Ershler William B, De Castro Laura M, Pakbaz Zahra, Moynahan Aaron, Weycker Derek, Delea Thomas E, Agodoa Irene, Cong Ze
Department of Hematology, Inova Schar Cancer Institute, Fairfax, Virginia.
Department of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania.
Curr Ther Res Clin Exp. 2023 Feb 23;98:100696. doi: 10.1016/j.curtheres.2023.100696. eCollection 2023.
Sickle cell disease (SCD) is an inherited, chronic, multifaceted blood disorder. Patients with SCD develop anemia, which has been associated with end-organ damage (EOD).
This retrospective, observational, repeated-measures study systematically characterizes the relationship between hemoglobin (Hb) level and EOD in adolescent and adult patients with SCD
The study population comprised patients with SCD aged ≥12 years with available Hb data from a US provider-centric health care database. For each patient, each Hb value over time was included as a separate observation. Study outcomes-the onset of any new EOD, including chronic kidney disease, pulmonary hypertension, stroke, and leg ulcer-were ascertained during the 1-year period after each Hb assessment. The association between Hb levels and risk of new EOD was estimated using multivariable generalized estimating equations.
A total of 16,043 unique patients with SCD contributed 44,913 observations. Adjusted odds of any EOD during the 1-year follow-up were significantly lower with higher Hb level. Risk reductions with higher Hb levels for chronic kidney disease, pulmonary hypertension, and leg ulcer were comparable. The risk of new EOD was significantly lower among adolescent and adult patients with higher Hb levels.
In patients with SCD, higher Hb levels are associated with a reduced risk of developing EOD. Therapeutic strategies that result in higher Hb levels may offer clinical and economic value for patients with SCD. (. 2023; 84:XXX-XXX).
镰状细胞病（SCD）是一种遗传性慢性、多方面的血液疾病。SCD患者会出现贫血，这与终末器官损伤（EOD）有关。
这项回顾性、观察性、重复测量研究系统地描述了青少年和成年SCD患者血红蛋白（Hb）水平与EOD之间的关系。
研究人群包括年龄≥12岁、可从以美国医疗服务提供者为中心的医疗保健数据库获取Hb数据的SCD患者。对于每位患者，随时间的每个Hb值都作为一个单独的观察值纳入。研究结局——任何新的EOD的发生，包括慢性肾病、肺动脉高压、中风和腿部溃疡——在每次Hb评估后的1年内确定。使用多变量广义估计方程估计Hb水平与新EOD风险之间的关联。
共有16,043例独特的SCD患者贡献了44,913个观察值。随访1年期间，Hb水平越高，任何EOD的校正比值显著越低。Hb水平较高对慢性肾病、肺动脉高压和腿部溃疡的风险降低作用相当。Hb水平较高的青少年和成年患者发生新EOD的风险显著较低。
在SCD患者中，较高的Hb水平与发生EOD的风险降低相关。导致较高Hb水平的治疗策略可能对SCD患者具有临床和经济价值。（. 2023; 84:XXX - XXX）
