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

dc.contributor.author

Farrell, Ann T

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Panepinto, Julie

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Carroll, C Patrick

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Darbari, Deepika S

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Desai, Ankit A

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King, Allison A

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Adams, Robert J

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Barber, Tabitha D

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Brandow, Amanda M

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DeBaun, Michael R

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Donahue, Manus J

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Gupta, Kalpna

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Hankins, Jane S

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Kameka, Michelle

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Kirkham, Fenella J

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Luksenburg, Harvey

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Miller, Shirley

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Oneal, Patricia Ann

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Rees, David C

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Setse, Rosanna

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Sheehan, Vivien A

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Strouse, John

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Stucky, Cheryl L

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Werner, Ellen M

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Wood, John C

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Zempsky, William T

dc.date.accessioned

2020-07-01T20:08:14Z

dc.date.available

2020-07-01T20:08:14Z

dc.date.issued

2019-12

dc.date.updated

2020-07-01T20:08:13Z

dc.description.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.

dc.identifier

429244

dc.identifier.issn

2473-9529

dc.identifier.issn

2473-9537

dc.identifier.uri

https://hdl.handle.net/10161/21140

dc.language

eng

dc.publisher

American Society of Hematology

dc.relation.ispartof

Blood advances

dc.relation.isversionof

10.1182/bloodadvances.2019000882

dc.subject

Science & Technology

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Life Sciences & Biomedicine

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Hematology

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QUALITY-OF-LIFE

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ACUTE CHEST SYNDROME

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SILENT CEREBRAL INFARCTS

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NEUROPATHIC PAIN

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VASOOCCLUSIVE CRISIS

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SELF-EFFICACY

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HEALTH-CARE

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BACK-PAIN

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FUNCTIONAL CONNECTIVITY

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PSYCHOMETRIC PROPERTIES

dc.title

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

dc.type

Journal article

duke.contributor.orcid

Strouse, John|0000-0003-0341-1457

pubs.begin-page

3982

pubs.end-page

4001

pubs.issue

23

pubs.organisational-group

School of Medicine

pubs.organisational-group

Pediatrics, Hematology-Oncology

pubs.organisational-group

Medicine, Hematology

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Duke

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Pediatrics

pubs.organisational-group

Clinical Science Departments

pubs.organisational-group

Medicine

pubs.publication-status

Published

pubs.volume

3

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