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

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

Zempsky, William T

2020-07-01T20:08:14Z

2020-07-01T20:08:14Z

2019-12

2020-07-01T20:08:13Z

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.

429244

2473-9529

2473-9537

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

eng

American Society of Hematology

Blood advances

10.1182/bloodadvances.2019000882

Science & Technology

Life Sciences & Biomedicine

Hematology

QUALITY-OF-LIFE

ACUTE CHEST SYNDROME

SILENT CEREBRAL INFARCTS

NEUROPATHIC PAIN

VASOOCCLUSIVE CRISIS

SELF-EFFICACY

HEALTH-CARE

BACK-PAIN

FUNCTIONAL CONNECTIVITY

PSYCHOMETRIC PROPERTIES

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

Journal article

Strouse, John|0000-0003-0341-1457

3982

4001

23

School of Medicine

Pediatrics, Hematology-Oncology

Medicine, Hematology

Duke

Pediatrics

Clinical Science Departments

Medicine

Published

3