Ann Am Thorac Soc. 2019 Sep;16(9):e17-e32. doi: 10.1513/AnnalsATS.201906-433ST.
Pulmonary complications of sickle cell disease (SCD) are diverse and encompass acute and chronic disease. The understanding of the natural history of pulmonary complications of SCD is limited, no specific therapies exist, and these complications are a primary cause of morbidity and mortality. We gathered a multidisciplinary group of pediatric and adult hematologists, pulmonologists, and emergency medicine physicians with expertise in SCD-related lung disease along with an SCD patient advocate for an American Thoracic Society-sponsored workshop to review the literature and identify key unanswered clinical and research questions. Participants were divided into four subcommittees on the basis of expertise: ) acute chest syndrome, ) lower airways disease and pulmonary function, ) sleep-disordered breathing and hypoxia, and ) pulmonary vascular complications of SCD. Before the workshop, a comprehensive literature review of each subtopic was conducted. Clinically important questions were developed after literature review and were finalized by group discussion and consensus. Current knowledge is based on small, predominantly observational studies, few multicenter longitudinal studies, and even fewer high-quality interventional trials specifically targeting the pulmonary complications of SCD. Each subcommittee identified the three or four most important unanswered questions in their topic area for researchers to direct the next steps of clinical investigation. Important and clinically relevant questions regarding sickle cell lung disease remain unanswered. High-quality, multicenter, longitudinal studies and randomized clinical trials designed and implemented by teams of multidisciplinary clinician-investigators are needed to improve the care of individuals with SCD.
镰状细胞病（SCD）的肺部并发症多种多样，包括急性和慢性疾病。对 SCD 肺部并发症自然史的了解有限，没有特定的治疗方法，这些并发症是发病率和死亡率的主要原因。我们召集了一组具有小儿科和成人血液病学家、肺病学家和急诊医学专家专长的多学科团队，以及一位 SCD 患者倡导者，参加了美国胸科学会赞助的研讨会，以审查文献并确定关键的未解答的临床和研究问题。参与者根据专业知识分为四个小组委员会：）急性胸部综合征，）下呼吸道疾病和肺功能，）睡眠呼吸障碍和缺氧，以及）SCD 的肺血管并发症。在研讨会之前，对每个子主题进行了全面的文献综述。在文献综述后制定了具有临床重要性的问题，并通过小组讨论和共识最终确定。目前的知识基于小型的、主要是观察性研究、少数多中心纵向研究，甚至更少的专门针对 SCD 肺部并发症的高质量干预性试验。每个小组委员会确定了他们主题领域中最重要的三到四个未解答的问题，供研究人员指导下一步的临床研究。有关镰状细胞肺病的重要和临床相关问题仍未得到解答。需要由多学科临床研究人员团队设计和实施高质量、多中心、纵向研究和随机临床试验，以改善 SCD 患者的护理。
