Sickle Cell Disease
Data Standard

Adoption of data standards for clinical research includes pharmaceutical companies, regulatory agencies such as the U.S. Food and Drug Administration, academic and government-based clinical researchers and government agencies such as the National Heart, Lung, and Blood Institute.

Prior to reviewing the Data Standards page, we recommended review of comprehensive list of Core elements and overview of domains/subdomains and related forms and instruments:

Start-Up Resource

Highlights Document

Data Standards

Outcomes and End Points
Caregiver Burden
CRF Module/Guideline CDEs
Caregiver Burden Scale (CGBS) CDE Details
Coping Skills/Pain Coping
CRF Module/Guideline CDEs
Coping Strategies Questionnaire ? Revised for SCD CDE Details
Executive Functioning and Attention
CRF Module/Guideline CDEs
NIH Toolbox Cognitive Battery CDE Details
General Function
CRF Module/Guideline CDEs
Canadian Occupational Performance Measure (COPM) CDE Details
Mortality/Survival
CRF Module/Guideline CDEs
Death Form CDE Details
Negative Affect: Mixed
CRF Module/Guideline CDEs
PROMIS Pediatric Physical Stress Experience CDE Details
Pain Mechanism
CRF Module/Guideline CDEs
Quantitative Sensory Testing (QST) CDE Details
Pain: Mixed
CRF Module/Guideline CDEs
PedsQL Pain and Hurt, SCD modules - Core CDE Details
Patient Reported Outcomes Summary
CRF Module/Guideline CDEs
Patient Reported Outcomes Recommendations Summary CDE Details
Sleep Impact/Sleep Disturbance
CRF Module/Guideline CDEs
Polysomnography CDE Details
Stigma
CRF Module/Guideline CDEs
Measure of Sickle Cell Stigma CDE Details
Working Memory
CRF Module/Guideline CDEs
NIH Toolbox Cognitive Battery CDE Details
Additional Proposed Instruments
Other
CRF Module/Guideline CDEs
Behavior Rating Inventory of Executive Function (BRIEF) - Second Edition (BRIEF-2) CDE Details
Classification of Seizures CDE Details
Conners Continuous Performance Test 3rd Edition (Conners CPT 3) CDE Details
Follow Up Status Form - BMT CTN 1507 CDE Details
Haplo Sickle Cell Enrollment Form: Segment A CDE Details
Hematopoiesis Form - BMTCTN 1507A CDE Details
HLA Form - 1507 CDE Details
Hospital Discharge CDE Details
Infection Form (IFN) CDE Details
Intake Medical History CDE Details
Migraine Disability Assessment Test (MIDAS) CDE Details
Miscellaneous Sickle Cell-related CDEs CDE Details
Neurological Assessment: LOC, PTA, and AOC CDE Details
Neurological Exam CDE Details
Pediatric Migraine Disability Assessment Questionnaire (PedMIDAS) CDE Details
Pediatric Stroke Outcome Measure Short Neuro Exam (PSOM-SNE) - Child Version (Children Aged 2 Year and Older) CDE Details
Physical Exam Form - BMT CTN 1507 CDE Details
Protocol Deviation/Violation Form (PDV) - BMTCTN 15070 CDE Details
Protocol Deviation/Violation Review Form (PDR) - BMTCTN 15070 CDE Details
Questionnaire for Verifying Stroke-Free Status (QVSFS) CDE Details
SCD Events of Special Interest Form (ESI) CDE Details
Selected Debaun CDEs CDE Details
Stroke Specific Quality of Life Scale (SS-QOL) CDE Details
Study Discontinuation/Completion CDE Details
Toxicity Form CDE Details
Transplant Form (TXP) - BMTCTN 15070 CDE Details
Vessel Carotid Ultrasound CDE Details
Vessel Imaging Angiography CDE Details
Wechsler Abbreviated Scale of Intelligence - Second Edition (WASI-II) CDE Details
Withdrawal of Consent Form (WOC) CDE Details
Phen-X
CRF Module/Guideline CDEs
PhenX CDEs CDE Details
PhenX Protocol: Complete Blood Count (CBC) CDE Details
PhenX Toolkit: Alcohol - Lifetime Use CDE Details
PhenX Toolkit: Annual Pain Frequency of Sickle Cell Pain Episodes Per Year CDE Details
PhenX Toolkit: Aspartate Aminotransferase level CDE Details
PhenX Toolkit: Bilirubin level CDE Details
PhenX Toolkit: Birthplace CDE Details
PhenX Toolkit: Birthplace of Grandparents CDE Details
PhenX Toolkit: Birthplace of Parents CDE Details
PhenX Toolkit: Body temperature CDE Details
PhenX Toolkit: Chest X-ray CDE Details
PhenX Toolkit: Child-Reported Parental Education Attainment CDE Details
PhenX Toolkit: Current Age CDE Details
PhenX Toolkit: Current Employment Status CDE Details
PhenX Toolkit: Functionality After Stroke CDE Details
PhenX Toolkit: Gender Data Collection Worksheet CDE Details
PhenX Toolkit: Haptoglobin level CDE Details
PhenX Toolkit: Height - Self-Reported Height CDE Details
PhenX Toolkit: Height - Standing Height CDE Details
PhenX Toolkit: Hemoglobin Characterization CDE Details
PhenX Toolkit: History of Transfusion CDE Details
PhenX Toolkit: Lactate Dehydrogenase level CDE Details
PhenX Toolkit: Lung Function - Diffusion Capacity CDE Details
PhenX Toolkit: Lung Function - Lung Volume CDE Details
PhenX Toolkit: Marital Status CDE Details
PhenX Toolkit: Marital Status of Primary Caregiver CDE Details
PhenX Toolkit: Medication Inventory CDE Details
PhenX Toolkit: N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) level CDE Details
PhenX Toolkit: Neurology, QOL Health Services Recovery and Recurrence Questionnaire - Pediatric CDE Details
PhenX Toolkit: Pediatric School Performance CDE Details
PhenX Toolkit: Pulse Oximetry (Rest) CDE Details
PhenX Toolkit: Quality of Care - Adults CDE Details
PhenX Toolkit: Quality of Care - Children CDE Details
PhenX Toolkit: Quality of Life - Adult CDE Details
PhenX Toolkit: Quality of Life - Pediatric CDE Details
PhenX Toolkit: Quality of Life in Sickle Cell Disease CDE Details
PhenX Toolkit: Reticulocyte Count CDE Details
PhenX Toolkit: Self-efficacy in Sickle Cell Disease CDE Details
PhenX Toolkit: Serum Creatinine CDE Details
PhenX Toolkit: Serum or Plasma Ferritin CDE Details
PhenX Toolkit: Stroke Impact/Outcome - Pediatric CDE Details
PhenX Toolkit: Substances - Lifetime Use CDE Details
PhenX Toolkit: Urinary Creatinine CDE Details
PhenX Toolkit: Urinary Microalbumin CDE Details
PhenX Toolkit: Weight - Measured Weight CDE Details
PhenX Toolkit: Weight - Self-Reported CDE Details
PhenX Toolkit: Years Living in the U.S. CDE Details

