Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Nashville, Tennesse.
Vanderbilt-Meharry Sickle Cell Disease Center of Excellence, Nashville, Tennesse.
Am J Hematol. 2018 Sep;93(9):1153-1160. doi: 10.1002/ajh.25202. Epub 2018 Jul 30.
Chronic end-organ complications result in morbidity and mortality in adults with sickle cell disease (SCD). In a retrospective-prospective cohort of 150 adults with SCD who received standard care screening for pulmonary function abnormalities, cardiac disease, and renal assessment from January 2003 to 2016, we tested the hypothesis that clustering of end-organ disease is common and multiple organ impairment predicts mortality. Any end-organ disease occurred in 59.3% of individuals, and 24.0% developed multiple organ (>1) end-organ disease. The number of end-organs affected was associated with mortality (P ≤ .001); 8.2% (5 of 61) of individuals with no affected end-organ, 9.4% (5 of 53) of those with 1 affected organ, 20.7% (6 of 29) of those with 2 affected end-organs, and 85.7% (6 of 7) with 3 affected end-organs died over a median follow up period of 8.7 (interquartile range 3.5-11.4) years. Of the 22 individuals who died, 77.3% had evidence of any SCD-related end-organ impairment, and this was the primary or secondary cause of death in 45.0%. SCD-related chronic impairment in multiple organs, and its association with mortality, highlights the need to understand the common mechanisms underlying chronic end-organ damage in SCD, and the urgent need to develop interventions to prevent irreversible end-organ complications in SCD.
慢性终末器官并发症导致镰状细胞病 (SCD) 成人的发病率和死亡率。在一项回顾性前瞻性队列研究中，对 2003 年 1 月至 2016 年期间接受标准护理筛查的 150 例 SCD 成人的肺功能异常、心脏疾病和肾脏评估，我们检验了以下假设：终末器官疾病的聚类是常见的，多个器官损伤预测死亡率。任何终末器官疾病的发生率为 59.3%，24.0%的患者发生多种器官（>1）终末器官疾病。受影响的终末器官数量与死亡率相关（P≤0.001）；8.2%（5/61）无受影响的终末器官，9.4%（5/53）有 1 个受影响的器官，20.7%（6/29）有 2 个受影响的终末器官，85.7%（6/7）有 3 个受影响的终末器官，中位随访时间为 8.7 年（四分位距 3.5-11.4 年）。在 22 例死亡患者中，77.3%的患者有任何 SCD 相关的终末器官损伤证据，其中 45.0%的死亡是 SCD 相关的终末器官损伤的主要或次要原因。SCD 相关的多器官慢性损伤及其与死亡率的关联，突出了需要了解 SCD 慢性终末器官损伤的常见机制，并迫切需要开发干预措施来预防 SCD 中不可逆的终末器官并发症。
