Blood Advances (Dec 2019)
End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain
- Ann T. Farrell,
- Julie Panepinto,
- C. Patrick Carroll,
- Deepika S. Darbari,
- Ankit A. Desai,
- Allison A. King,
- Robert J. Adams,
- Tabitha D. Barber,
- Amanda M. Brandow,
- Michael R. DeBaun,
- Manus J. Donahue,
- Kalpna Gupta,
- Jane S. Hankins,
- Michelle Kameka,
- Fenella J. Kirkham,
- Harvey Luksenburg,
- Shirley Miller,
- Patricia Ann Oneal,
- David C. Rees,
- Rosanna Setse,
- Vivien A. Sheehan,
- John Strouse,
- Cheryl L. Stucky,
- Ellen M. Werner,
- John C. Wood,
- William T. Zempsky
Affiliations
- Ann T. Farrell
- US Food and Drug Administration, White Oak, MD;
- Julie Panepinto
- Pediatric Hematology, Medical College of Wisconsin/Children's Wisconsin, Milwaukee, WI;; Julie Panepinto, Medical College of Wisconsin, 8701 Watertown Plank Rd, MS 756, Milwaukee, WI 53226;
- C. Patrick Carroll
- Department of Psychiatry and Behavioral Sciences, Johns Hopkins School of Medicine, Baltimore, MD;
- Deepika S. Darbari
- Children's National Medical Center, Washington, DC;
- Ankit A. Desai
- Krannert Institute of Cardiology, Indiana University, Bloomington, IN;
- Allison A. King
- Division of Hematology and Oncology in Pediatrics and Medicine, Washington University School of Medicine, St. Louis, MO;
- Robert J. Adams
- Department of Neurology, Medical University of South Carolina, Charleston, SC;
- Tabitha D. Barber
- Patient Advocate, Rochester, NY;
- Amanda M. Brandow
- Pediatric Hematology, Medical College of Wisconsin/Children's Wisconsin, Milwaukee, WI;
- Michael R. DeBaun
- Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, Nashville, TN;
- Manus J. Donahue
- Department of Radiology and Radiological Sciences, School of Medicine, Vanderbilt University, Nashville, TN;; Department of Neurology, School of Medicine, Vanderbilt University, Nashville, TN;; Department of Psychiatry, School of Medicine, Vanderbilt University, Nashville, TN;
- Kalpna Gupta
- Division of Hematology, Oncology, and Transplantation, Department of Medicine, Medical School, University of Minnesota, Minneapolis, MN;
- Jane S. Hankins
- Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN;
- Michelle Kameka
- Nicole Wertheim College of Nursing and Health Sciences, Florida International University, Miami, FL;
- Fenella J. Kirkham
- Developmental Neurosciences Unit, UCL Great Ormond Street Institute of Child Health, London, United Kingdom;; Biomedical Research Unit, UCL Great Ormond Street Institute of Child Health, London, United Kingdom;
- Harvey Luksenburg
- National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;
- Shirley Miller
- Atrium Healthcare, Charlotte, NC;
- Patricia Ann Oneal
- US Food and Drug Administration, White Oak, MD;
- David C. Rees
- Department of Haematological Medicine, King's College Hospital, London, United Kingdom;; School of Cancer and Pharmaceutical Sciences, King's College London, London, United Kingdom;
- Rosanna Setse
- US Food and Drug Administration, White Oak, MD;
- Vivien A. Sheehan
- Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX;
- John Strouse
- Division of Hematology, Department of Medicine, Duke University School of Medicine, Durham, NC;; Division of Pediatric Hematology/Oncology, Department of Pediatrics, Duke University School of Medicine, Durham, NC;
- Cheryl L. Stucky
- Department of Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI;
- Ellen M. Werner
- National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD;
- John C. Wood
- Children's Hospital Los Angeles, Los Angeles, CA
- William T. Zempsky
- Department of Pediatrics, Connecticut Children's/School of Medicine, University of Connecticut, Hartford, CT
- Journal volume & issue
-
Vol. 3,
no. 23
pp. 3982 – 4001
Abstract
Abstract: To 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.
