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

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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Citation

Published Version (Please cite this version)

10.1182/bloodadvances.2019000882

Publication Info

Farrell, Ann T, Julie Panepinto, C Patrick Carroll, Deepika S Darbari, Ankit A Desai, Allison A King, Robert J Adams, Tabitha D Barber, et al. (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 Retrieved from https://hdl.handle.net/10161/21140.

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Scholars@Duke

Strouse

John J. Strouse

Associate Professor of Medicine

My research has focused on the epidemiology, risk factors, and prevention of the pulmonary and central nervous system complications of sickle cell disease and includes retrospective and prospective cohort studies and clinical trials.  I received my Ph.D. in clinical investigation from the Johns Hopkins Bloomberg School of Public Health for a series of studies to identify predictors of cognitive function in children with sickle cell disease.  This work has expanded to the evaluation of the interaction between environment and disease in both children and adults and the functional evaluation of adults with sickle cell disease.  My other research interests include the application of large clinical, research, and administrative databases to the study of rare hematological diseases and interventions to improve quality of and access to care for sickle cell disease. I serve on the American Society of Hematology Sickle Cell Taskforce and Sickle Cell Pain Guideline Panel and am co-chair of the American Society of Hematology Healthcare Professional Education and Training Work Group.

 


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