non-significant differences between both groups were reported for survival without acute chest syndrome, acute anemia, cerebral complication, severe infection, and vaso-occlusive crisis. Significantly lower hospitalization rate and days per 100 patient-years were observed in the screened compared with the unscreened group (0.27 vs. 0.63 and 1.25 vs. 2.82, p = 0.0006 and