@article{6d070967506b43e3a8b0d5ccd80a994a,
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",
note = "Funding Information: 1US Food and Drug Administration, White Oak, MD; 2Pediatric Hematology, Medical College of Wisconsin/Children{\textquoteright}s Wisconsin, Milwaukee, WI; 3Krannert Institute of Cardiology, Department of Medicine, Indiana University, Indianapolis, IN; 4Division of Hematology/Oncology, Department of Medicine, Vanderbilt University School of Medicine, Nashville, TN; 5Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL; 6UCSF Benioff Children{\textquoteright}s Hospital, Oakland, CA; 7Division of Hematology/Oncology, Boston Children{\textquoteright}s Hospital, Boston, MA; 8Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; 9Department of Pediatrics, Harvard Medical School, Boston, MA; 10Children{\textquoteright}s Hospitals and Clinics of MN, Minneapolis, MN; 11Sickle Cell Foundation of Minnesota, Minneapolis, MN; 12National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD; 13Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA; 14Department of Pediatrics, Columbia University Medical Center, New York, NY; 15Division of Hematology, Department of Medicine, University of Colorado, Aurora, CO; 16Division of Hematology-Oncology and Pittsburgh Heart, Lung, Blood and Vascular Medicine Institute, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA; 17The Pulmonary Center, Boston University School of Medicine, Boston, MA; 18Department of Microbiology, Immunology & Molecular Genetics, 19Department of Pediatrics, and 20Department of Molecular & Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA; 21Aflac Cancer and Blood Disorders Center, Emory University, Children{\textquoteright}s Healthcare of Atlanta, Atlanta, GA; 22Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC; 23Department of Haematology and Blood Transfusion, School of Medicine, Muhimbili University of Health and Allied Sciences, Dar-es-Salaam, Tanzania; 24Cancer and Blood Disease Institute, Cincinnati Children{\textquoteright}s Hospital Medical Center, Cincinnati, OH; 25Department of Pediatrics, University of Cincinnati, Cincinnati, OH; 26Division of Hematology, Montefiore Health System, Albert Einstein College of Medicine, New York, NY; 27Division of Pediatric Emergency Medicine, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA; 28Children{\textquoteright}s Healthcare of Atlanta, Atlanta, GA; 29The Hospital for Sick Children, Toronto, ON, Canada; 30University of Toronto, Toronto, ON, Canada; 31Center for Biologics Evaluation and Research, US Food and Drug Administration, Silver Spring, MD; and 32Division of Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO Funding Information: ASH received support from the Doris Duke Charitable Foundation (DDCF) and the ASH Foundation to cover meeting expenses. Funding Information: NIH, National Institutes of Health; TCD, transcranial Doppler; TIA, transient ischemic attack. Funding Information: This article summarizes topics addressed at the US Food and Drug Administration (FDA)?American Society of Hematology (ASH) Sickle Cell Disease Clinical End Points Workshop. ASH and FDA engaged 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. This work, plus >30 preparatory calls with the panels and engaging discussions at the workshop, contributed to the development of 2 workshop articles that present the findings of the panels. The Contribution section details how the authors were involved in the development of the actual manuscripts. The authors thank Kathi E. Hanna, the contracted science writer who provided summaries based on discussions at workshop and initial summaries submitted by panels, prepared drafts of the manuscript, managed its review, and prepared it for submission. The authors acknowledge Peter Marks (Center for Biologics Evaluation and Research, FDA) and 2018 ASH President Alexis A. Thompson (Ann & Robert H. Lurie Children?s Hospital of Chicago, Feinberg School of Medicine, Northwestern University) for their support and involvement with the 2018 FDA-ASH Sickle Cell Disease Clinical Endpoints Workshop. ASH received support from the Doris Duke Charitable Foundation (DDCF) and the ASH Foundation to cover meeting expenses. The views of the authors represent their own and should not be interpreted to reflect the official policy of the US FDA. Publisher Copyright: {\textcopyright} 2019 American Society of Hematology. All rights reserved.",
year = "2019",
doi = "10.1182/bloodadvances.2019000883",
language = "English (US)",
volume = "3",
pages = "4002--4020",
journal = "Blood Advances",
issn = "2473-9529",
publisher = "American Society of Hematology",
number = "23",
}