History

Working groups were formed to review five clinical research study domains:

  • Genetics/Assays;
  • Physical Examination/Medical History;
  • Cardiopulmonary, Renal and Cerebrovascular;
  • Outcomes; and
  • Monitoring Side Effects.

These working groups were comprised of SCD adult and pediatric specialists, patient advocates, genetic therapy specialists, neuropsychologists, nurses, data managers, manufacturers, industry members, and academics. After a Public Review period and follow-up with working groups, version 1.0 of the standardized data forms have been released. The forms will also be made available through the National Library of Medicine (NLM) website. This will be an iterative process, and feedback from users will be encouraged.

 

Cardiopulmonary, Renal and Cerebrovascular Working Group

 

Michael DeBaun, MD, MPH (Co-Chair)
Vanderbilt University Medical Center, Nashville, Tennessee

Alexis Thompson, MD, MPH (Co-Chair)
Northwestern University, Chicago, Illinois

Anjulika Chawla, MD
bluebird bio, Cambridge, Massachusetts

Robyn Cohen, MD
Boston Medical Center, Boston, Massachusetts

Courtney Fitzhugh, MD
National Heart, Lung and Blood Institute, Bethesda, Maryland

Lisa Garrett, RN, CCRP
Washington University in St. Louis, St. Louis, Missouri

Eileen Hansbury, PA
Boston Children’s Hospital, Boston, Massachusetts

Tabitha Hendershot
Research Triangle Institute, Washington, DC

Kathleen Hewitt, DND, RN, CPNQ
American Society of Hematology Research Collaborative, Gainesville, Virginia

Lori Jordan, MD, PhD
Vanderbilt Unversity Medical Center, Nashville, Tennessee

Gregory Kato, MD
CSL Behring, King of Prussia, Pennsylvania

Chava Kimchi-Sartay, PhD
Food and Drug Administration, Silver Spring, Maryland

Allison King, MD, MPH, PhD
Washington University of St. Louis, St. Louis, Missouri

