• Elizabeth S Klings, Roberto F Machado, Robyn J Barst, Claudia R Morris, Kamal K Mubarak, Victor R Gordeuk, Gregory J Kato, Kenneth I Ataga, J Simon Gibbs, Oswaldo Castro, Erika B Rosenzweig, Namita Sood, Lewis Hsu, Kevin C Wilson, Marilyn J Telen, Laura M Decastro, Lakshmanan Krishnamurti, Martin H Steinberg, David B Badesch, Mark T Gladwin, and American Thoracic Society Ad Hoc Committee on Pulmonary Hypertension of Sickle Cell Disease.
• Am. J. Respir. Crit. Care Med. 2014 Mar 15; 189 (6): 727-40.

BackgroundIn adults with sickle cell disease (SCD), an increased tricuspid regurgitant velocity (TRV) measured by Doppler echocardiography, an increased serum N-terminal pro-brain natriuretic peptide (NT-pro-BNP) level, and pulmonary hypertension (PH) diagnosed by right heart catheterization (RHC) are independent risk factors for mortality.MethodsA multidisciplinary committee was formed by clinician-investigators experienced in the management of patients with PH and/or SCD. Clinically important questions were posed, related evidence was appraised, and questions were answered with evidence-based recommendations. Target audiences include all clinicians who take care of patients with SCD.ResultsMortality risk stratification guides decision making. An increased risk for mortality is defined as a TRV equal to or greater than 2.5 m/second, an NT-pro-BNP level equal to or greater than 160 pg/ml, or RHC-confirmed PH. For patients identified as having increased mortality risk, we make a strong recommendation for hydroxyurea as first-line therapy and a weak recommendation for chronic transfusions as an alternative therapy. For all patients with SCD with elevated TRV alone or elevated NT-pro-BNP alone, and for patients with SCD with RHC-confirmed PH with elevated pulmonary artery wedge pressure and low pulmonary vascular resistance, we make a strong recommendation against PAH-specific therapy. However, for select patients with SCD with RHC-confirmed PH who have elevated pulmonary vascular resistance and normal pulmonary capillary wedge pressure, we make a weak recommendation for either prostacyclin agonist or endothelin receptor antagonist therapy and a strong recommendation against phosphodiesterase-5 inhibitor therapy.ConclusionsEvidence-based recommendations for the management of patients with SCD with increased mortality risk are provided, but will require frequent reassessment and updating.

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