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

Research output: Contribution to journalReview article


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

External Institution(s)

  • United States Food and Drug Administration
  • Medical College of Wisconsin
  • Johns Hopkins University
  • Children's National Medical Center
  • Washington University St. Louis
  • Patient Advocate
  • Vanderbilt University
  • University of Minnesota Twin Cities
  • St. Jude Children Research Hospital
  • Florida International University
  • Great Ormond Street Hospital for Children NHS Trust
  • National Institutes of Health
  • Atrium Health
  • King's College Hospital
  • King's College London
  • Baylor College of Medicine
  • Duke University
  • University of Southern California
  • University of Connecticut


Original languageEnglish (US)
Pages (from-to)3982-4001
Number of pages20
JournalBlood advances
Issue number23
StatusPublished - Jan 1 2019


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.

Citation formats


Farrell, A. T., Panepinto, J., Carroll, C. P., Darbari, D. S., Desai, A. A., King, A. A., ... Zempsky, W. T. (2019). End points for sickle cell disease clinical trials: Patient-reported outcomes, pain, and the brain. Blood advances, 3(23), 3982-4001. https://doi.org/10.1182/bloodadvances.2019000882


Farrell, AT, Panepinto, J, Carroll, CP, Darbari, DS, Desai, AA, King, AA, Adams, RJ, Barber, TD, Brandow, AM, DeBaun, MR, Donahue, MJ, Gupta, K, Hankins, JS, Kameka, M, Kirkham, FJ, Luksenburg, H, Miller, S, Oneal, PA, Rees, DC, Setse, R, Sheehan, VA, Strouse, J, Stucky, CL, Werner, EM, Wood, JC & Zempsky, WT 2019, 'End points for sickle cell disease clinical trials: Patient-reported outcomes, pain, and the brain', Blood advances, vol. 3, no. 23, pp. 3982-4001. https://doi.org/10.1182/bloodadvances.2019000882