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

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

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.

2473-9529
3982-4001
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.
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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.
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Farrell, Ann T., Panepinto, Julie, Carroll, C. Patrick, Darbari, Deepika S., Desai, Ankit A., King, Allison A., Adams, Robert J., Barber, Tabitha D., Brandow, Amanda M., DeBaun, Michael R., Donahue, Manus J., Gupta, Kalpna, Hankins, Jane S., Kameka, Michelle, Kirkham, Fenella J., Luksenburg, Harvey, Miller, Shirley, Oneal, Patricia Ann, Rees, David C., Setse, Rosanna, Sheehan, Vivien A., Strouse, John, Stucky, Cheryl L., Werner, Ellen M., Wood, John C. and Zempsky, William T. (2019) End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Blood Advances, 3 (23), 3982-4001. (doi:10.1182/bloodadvances.2019000882).

Record type: Review

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.

Full text not available from this repository.

More information

Accepted/In Press date: 26 September 2019
e-pub ahead of print date: 6 December 2019
Published date: 10 December 2019

Identifiers

Local EPrints ID: 440737
URI: http://eprints.soton.ac.uk/id/eprint/440737
ISSN: 2473-9529
PURE UUID: 84cf62c6-37e2-4f13-9c85-a4f04250a260
ORCID for Fenella J. Kirkham: ORCID iD orcid.org/0000-0002-2443-7958

Catalogue record

Date deposited: 14 May 2020 16:46
Last modified: 07 Oct 2020 01:46

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Contributors

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

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