1. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
- Author
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Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, and Shenoy S
- Subjects
- Heart Diseases pathology, Humans, Lung Diseases pathology, Renal Insufficiency, Chronic pathology, Anemia, Sickle Cell physiopathology, Heart Diseases etiology, Lung Diseases etiology, Renal Insufficiency, Chronic etiology
- Abstract
To address the global burden of sickle cell disease 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 outcome, pain (non-patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.
- Published
- 2019
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