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

Author:

Farrell Ann T.1,Panepinto Julie2,Desai Ankit A.3,Kassim Adetola A.4,Lebensburger Jeffrey5,Walters Mark C.6,Bauer Daniel E.789,Blaylark Rae M.1011,DiMichele Donna M.12,Gladwin Mark T.13,Green Nancy S.14,Hassell Kathryn15,Kato Gregory J.16,Klings Elizabeth S.17,Kohn Donald B.181920,Krishnamurti Lakshmanan21,Little Jane22,Makani Julie23,Malik Punam2425,McGann Patrick T.2425,Minniti Caterina26,Morris Claudia R.2728,Odame Isaac2930,Oneal Patricia Ann1,Setse Rosanna1,Sharma Poornima31,Shenoy Shalini32

Affiliation:

1. US Food and Drug Administration, White Oak, MD;

2. Pediatric Hematology, Medical College of Wisconsin/Children's Wisconsin, Milwaukee, WI;

3. Krannert Institute of Cardiology, Department of Medicine, Indiana University, Indianapolis, IN;

4. Division of Hematology/Oncology, Department of Medicine, Vanderbilt University School of Medicine, Nashville, TN;

5. Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL;

6. UCSF Benioff Children’s Hospital, Oakland, CA;

7. Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA;

8. Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA;

9. Department of Pediatrics, Harvard Medical School, Boston, MA;

10. Children’s Hospitals and Clinics of MN, Minneapolis, MN;

11. Sickle Cell Foundation of Minnesota, Minneapolis, MN;

12. National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;

13. Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA;

14. Department of Pediatrics, Columbia University Medical Center, New York, NY;

15. Division of Hematology, Department of Medicine, University of Colorado, Aurora, CO;

16. Division of Hematology-Oncology and Pittsburgh Heart, Lung, Blood and Vascular Medicine Institute, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA;

17. The Pulmonary Center, Boston University School of Medicine, Boston, MA;

18. Department of Microbiology, Immunology & Molecular Genetics,

19. Department of Pediatrics, and

20. Department of Molecular & Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA;

21. Aflac Cancer and Blood Disorders Center, Emory University, Children's Healthcare of Atlanta, Atlanta, GA;

22. Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC;

23. Department of Haematology and Blood Transfusion, School of Medicine, Muhimbili University of Health and Allied Sciences, Dar-es-Salaam, Tanzania;

24. Cancer and Blood Disease Institute, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;

25. Department of Pediatrics, University of Cincinnati, Cincinnati, OH;

26. Division of Hematology, Montefiore Health System, Albert Einstein College of Medicine, New York, NY;

27. Division of Pediatric Emergency Medicine, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA;

28. Children’s Healthcare of Atlanta, Atlanta, GA;

29. The Hospital for Sick Children, Toronto, ON, Canada;

30. University of Toronto, Toronto, ON, Canada;

31. Center for Biologics Evaluation and Research, US Food and Drug Administration, Silver Spring, MD; and

32. Division of Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO

Abstract

AbstractTo 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.

Publisher

American Society of Hematology

Subject

Hematology

Reference169 articles.

1. Population estimates of sickle cell disease in the U.S;Hassell;Am J Prev Med,2010

2. End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain;Farrell;Blood Adv,2019

3. FDA-NIH Biomarker Working Group . BEST (Biomarkers, EndpointS, and other Tools) resource. Available at: https://www.ncbi.nlm.nih.gov/books/NBK326791/. Accessed 25 September 2019.

4. Causes of death in sickle cell disease: an autopsy study;Manci;Br J Haematol,2003

5. Causes of death and early life determinants of survival in homozygous sickle cell disease: the Jamaican cohort study from birth;Serjeant;PLoS One,2018

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3