End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain-Reference-Cited by-同舟云学术

End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

Published:2019-12-06 Issue:23 Volume:3 Page:3982-4001
ISSN:2473-9529
Container-title:Blood Advances
language:en
Short-container-title:

Author:

Farrell Ann T.¹,Panepinto Julie²,Carroll C. Patrick³,Darbari Deepika S.⁴,Desai Ankit A.⁵,King Allison A.⁶,Adams Robert J.⁷,Barber Tabitha D.⁸,Brandow Amanda M.²,DeBaun Michael R.⁹,Donahue Manus J.¹⁰¹¹¹²,Gupta Kalpna¹³,Hankins Jane S.¹⁴,Kameka Michelle¹⁵,Kirkham Fenella J.¹⁶¹⁷,Luksenburg Harvey¹⁸,Miller Shirley¹⁹,Oneal Patricia Ann¹,Rees David C.²⁰²¹,Setse Rosanna¹,Sheehan Vivien A.²²,Strouse John²³²⁴,Stucky Cheryl L.²⁵,Werner Ellen M.¹⁸,Wood John C.²⁶,Zempsky William T.²⁷

Affiliation:

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

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

3. Department of Psychiatry and Behavioral Sciences, Johns Hopkins School of Medicine, Baltimore, MD;

4. Children’s National Medical Center, Washington, DC;

5. Krannert Institute of Cardiology, Indiana University, Bloomington, IN;

6. Division of Hematology and Oncology in Pediatrics and Medicine, Washington University School of Medicine, St. Louis, MO;

7. Department of Neurology, Medical University of South Carolina, Charleston, SC;

8. Patient Advocate, Rochester, NY;

9. Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, TN;

10. Department of Radiology and Radiological Sciences,

11. Department of Neurology, and

12. Department of Psychiatry, School of Medicine, Vanderbilt University, Nashville, TN;

13. Division of Hematology, Oncology, and Transplantation, Department of Medicine, Medical School, University of Minnesota, Minneapolis, MN;

14. Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN;

15. Nicole Wertheim College of Nursing and Health Sciences, Florida International University, Miami, FL;

16. Developmental Neurosciences Unit and

17. Biomedical Research Unit, UCL Great Ormond Street Institute of Child Health, London, United Kingdom;

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

19. Atrium Healthcare, Charlotte, NC;

20. Department of Haematological Medicine, King's College Hospital, London, United Kingdom;

21. School of Cancer and Pharmaceutical Sciences, King’s College London, London, United Kingdom;

22. Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX;

23. Division of Hematology, Department of Medicine, and

24. Division of Pediatric Hematology/Oncology, Department of Pediatrics, Duke University School of Medicine, Durham, NC;

25. Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI;

26. Children’s Hospital Los Angeles, Los Angeles, CA; and

27. Department of Pediatrics, Connecticut Children’s/School of Medicine, University of Connecticut, Hartford, CT

Abstract

AbstractTo address the global burden of sickle cell disease (SCD) 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 outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

Publisher

American Society of Hematology

Subject

Hematology

Link

http://ashpublications.org/bloodadvances/article-pdf/3/23/3982/1716994/advancesadv2019000882.pdf

Reference205 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: renal and cardiopulmonary, cure, and low-resource settings;Farrell;Blood Adv,2019

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

4. U.S. Food and Drug Administration. Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. Available at: www.fda.gov/downloads/drugs/guidances/ucm193282.pdf. Accessed 31 August 2018.

5. The voice of the patient: sickle cell disease;U.S. Food and Drug Administration. Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research.

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