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

ABSTRACT: 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 iden

SUBMITTER: Farrell AT 

PROVIDER: S-EPMC6963237 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature