Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Am J Hematol. 2011 Apr;86(4):357-61. doi: 10.1002/ajh.21986.
Children with sickle cell anemia (SCA) and a primary overt stroke are at high risk of recurrent (secondary) stroke. Chronic transfusion therapy dramatically reduces but does not eliminate this high risk, and inevitably results in transfusion-related hemosiderosis. We previously reported the use of hydroxyurea/phlebotomy as an alternative to transfusions to reduce the risk of secondary stroke and improve management of iron overload in 35 children with SCA. To report long-term results, we retrospectively reviewed clinical and laboratory data through October 2008. With a median of 5.6 years and total of 219 patient-years of follow-up, 10 of 35 patients (29%) had recurrent stroke after switching to hydroxyurea; seven were previously reported and three new strokes occurred during extended follow-up. The overall secondary stroke event rate was 4.6 per 100 patient-years. Children on hydroxyurea received serial phlebotomy and had lower mean serum ferritin values than children on transfusions (591 ng/mL vs. 3410 ng/mL, P = 0.02). In this cohort, long-term hydroxyurea treatment reduced but did not eliminate the risk of stroke recurrence and, uniquely, allowed phlebotomy to reduce iron overload. Long-term assessments of this therapy should evaluate risk factors for secondary stroke and assessments of hemosiderosis, neurocognitive outcome, and health-related quality of life.
患有镰状细胞贫血症 (SCA) 且初次出现显性中风的儿童存在再次发生（继发性）中风的高风险。慢性输血疗法可显著降低但不能消除这种高风险，且不可避免地导致与输血相关的血色素沉着症。我们之前曾报道过使用羟基脲/放血作为替代输血的方法，以降低二次中风的风险并改善镰状细胞贫血症患儿铁过载的管理。为了报告长期结果，我们通过 2008 年 10 月回顾性地审查了临床和实验室数据。在中位随访 5.6 年和总共 219 患者年中，35 名患者中有 10 名（29%）在改用羟基脲后发生了复发性中风；其中 7 例为之前报道过的，3 例新的中风发生在延长随访期间。总体继发性中风发生率为每 100 患者年 4.6 例。接受羟基脲治疗的儿童进行了连续放血，血清铁蛋白值低于接受输血的儿童（591ng/ml 比 3410ng/ml，P=0.02）。在该队列中，长期羟基脲治疗降低但不能消除中风复发的风险，而且独特的是，允许放血来减少铁过载。对这种治疗方法的长期评估应评估继发性中风的风险因素以及铁过载、神经认知结局和健康相关生活质量的评估。
