Identifying Clinical and Research Priorities in Sickle Cell Lung Disease An Official American Thoracic Society Workshop Report

• Authors: A. Parker Ruhl, National Institute of Allergy and Infectious Diseases; S. Christy Sadreameli, Johns Hopkins School of Medicine; Julian L. Allen, Childrens Hospital of Philadelphia; Debra P. Bennett, Independent Patient Advocate; Andrew D. Campbell, Childrens National Medical Center; Thomas D. Coates, Childrens Hospital Los Angeles; Dapa A. Diallo, University of Sciences, Techniques and Technologies of Bamako (USTTB); Joshua J. Field, Medical College of Wisconsin; Elizabeth K. Fiorino, Cohen Children's Medical Center; Claudia Morris, Emory University
• Journal Title: Annals of the American Thoracic Society
• Volume: Volume 16, Number 9
• Publisher: American Thoracic Society | 2019-09-01, Pages E17-E32
• Type of Work: Article | Final Publisher PDF
• Publisher DOI: http://doi.org/10.1513/AnnalsATS.201906-433ST
• Subjects: Health Sciences, Medicine and Surgery
• Keywords: Science & Technology; Life Sciences & Biomedicine; Respiratory System; sickle cell disease; acute chest syndrome; asthma; sleep disorders; pulmonary hypertension; ACUTE CHEST SYNDROME; OBSTRUCTIVE SLEEP-APNEA; PLACENTA GROWTH-FACTOR; TRICUSPID REGURGITATION VELOCITY; ACUTE PULMONARY COMPLICATIONS; ENVIRONMENTAL TOBACCO-SMOKE; FORCED EXPIRATORY VOLUME; CAPILLARY BLOOD-VOLUME; RISK-FACTORS; AIRWAY HYPERRESPONSIVENESS

Abstract

Background: 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. Methods: 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: 1) acute chest syndrome, 2) lower airways disease and pulmonary function, 3) sleep-disordered breathing and hypoxia, and 4) 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. Results: 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. Conclusions: 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.