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

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.

Tricuspid regurgitant jet velocity and myocardial tissue Doppler parameters predict mortality in a cohort of patients with sickle cell disease spanning from pediatric to adult age groups ‐ revisiting this controversial concept after 16 years of additional evidence

“…The role of echocardiography and TRV as a predictive marker of cardiovascular mortality in the pediatric, adolescent and young adult SCD population has recently been reported in a large cohort 22 . The American Thoracic Society recently cited long term studies of TRV and risk for cardiovascular morbidity and mortality in the adolescent and young adult population as a critical unmet area of research 23 …”

Perioperative Events of Laparoscopic Cholecystectomy in Patients with Hemoglobinopathies

“…19 Acute chest syndrome is a common cause of postoperative death in sickle cell disease patients characterized by fever, dyspnea, cough and chest pain with new lung infiltration on chest radiograph after exclusion of atelectasis. 20 Hemolysis is a well-known complication of sickle cell disease. Three cases (4.8%) presented with hemolysis post operatively: pallor, tea colored urine and reduction of hemoglobin level below 9 g/dl.…”

Management of chronic respiratory complications in children and adolescents with sickle cell disease

“…The clinical and prognostic meaning of lung function alterations in children with SCD is uncertain, due to the paucity of long-term longitudinal data and the use of various interpretative strategies across different studies [4]. Abnormal lung function patterns are not associated with past or future rates of pain [12], whereas there is conflicting data regarding the relationship with future acute chest syndrome episodes [7,12].…”

“…Respiratory disease is among the leading causes of morbidity and mortality in SCD, with death most commonly occurring in the context of severe acute chest syndrome, pulmonary embolism and/or pulmonary hypertension [3]. There is increasing focus on respiratory care of patients with SCD, and the number of studies investigating respiratory complications has markedly increased in recent years [4].…”