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

Farrell, AT; Panepinto, J; Carroll, CP; Darbari, DS; Desai, AA; King, AA; Adams, RJ; ... Zempsky, WT; + view all (2019) End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Blood Advances , 3 (23) pp. 3982-4001. 10.1182/bloodadvances.2019000882. Green open access

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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.

Type: Article
Title: End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain
Open access status: An open access version is available from UCL Discovery
DOI: 10.1182/bloodadvances.2019000882
Publisher version: https://doi.org/10.1182/bloodadvances.2019000882
Language: English
Additional information: This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: biological markers, pain, surrogate endpoints, brain, sickle cell anemia
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10094600
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