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. 2019 Dec 10;3(23):3982-4001.
doi: 10.1182/bloodadvances.2019000882.

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

Ann T Farrell  1 , Julie Panepinto  2 , C Patrick Carroll  3 , Deepika S Darbari  4 , Ankit A Desai  5 , Allison A King  6 , Robert J Adams  7 , Tabitha D Barber  8 , Amanda M Brandow  2 , Michael R DeBaun  9 , Manus J Donahue  10   11   12 , Kalpna Gupta  13 , Jane S Hankins  14 , Michelle Kameka  15 , Fenella J Kirkham  16   17 , Harvey Luksenburg  18 , Shirley Miller  19 , Patricia Ann Oneal  1 , David C Rees  20   21 , Rosanna Setse  1 , Vivien A Sheehan  22 , John Strouse  23   24 , Cheryl L Stucky  25 , Ellen M Werner  18 , John C Wood  26 , William T Zempsky  27
Affiliations

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

Ann T Farrell et al. Blood Adv. .

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 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.

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Conflict of interest statement

Conflict-of-interest disclosure: For full conflict-of-interest information, please see the supplemental disclosure file.

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