Tehseen Sarah, Joiner Clinton H, Lane Peter A, Yee Marianne E
Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.
Department of Pediatrics, Division of Pediatric Hematology/Oncology, Emory University, Atlanta, Georgia.
Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26665. Epub 2017 Jun 14.
Renal damage is a progressive complication of sickle cell disease (SCD) that begins in childhood and may progress to renal failure and early mortality in 12% of adults with hemoglobin SS (HbSS) SCD. Early sickle nephropathy is characterized by hyperfiltration and microalbuminuria; therefore, urine albumin to creatinine ratio (ACR) is an effective screening tool for its detection.
This study investigated the effect of hydroxyurea (HU) therapy on urine ACR levels among children with SCD. A retrospective review was conducted to identify all patients with HbSS or HbSβ thalassemia of age 7-18 years who began HU therapy in 2011-2013; a control group of patients not on HU were matched by age and baseline hemoglobin. All urine ACR measurements ≤24 months prior to and ≥24 months after HU initiation were recorded.
There were 63 eligible patients on HU and 13 (25%) with albuminuria prior to HU initiation. Among those with baseline albuminuria, the median ACR was 96 mg/g prior to HU, 39 mg/g at 1 year (P = 0.02), and 25 mg/g at 2 years (P = 0.03). Albuminuria normalized in 37.5% (6/16) after 1 year and 61% (8/13) after 2 years of HU therapy. Among those without albuminuria prior to HU, 13% (6/47) developed albuminuria during HU therapy. Sixteen percent (13/80) of control patients had albuminuria in the beginning of study period, which normalized in 15% (two of 13) of patients at 1-year follow up.
Introduction of HU is associated with significant decreases in urine ACR in children with SCD and albuminuria.
肾损伤是镰状细胞病（SCD）的一种进行性并发症，始于儿童期，在12%的血红蛋白SS（HbSS）型SCD成人患者中可能进展为肾衰竭并导致早期死亡。早期镰状肾病的特征是超滤和微量白蛋白尿；因此，尿白蛋白肌酐比值（ACR）是检测该病的有效筛查工具。
本研究调查了羟基脲（HU）治疗对SCD儿童尿ACR水平的影响。进行了一项回顾性研究，以确定2011年至2013年开始接受HU治疗的所有7至18岁的HbSS或HbSβ地中海贫血患者；未接受HU治疗的对照组患者按年龄和基线血红蛋白进行匹配。记录了HU开始前≤24个月和开始后≥24个月的所有尿ACR测量值。
有63例符合条件的患者接受HU治疗，其中13例（25%）在开始HU治疗前存在蛋白尿。在基线蛋白尿患者中，HU治疗前ACR中位数为96mg/g，1年时为39mg/g（P = 0.02），2年时为25mg/g（P = 0.03）。HU治疗1年后，37.5%（6/16）的患者蛋白尿恢复正常，2年后为61%（8/13）。在HU治疗前无蛋白尿的患者中，13%（6/47）在HU治疗期间出现蛋白尿。16%（13/80）的对照患者在研究开始时存在蛋白尿，在1年随访时，15%（13例中的2例）患者蛋白尿恢复正常。
对于患有SCD和蛋白尿的儿童，引入HU与尿ACR显著降低有关。
