End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings

Ann T. Farrell; Julie Panepinto; Ankit A. Desai; Adetola A. Kassim; Jeffrey Lebensburger; Mark C. Walters; Daniel E. Bauer; Rae M. Blaylark; Donna M. DiMichele; Mark T. Gladwin; Nancy S. Green; Kathryn Hassell; Gregory J. Kato; Elizabeth S. Klings; Donald B. Kohn; Lakshmanan Krishnamurti; Jane Little; Julie Makani; Punam Malik; Patrick T. McGann; Caterina Minniti; Claudia R. Morris; Isaac Odame; Patricia Ann Oneal; Rosanna Setse; Poornima Sharma; Shalini Shenoy

doi:10.1182/bloodadvances.2019000883

End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings

Ann T. Farrell, Julie Panepinto, Ankit A. Desai, Adetola A. Kassim, Jeffrey Lebensburger, Mark C. Walters, Daniel E. Bauer, Rae M. Blaylark, Donna M. DiMichele, Mark T. Gladwin, Nancy S. Green, Kathryn Hassell, Gregory J. Kato, Elizabeth S. Klings, Donald B. Kohn, Lakshmanan Krishnamurti, Jane Little, Julie Makani, Punam Malik, Patrick T. McGannCaterina Minniti, Claudia R. Morris, Isaac Odame, Patricia Ann Oneal, Rosanna Setse, Poornima Sharma, Shalini Shenoy

Medicine

Research output: Contribution to journal › Review article › peer-review

21 Scopus citations

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.

Original language	English (US)
Pages (from-to)	4002-4020
Number of pages	19
Journal	Blood Advances
Volume	3
Issue number	23
DOIs	https://doi.org/10.1182/bloodadvances.2019000883
State	Published - 2019

ASJC Scopus subject areas

Hematology

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10.1182/bloodadvances.2019000883

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Farrell, A. T., Panepinto, J., Desai, A. A., Kassim, A. A., Lebensburger, J., Walters, M. C., Bauer, D. E., Blaylark, R. M., DiMichele, D. M., Gladwin, M. T., Green, N. S., Hassell, K., Kato, G. J., Klings, E. S., Kohn, D. B., Krishnamurti, L., Little, J., Makani, J., Malik, P., ... Shenoy, S. (2019). End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings. Blood Advances, 3(23), 4002-4020. https://doi.org/10.1182/bloodadvances.2019000883

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 & Shenoy, S 2019, 'End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings', Blood Advances, vol. 3, no. 23, pp. 4002-4020. https://doi.org/10.1182/bloodadvances.2019000883

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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.",

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AU - Kassim, Adetola A.

AU - Lebensburger, Jeffrey

AU - Walters, Mark C.

AU - Bauer, Daniel E.

AU - Blaylark, Rae M.

AU - DiMichele, Donna M.

AU - Gladwin, Mark T.

AU - Green, Nancy S.

AU - Hassell, Kathryn

AU - Kato, Gregory J.

AU - Klings, Elizabeth S.

AU - Kohn, Donald B.

AU - Krishnamurti, Lakshmanan

AU - Little, Jane

AU - Makani, Julie

AU - Malik, Punam

AU - McGann, Patrick T.

AU - Minniti, Caterina

AU - Morris, Claudia R.

AU - Odame, Isaac

AU - Oneal, Patricia Ann

AU - Setse, Rosanna

AU - Sharma, Poornima

AU - Shenoy, Shalini

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AB - 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.

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End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings

Abstract

ASJC Scopus subject areas

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