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 journalReview article

1 Scopus citations


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 languageEnglish (US)
Pages (from-to)4002-4020
Number of pages19
JournalBlood Advances
Issue number23
StatePublished - Jan 1 2019


ASJC Scopus subject areas

  • Hematology

Cite this

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