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

Ann T. Farrell, Julie Panepinto, C. Patrick Carroll, Deepika S. Darbari, Ankit A. Desai, Allison A. King, Robert J. Adams, Tabitha D. Barber, Amanda M. Brandow, Michael R. DeBaun, Manus J. Donahue, Kalpna Gupta, Jane S. Hankins, Michelle Kameka, Fenella J. Kirkham, Harvey Luksenburg, Shirley Miller, Patricia Ann Oneal, David C. Rees, Rosanna SetseVivien A. Sheehan, John Strouse, Cheryl L. Stucky, Ellen M. Werner, John C. Wood, William T. Zempsky

Research output: Contribution to journalReview articlepeer-review

42 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)3982-4001
Number of pages20
JournalBlood Advances
Issue number23
StatePublished - 2019

ASJC Scopus subject areas

  • Hematology


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