Elizabeth S. Klings, MD
Boston University School of Medicine, Boston, Massachusetts

Lakshmanan Krishnamurti, MD
Emory University School of Medicine, Atlanta, Georgia

Jeffrey Lebensburger, DO
University of Alabama Birmingham, Birmingham, Alabama

Robert Lindblad, MD
Emmes, Rockville, Maryland

Charity Oyedeji, MD
Patient Advocate

Duke University, Durham, North Carolina

Vandana Sachdev, MD
National Heart, Lung and Blood Institute, Bethesda, Maryland

Santosh Saraf, MD
University of Illinois at Chicago, Chicago, Illinois

 

Genetics and Assays Working Group

 

Julie Kanter, MD (Co-Chair)
Medical University of South Carolina, Charleston, South Carolina

John Pierciey, MSc. (Co-Chair)
bluebird bio, Cambridge, Massachusetts

Gang Bao, PhD
Rice University, Houston, Texas

Francine Baker
Patient Advocate

Carlo Brugnara, MD
Boston Children’s Hospital, Boston, Massachusetts

Ashraf El Fiky, MBBCh, PhD
Emmes, Rockville, Maryland

Bindu George, MD
Food and Drug Administration, Silver Spring, Maryland

Joseph Gold, PhD
City of Hope, Duarte, California

Allison Intondi, PhD
Integral Medicines, San Francisco, California

Alison Kujawski, CHES
American Society of Gene & Cell Therapy, Washington, DC

LaTasha Lee, PhD, MPH
National Minority Quailty Forum, Washington, DC

John Manis, MD
Boston Children’s Hospital, Boston, Massachusetts

David C. Shyr, MD
Stanford Medicine, Palo Alto, California

John Tisdale, MD
National Institute of Health, Bethesda, Maryland

 

Monitoring Side Effects Working Group

 

Punam Malik, MD (Co-Chair)
Cincinnati Children's Hospital, Cincinnati, Ohio

Mark Walters, MD (Co-Chair)
University of California San Francisco, Oakland, California

Smita Bhatia, MD
University of Alabama at Birmingham, Birmingham, Alabama

Charles Chesson, PhD
Amerian Society of Hematology Research Collaborative, Washington, DC

Nancy Di Fronzo, PhD
National Institute of Health, Bethesda, Maryland

Mary Eapen, MRCPI, MS, MBBS
Medical College of Wisconsin, Milwaukee, Wisconsin

Barbara Kroner, PhD
Research Triangle Institute, Durham, North Carolina

Navneet Majhail, MD, MS
Cleveland Clinic, Cleveland, Ohio

Joseph McIntosh, MD
Aruvant Sciences, Inc., Basel, Switzerland

Jeremy Pantin, MD
HCA Healthcare, Nashville, Tennessee

Lydia Harley Pecker, MD
Johns Hopkins University, Baltimore, Maryland

Katy Rezvani, MD, PhD
The University of Texas, Houston, Texas

Shalini Shenoy, MD
Washington University School of Medicine, St. Louis, Missouri

 

Outcomes Working Group

 

Patrick Carroll, MD (Chair)
Johns Hopkins Medicine, Baltimore, Maryland

Francine Baker
Patient Advocate

M. A. Bender, MD, PhD
University of Washington, Seattle, Washington

Andres Brainsky, MD
CSL Behring, King of Prussia, Pennsylvania

Amanda Brandow, DO
Medical College of Wisconsin, Milwaukee, Wisconsin

Traci Clemons, PhD
Emmes, Rockville, Maryland

Beth Devine, PhD, PharmD, MBA
University of Washington, Seattle, Washington

Meghan Gallagher
bluebird bio, Cambridge, Massachusetts

Susan Geyer, PhD
Mayo Clinic, Tampa, Florida

Gregory Kato, MD
CSL Behring, King of Prussia, Pennsylvania

Megha Kaushal, MD
Food and Drug Administration, Silver Spring, Maryland

Julie Panepinto, MD, MSPH, FAAP
Medical College of Wisconsin, Milwaukee, Wisconsin

Jennifer Popovic, DVM
Research Triangle Institute, Waltham, Massachusetts

Amy Sobota, MD, MPH
Boston University, Boston, Massachusetts

Marsha Treadwell, PhD
University of California San Francisco, Oakland, California

Shauna Whisenton
American Society of Hematology Research Collaborative, Silver Spring, Maryland

Teonna Woolford
Patient/Advocate

 

Physical Examination/Medical History Working Group

 

