End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.

To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel.

Common Prediction Equations Overestimate Measured Resting Metabolic Rate in Young Hispanic Women.

The accuracy of 6 resting metabolic rate (RMR) prediction equations to indirect calorimetry was compared in 38 Hispanic women (age = 30 ± 7 years; body mass index = 28.9 ± 7.2 kg/m; body fat = 42% ± 8%).

A qualitative analysis of best self-management practices: sickle cell disease.

Background: Sickle cell disease (SCD) is associated with serious comorbidities resulting in a shortened lifespan, and many clients suffer from frequent pain episodes. However, others successfully manage their disease in the outpatient setting without the need for frequent health care utilization. The purpose of this project was to describe specific strategies used by adult clients with sickle cell disease to achieve optimal physical health.