Pediatrics, Referral Center for Sickle Cell Disease, Centre Hospitalier Intercommunal of Créteil, 40 avenue de Verdun, Créteil, France.
Blood. 2011 Jan 27;117(4):1130-40; quiz 1436. doi: 10.1182/blood-2010-06-293514. Epub 2010 Nov 10.
Transcranial Doppler (TCD) is used to detect children with sickle cell anemia (SCA) who are at risk for stroke, and transfusion programs significantly reduce stroke risk in patients with abnormal TCD. We describe the predictive factors and outcomes of cerebral vasculopathy in the Créteil newborn SCA cohort (n = 217 SS/Sβ(0)), who were early and yearly screened with TCD since 1992. Magnetic resonance imaging/magnetic resonance angiography was performed every 2 years after age 5 (or earlier in case of abnormal TCD). A transfusion program was recommended to patients with abnormal TCD and/or stenoses, hydroxyurea to symptomatic patients in absence of macrovasculopathy, and stem cell transplantation to those with human leukocyte antigen-genoidentical donor. Mean follow-up was 7.7 years (1609 patient-years). The cumulative risks by age 18 years were 1.9% (95% confidence interval [95% CI] 0.6%-5.9%) for overt stroke, 29.6% (95% CI 22.8%-38%) for abnormal TCD, which reached a plateau at age 9, whereas they were 22.6% (95% CI 15.0%-33.2%) for stenosis and 37.1% (95% CI 26.3%-50.7%) for silent stroke by age 14. Cumulating all events (stroke, abnormal TCD, stenoses, silent strokes), the cerebral risk by age 14 was 49.9% (95% CI 40.5%-59.3%); the independent predictive factors for cerebral risk were baseline reticulocytes count (hazard ratio 1.003/L × 10(9)/L increase, 95% CI 1.000-1.006; P = .04) and lactate dehydrogenase level (hazard ratio 2.78/1 IU/mL increase, 95% CI1.33-5.81; P = .007). Thus, early TCD screening and intensification therapy allowed the reduction of stroke-risk by age 18 from the previously reported 11% to 1.9%. In contrast, the 50% cumulative cerebral risk suggests the need for more preventive intervention.
经颅多普勒 (TCD) 用于检测镰状细胞贫血 (SCA) 患儿中存在卒中风险的患者，而输血方案可显著降低 TCD 异常患者的卒中风险。我们描述了在 1992 年开始对 217 名 SS/Sβ(0)型克里特尔新生儿 SCA 队列进行早期和每年 TCD 筛查的脑血管病变的预测因素和结局。在年龄超过 5 岁后（或 TCD 异常时更早），每 2 年进行磁共振成像/磁共振血管造影检查。对 TCD 异常和/或狭窄的患者推荐输血方案，对无症状但存在大血管病变的患者推荐使用羟基脲，对人类白细胞抗原基因型相同供体的患者推荐进行干细胞移植。中位随访时间为 7.7 年（1609 患者年）。18 岁时的累积风险分别为显性卒中 1.9%（95%置信区间 [95%CI]0.6%-5.9%），TCD 异常 29.6%（95%CI 22.8%-38%），9 岁时达到平台期，而狭窄的 22.6%（95%CI 15.0%-33.2%）和无症状卒中的 37.1%（95%CI 26.3%-50.7%）则在 14 岁时达到。将所有事件（卒中、TCD 异常、狭窄、无症状卒中）累积起来，14 岁时的脑风险为 49.9%（95%CI 40.5%-59.3%）；脑风险的独立预测因素为基线网织红细胞计数（每增加 1.003/L×10(9)/L，危险比为 1.000-1.006；P =.04）和乳酸脱氢酶水平（每增加 1IU/mL，危险比为 2.78/1IU/mL，95%CI1.33-5.81；P =.007）。因此，早期 TCD 筛查和强化治疗可将 18 岁时的卒中风险从之前报告的 11%降至 1.9%。相比之下，50%的累积脑风险表明需要更多的预防干预。
