Lê Phu-Quoc, Ferster Alina, Dedeken Laurence, Vermylen Christiane, Vanderfaeillie Anna, Rozen Laurence, Heijmans Catherine, Huybrechts Sophie, Devalck Christine, Cotton Frédéric, Ketelslegers Olivier, Dresse Marie-Françoise, Fils Jean-François, Gulbis Béatrice
1 Hemato-Oncology, Hopital Universitaire des Enfants Reine Fabiola, Brussels, Belgium.
2 Hemato-Oncology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
J Med Screen. 2018 Jun;25(2):57-63. doi: 10.1177/0969141317701166. Epub 2017 Jul 7.
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 <0.0001). Conclusion These data confirm the benefit of a neonatal screening programme in reducing bacteremia and hospitalization.
目的 比较通过新生儿筛查诊断出的镰状细胞病患者与未通过筛查的患者的治疗结果。方法 在比利时进行的一项多中心观察性研究中,分别对167例经筛查和93例未经筛查的镰状细胞病患者进行了分析,随访时间分别为1116和958患者年。两组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)上进行匹配。所有比较均采用Bonferroni校正。结果 经筛查组无菌血症生存率的Kaplan-Meier估计值显著高于未筛查组(94.47%;[95%CI,88.64 - 97.36%] 对 83.78% [95%CI,72.27 - 90.42%]),p = 0.032。两组在无急性胸综合征、急性贫血、脑部并发症、严重感染和血管闭塞性危机的生存率方面报告无显著差异。与未筛查组相比,经筛查组的住院率和每100患者年的住院天数显著更低(0.27对0.63以及1.25对2.82,p = 0.0006和<0.0001)。结论 这些数据证实了新生儿筛查计划在降低菌血症和住院率方面的益处。