Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas, USA.
Division of Critical Care, Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, Texas, USA.
Pediatr Blood Cancer. 2024 Aug;71(8):e31061. doi: 10.1002/pbc.31061. Epub 2024 Jun 5.
Chronic hemolytic anemia and vascular occlusion are hallmarks of sickle cell disease (SCD). Blood transfusions are critical for supportive and preventive management of SCD complications. Patients with SCD are at risk for hyperhemolysis syndrome (HHS), a subtype of delayed hemolytic transfusion reactions. HHS management includes intravenous immunoglobulin, corticosteroids, and avoidance of further transfusions. Not all patients respond to first-line agents. Eculizumab, which blocks terminal complement activation, has been proposed as second-line management of HHS. We describe two patients who received eculizumab for refractory HHS. In our experience, eculizumab is a safe and effective option for refractory pediatric HHS.
慢性溶血性贫血和血管闭塞是镰状细胞病（SCD）的特征。输血对于 SCD 并发症的支持和预防管理至关重要。SCD 患者有发生高血红蛋白血症综合征（HHS）的风险，这是迟发性溶血性输血反应的一个亚型。HHS 的治疗包括静脉注射免疫球蛋白、皮质类固醇和避免进一步输血。并非所有患者对一线药物都有反应。依库珠单抗可阻断末端补体激活，被提议作为 HHS 的二线治疗。我们描述了两名接受依库珠单抗治疗难治性 HHS 的患者。根据我们的经验，依库珠单抗是治疗难治性儿童 HHS 的一种安全有效的选择。
