| Literature DB >> 31809537 |
Ann T Farrell1, Julie Panepinto2, Ankit A Desai3, Adetola A Kassim4, Jeffrey Lebensburger5, Mark C Walters6, Daniel E Bauer7,8,9, Rae M Blaylark10,11, Donna M DiMichele12, Mark T Gladwin13, Nancy S Green14, Kathryn Hassell15, Gregory J Kato16, Elizabeth S Klings17, Donald B Kohn18,19,20, Lakshmanan Krishnamurti21, Jane Little22, Julie Makani23, Punam Malik24,25, Patrick T McGann24,25, Caterina Minniti26, Claudia R Morris27,28, Isaac Odame29,30, Patricia Ann Oneal1, Rosanna Setse1, Poornima Sharma31, Shalini Shenoy32.
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.Entities:
Year: 2019 PMID： 31809537 PMCID： PMC6963248 DOI： 10.1182/bloodadvances.2019000883
Source DB: PubMed Journal: Blood Adv ISSN： 2473-9529