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.

Data Standards

NIH Resources
NIH Resources
CRF Module/Guideline CDEs
PROMIS Item Bank v. 1.0 - Pain Intensity Scale CDE Details
PROMIS Item Bank v. 1.0 - Satisfaction with Participation in Discretionary Social Activities Calibrated Items CDE Details
PROMIS Item Bank v. 1.0 - Satisfaction with Participation in Social Roles Calibrated Items CDE Details
PROMIS Item Bank v1.0 - Dyspnea Functional Limitations CDE Details
PROMIS Item Bank v1.0 - Dyspnea Severity CDE Details
PROMIS Item Bank v1.0 - Emotional Distress - Anxiety CDE Details
PROMIS Item Bank v1.0 - Emotional Distress - Depression CDE Details
PROMIS Item Bank v1.0 - Fatigue Calibrated Items CDE Details
PROMIS Item Bank v1.0 - Self-Efficacy for Managing Emotions CDE Details
PROMIS Item Bank v1.0 - Sleep Disturbance CDE Details
PROMIS Item Bank v1.0 - Sleep Related Impairment CDE Details
PROMIS Item Bank v1.1 - Anger CDE Details
PROMIS Item Bank v1.1 - Pain Behavior CDE Details
PROMIS Item Bank v1.1 - Pain Interference CDE Details
PROMIS Item Bank v1.2 - Global Health CDE Details
PROMIS Item Bank v2.0 - Cognitive Function CDE Details
PROMIS Item Bank v2.0 - Physical Function CDE Details
PROMIS Item Bank v2.0 - Satisfaction with Social Roles and Activities Calibrated Items CDE Details
PROMIS Numeric Rating Scale v. 1.0 - Pain Intensity 1a CDE Details
PROMIS Pediatric Item Bank v1.0 - Life Satisfaction- Short Form 8a CDE Details
PROMIS Pediatric Item Bank v1.0 - Life Satisfaction-Short Form 4a CDE Details
PROMIS Pediatric Item Bank v1.0 - Meaning and Purpose- Short Form 8a CDE Details
PROMIS Pediatric Item Bank v1.0 - Meaning and Purpose-Short Form 4a CDE Details
PROMIS Scale v2.0 - Nociceptive Pain Quality 5a CDE Details
Genetics
Genetics
CRF Module/Guideline CDEs
HLA Form - 1507 CDE Details
Genotype
CRF Module/Guideline CDEs
HLA Form - 1507 CDE Details
Participant History and Family History
Clinical Trial Participation
CRF Module/Guideline CDEs
Withdrawal of Consent Form (WOC) CDE Details
General Health History
CRF Module/Guideline CDEs
Intake Medical History CDE Details
Toxicity Form CDE Details
Assessments and Examinations
Vital Signs and Laboratory Tests
CRF Module/Guideline CDEs
PhenX Toolkit: Body temperature CDE Details
Safety Data
Adverse Events
CRF Module/Guideline CDEs
Adverse Events CDE Details
Outcomes and End Points
Cardiac Function Testing/Status
CRF Module/Guideline CDEs
Toxicity Form CDE Details
Global Outcome
CRF Module/Guideline CDEs
PROMIS Item Bank v1.2 - Global Health CDE Details
Health-Related Quality of Life Outcome
CRF Module/Guideline CDEs
Stroke Specific Quality of Life Scale (SS-QOL) CDE Details
Pulmonary Function Testing/Respiratory Status
CRF Module/Guideline CDEs
PhenX Toolkit: Lung Function - Diffusion Capacity CDE Details
PhenX Toolkit: Lung Function - Lung Volume CDE Details
Quality of Life/Patient Reported Outcomes
CRF Module/Guideline CDEs
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Emotional Impact Item Bank CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Emotional Impact - Short Form CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Pain Episode Frequency and Severity Measure CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Pain Impact Item Bank CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Pain Impact - Short Form CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Sickle Cell Disease Medical History Checklist CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Sleep Impact Item Bank CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Sleep Impact - Short Form CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Social Functioning - Item Bank CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Social Functioning - Short Form CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Stiffness Impact - Item Bank CDE Details
Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) v2.0 Stiffness Impact - Short Form 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
PROMIS Item Bank v1.0 (PRO) CDE Details
PROMIS Pediatric Item Bank v1.0 - Life Satisfaction- Short Form 8a CDE Details
PROMIS Pediatric Item Bank v1.0 - Life Satisfaction-Short Form 4a CDE Details
Renal Function Testing/Status
CRF Module/Guideline CDEs
Toxicity Form CDE Details
Vascular Function Testing/Status
CRF Module/Guideline CDEs
Toxicity Form CDE Details

History

Working groups are currently formed to review five clinical research study domains:

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

These groups are comprised of SCD adult and pediatric specialists, patient advocates, genetic therapy specialists, neuropsychologists, nurses, data managers, manufacturers, industry members and academics. Members of each group will meet over a period of six months and then on an ad hoc as needed to complete the process. Over the six months the group members will systematically review the data forms, discuss the form content and vote until a consensus regarding the form content is reached. The product of the working group will then be reviewed by the CTD Subcommittee before posting the forms for Public Review on the CureSCi website (curesickle.org). After a Public Review period and follow-up with working groups, version 1.0 of the standardized data forms will be released to the CureSCi web site. The forms will also be made available through  the National Library of Medicine (NLM) website (https://cde.nlm.nih.gov/). This will be an iterative process and feedback from users will be encouraged.

The current recommendations are Proposed (Version 0.0) data standards.

References

References.

Updates

There are five (5) domain Work groups (WGs) which are being organized to review the clinical research studies in sickle cell disease:

  1. Genetics/Assays;
  2. Physical Examination/Medical History;  
  3. Cardiopulmonary and Renal Function;
  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 next steps are outlined below.  This page will be updated on a monthly basis to inform the public of the progress.