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

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

22 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
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

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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

@article{70fa6922c1b0462a9fc7ef6ea0fbe850,

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

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.",

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

year = "2019",

doi = "10.1182/bloodadvances.2019000883",

language = "English",

volume = "3",

pages = "4002--4020",

journal = "Blood Advances",

issn = "2473-9529",

number = "23",

}

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

TY - JOUR

T1 - End points for sickle cell disease clinical trials

T2 - Renal and cardiopulmonary, cure, and low-resource settings

AU - Farrell, Ann T.

AU - Panepinto, Julie

AU - Desai, Ankit A.

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

PY - 2019

Y1 - 2019

N2 - 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.

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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U2 - 10.1182/bloodadvances.2019000883

DO - 10.1182/bloodadvances.2019000883

M3 - Review article

C2 - 31809537

AN - SCOPUS:85076345059

SN - 2473-9529

VL - 3

SP - 4002

EP - 4020

JO - Blood Advances

JF - Blood Advances

IS - 23

ER -

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

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