Sophie Lanzkron, MD, MHS (Co-Chair)
Johns Hopkins School of Medicine, Balitmore, Maryland

Deepa Manwani, MD (Co-Chair)
Albert Einstein College of Medicine, Bronx, New York

Anirban Basu, PhD
University of Washington, Seattle, Washington

Velvet Brown-Watts
Patient Advocate/Caregiver

Victoria Coleman-Cowger, PhD
Emmes, Rockville, Maryland

Nahed El Kassar, MD, PhD
National Heart, Lung and Blood Institute, Bethesda, Maryland

Lisa Garrett, RN, CCRP
Washington University in St. Louis, St. Louis, Missouri

Stephanie Guarino, MD
Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware

Kavita Natrajan, MD
Food and Drug Administration , Silver Spring, Maryland

Helen Pan, PhD
Research Triangle Institute, Research Triangle Park, North Carolina

Robert Plovnick, MD, MS
American Society of Hematology, Washington, DC

Patricia Steinert, PhD, MBA
Center for International Blood & Marrow Transplant Research, CIBMTR®, Milwaukee, Wisconsin

Yvette Tanhehco, MD, PhD, MS
Columbia University Medical Center, New York, New York

Sara Vesely, PhD
The University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma

Kesha Weeks
Patient Advocate/Caregiver

 

NHLBI

 

Traci Mondoro, PhD
National Heart, Lung and Blood Institute, Bethesda, Maryland

Elizabeth Wagner, MPH
National Heart, Lung and Blood Institute, Bethesda, Maryland

Lis Welniak, PhD
National Heart, Lung and Blood Institute, Bethesda, Maryland

 

CDE Team

 

Sherita Alai, MS
Emmes, Rockville, Maryland

Karen Hewitt
Emmes, Rockville, Maryland

Rebecca Johnson, PhD
Emmes, Rockville, Maryland

Dan Sinnett, PhD
Emmes, Rockville, Maryland

Glenn Tucker
Emmes, Rockville, Maryland

References

Farrell, A. T., Panepinto, J., Carroll, C. P., Darbari, D. S., Desai, A. A., King, A. A., Adams, R. J., Barber, T. D., Brandow, A. M., DeBaun, M. R., Donahue, M. J., Gupta, K., Hankins, J. S., Kameka, M., Kirkham, F. J., Luksenburg, H., Miller, S., Oneal, P. A., Rees, D. C., Setse, R., … Zempsky, W. T. (2019). End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Blood advances, 3(23), 3982–4001. https://doi.org/10.1182/bloodadvances.2019000882
 
Farrell, A. T., Panepinto, J., Desai, A. A., Kassim, A. A., Lebensburger, J., Walters, M. C., Bauer, D. E., Blaylark, R. M., DiMichele, D. M., Gladwin, M. T., Green, N. S., Hassell, K., Kato, G. J., Klings, E. S., Kohn, D. B., Krishnamurti, L., Little, J., Makani, J., Malik, P., McGann, P. T., … Shenoy, S. (2019). End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood advances, 3(23), 4002–4020. https://doi.org/10.1182/bloodadvances.2019000883
 
Grinnon, S. T., Miller, K., Marler, J. R., Lu, Y., Stout, A., Odenkirchen, J., & Kunitz, S. (2012). National Institute of Neurological Disorders and Stroke Common Data Element Project - approach and methods. Clinical trials (London, England), 9(3), 322–329. https://doi.org/10.1177/1740774512438980
 
Hamilton, C. M., Strader, L. C., Pratt, J. G., Maiese, D., Hendershot, T., Kwok, R. K., Hammond, J. A., Huggins, W., Jackman, D., Pan, H., Nettles, D. S., Beaty, T. H., Farrer, L. A., Kraft, P., Marazita, M. L., Ordovas, J. M., Pato, C. N., Spitz, M. R., Wagener, D., Williams, M., … Haines, J. (2011). The PhenX Toolkit: get the most from your measures. American journal of epidemiology, 174(3), 253–260. https://doi.org/10.1093/aje/kwr193

Updates

There were five (5) domain working groups (WGs) which were organized to review the clinical research studies in sickle cell disease (SCD):

  1. Genetics/Assays;
  2. Physical Examination/Medical History;  
  3. Cardiopulmonary, Renal and Cerebrovascular;
  4. Outcomes; and,
  5. Monitoring Side Effects.

WGs are comprised of SCD adult and pediatric specialists, patient advocates, genetic therapy specialist, neuropsychologists, nurses, data managers, manufacturers, industry members, and academics.

The timeline of their work in 2020 is outlined below.