End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings

Academic Article


  • To address the global burden of sickle cell disease 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 outcome, pain (non–patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.
  • Published In

  • Blood Advances  Journal
  • Digital Object Identifier (doi)

    Author List

  • Farrell AT; Panepinto J; Desai AA; Kassim AA; Lebensburger J; Walters MC; Bauer DE; Blaylark RM; DiMichele DM; Gladwin MT
  • Start Page

  • 4002
  • End Page

  • 4020
  • Volume

  • 3
  • Issue

  • 23