DeBaun M R, Jordan L C, King A A, Schatz J, Vichinsky E, Fox C K, McKinstry R C, Telfer P, Kraut M A, Daraz L, Kirkham F J, Murad M H
Department of Pediatrics, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, and.
Division of Pediatric Neurology, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN.
Blood Adv. 2020 Apr 28;4(8):1554-1588. doi: 10.1182/bloodadvances.2019001142.
Central nervous system (CNS) complications are among the most common, devastating sequelae of sickle cell disease (SCD) occurring throughout the lifespan.
These evidence-based guidelines of the American Society of Hematology are intended to support the SCD community in decisions about prevention, diagnosis, and treatment of the most common neurological morbidities in SCD.
The Mayo Evidence-Based Practice Research Program supported the guideline development process, including updating or performing systematic evidence reviews. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE evidence-to-decision frameworks, to assess evidence and make recommendations.
The panel placed a higher value on maintaining cognitive function than on being alive with significantly less than baseline cognitive function. The panel developed 19 recommendations with evidence-based strategies to prevent, diagnose, and treat CNS complications of SCD in low-middle- and high-income settings.
Three of 19 recommendations immediately impact clinical care. These recommendations include: use of transcranial Doppler ultrasound screening and hydroxyurea for primary stroke prevention in children with hemoglobin SS (HbSS) and hemoglobin Sβ0 (HbSβ0) thalassemia living in low-middle-income settings; surveillance for developmental delay, cognitive impairments, and neurodevelopmental disorders in children; and use of magnetic resonance imaging of the brain without sedation to detect silent cerebral infarcts at least once in early-school-age children and once in adults with HbSS or HbSβ0 thalassemia. Individuals with SCD, their family members, and clinicians should become aware of and implement these recommendations to reduce the burden of CNS complications in children and adults with SCD.
中枢神经系统（CNS）并发症是镰状细胞病（SCD）最常见、最具破坏性的后遗症之一，可发生于患者一生的各个阶段。
美国血液学会的这些循证指南旨在为SCD群体在预防、诊断和治疗SCD最常见神经疾病方面的决策提供支持。
梅奥循证实践研究项目为指南制定过程提供支持，包括更新或进行系统的证据审查。专家小组采用推荐分级评估、制定和评价（GRADE）方法，包括GRADE证据到决策框架，来评估证据并提出建议。
专家小组更重视维持认知功能，而非在认知功能显著低于基线水平的情况下存活。专家小组制定了19条建议，以及基于证据的策略，以预防、诊断和治疗低收入、中等收入和高收入环境中SCD的CNS并发症。
19条建议中有3条对临床护理有直接影响。这些建议包括：对生活在低收入和中等收入环境中的血红蛋白SS（HbSS）和血红蛋白Sβ0（HbSβ0）地中海贫血患儿，使用经颅多普勒超声筛查和羟基脲进行原发性卒中预防；对儿童进行发育迟缓、认知障碍和神经发育障碍的监测；对HbSS或HbSβ0地中海贫血的学龄儿童早期和成人至少进行一次无镇静的脑部磁共振成像检查，以检测无症状性脑梗死。SCD患者及其家庭成员以及临床医生应了解并实施这些建议，以减轻SCD儿童和成人的CNS并发症负担。
