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. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

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/m2; body fat = 42% ± 8%). Paired t tests examined differences between predicted and measured RMR; significance defined as P < 0.05. Bias and agreement were displayed using Bland-Altman plots. Accuracy was defined when the predicted RMR was ± 10% of the measured RMR. Data were analyzed with SPSS (version 19). Only the equation of Owen et al was not significantly different from the measured RMR (1336 ± 142 and 1322 ± 203 kcal/d, respectively). The equation of Owen et al was accurate for 84.2% of women; RMR prediction equations had limited applicability for young Hispanic women.

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. METHOD: A Best Self-management Practices workshop was held in conjunction with the Sickle Cell Disease Association of America meeting. A panel discussion was organized; adult clients were recruited for participation. The workshop was divided into 3 topics: complementary and alternative medicine, psychosocial issues, and work/education/training. Panel discussions were audiotaped, transcribed, and analyzed using the constant comparative method. RESULTS: Seven adult patients with sickle cell disease and 1 social worker participated. The following themes emerged: self-awareness, emotional support, career selection and success factors, nutrition, advocacy, knowledge, physical, and complementary and alternative medicine. Self-awareness was the most reported strategy with emphasis on journaling and body awareness. Emotional support included spiritual support, friends, family, professional counseling, and spiritual support. A variety of suggestions were discussed related to the other themes. All participants used many strategies daily to maintain optimal health.