Arlet Jean-Benoît, Herquelot Eléonore, Lamarsalle Ludovic, Raguideau Fanny, Bartolucci Pablo
Reference Center for Sickle Cell Disease, Thalassemia and Other Red Blood Cell and Erythropoiesis Diseases, Department of Internal Medicine, Paris-Cité University and European Georges Pompidou University Hospital (AP-HP), 75015, Paris, France.
Heva, Lyon, France.
Lancet Reg Health Eur. 2024 Apr 6;40:100901. doi: 10.1016/j.lanepe.2024.100901. eCollection 2024 May.
Historically, sickle cell disease (SCD) patients experiencing frequent hospitalized vaso-occlusive crises (HVOC) have been associated with increased mortality, yet recent data reflecting the widespread use of hydroxyurea and advancements in disease management remain limited. Our study aims to assess the association between HVOC and mortality or severe complications in patients with SCD in this new treatment landscape.
This was a retrospective observational cohort study using the French national health data system. Between 01-01-2012 and 12-31-2018, all SCD patients ≥16 years old (ICD-10 codes D57.0-2) were included and followed until 12-31-2018. HVOC was defined as a hospitalization of ≥1 night with primary diagnosis of SCD with crisis, following an emergency room visit. The association between HVOC and severe complications was assessed with a Cox proportional hazards model.
In total, 8018 patients (56.6% females; 4538/8018) were included. The 2018 SCD standardized one-year period prevalence was 17.9 cases/100,000 person-years [17.4; 18.3]. The mean rate was 0.84 (1.88) HVOC/person-year. In 2018, 70% (5323/7605), 22% (1671/7605), and 8% (611/7605) of patients experienced 0, 1-2, or 3+ HVOCs, respectively. The median survival time between HVOCs was 415 days [386; 439]. Overall, 312 patients died (3.9%) with a mean age of 49.8 (19.4). Compared to patients without HVOC, the hazard ratios of death in patients with 1-2 or 3+ HVOCs the year prior to death were 1.67 [1.21; 2.30] and 3.70 [2.30; 5.93], respectively. Incidence of acute chest syndrome, pulmonary embolism, osteonecrosis, and sepsis increased with the HVOCs category, but not stroke. In 2018, 29.5% (180/611) of patients with 3+ HVOCs did not take hydroxyurea.
Patients must be closely monitored during their hospitalizations to intensify treatment and check treatment compliance. Innovative therapies are also required.
The study was funded by Novartis.
从历史上看，患有频繁住院血管闭塞性危机（HVOC）的镰状细胞病（SCD）患者死亡率有所增加，但反映羟基脲广泛使用和疾病管理进展的最新数据仍然有限。我们的研究旨在评估在这种新的治疗环境下，HVOC与SCD患者死亡率或严重并发症之间的关联。
这是一项使用法国国家卫生数据系统的回顾性观察队列研究。在2012年1月1日至2018年12月31日期间，纳入所有年龄≥16岁的SCD患者（国际疾病分类第十版代码D57.0 - 2），并随访至2018年12月31日。HVOC定义为在急诊就诊后，因SCD危机而进行的至少住院1晚的主要诊断。使用Cox比例风险模型评估HVOC与严重并发症之间的关联。
总共纳入了8018例患者（女性占56.6%；4538/8018）。2018年SCD标准化一年期患病率为17.9例/100,000人年[17.4；18.3]。平均发生率为0.84（1.88）次HVOC/人年。在2018年，分别有70%（5323/7605）、22%（1671/7605）和8%（611/7605）的患者经历了0次、1 - 2次或3次以上的HVOC。两次HVOC之间的中位生存时间为415天[386；439]。总体而言，312例患者死亡（3.9%），平均年龄为49.8（19.4）岁。与无HVOC的患者相比，在死亡前一年有1 - 2次或3次以上HVOC的患者的死亡风险比分别为1.67[1.21；2.30]和3.70[2.30；5.93]。急性胸部综合征、肺栓塞、骨坏死和败血症的发生率随HVOC类别增加，但中风并非如此。在2018年，3次以上HVOC的患者中有29.5%（180/611）未服用羟基脲。
在患者住院期间必须密切监测，以加强治疗并检查治疗依从性。还需要创新疗法。
该研究由诺华公司资助。
