PhenX and PRO Measures for Sickle Cell Disease Research

Dr. Ellen M. Werner PhenX Measures for Sickle Cell Disease Research

Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)

Project Scientist

NIH Common Data Element Initiatives WebinarNational Heart, Lung, and Blood Institute

September 8, 2015

Standard measures and protocols will provide common data elements (CDEs) for sickle cell disease (SCD)

Standard measures and associated CDEs are needed to improve data quality and ensure data comparability facilitate meta-analyses develop guidelines for research implementation

and implementation science

1a. Motivation for CDEs in Sickle Cell Disease

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Rationale for Using PhenX? B/c of Criteria for Selecting PhenX Measures

Clearly defined Well established Broadly applicable Validated Reproducible Specific Reliable Standard measurement protocols exist

Identify common measures to promote data comparability across SCD research projects and cohorts

Promote adoption of these measures to characterize SCD complications pathogenesis and prognosis with treatments

Use accepted methodologies to develop CDE (and in ASCQ-Me) and PROs

1b. Objectives of PhenX Sickle Cell Disease Project2014 Admin Supplement to NHGRI

2. PhenX-SCD Engage Research Communities

PhenXSteering Committe

e

RTI

Sickle Cell

Disease Research

& Scientific

Panel (SRSP)

SCD WG 1

SCD WG 2

Core Collection

NHLBI NHGRI

Sickle Cell Disease Research and Scientific Panel and WG– Process

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In-Person MeetingDecide Measures for Outreach

Introductory Teleconference

TeleconferenceReview Outreach Results

Address SC ConcernsSelect Final Measures for

Toolkit

Review by Steering Committee

Compile outreach results

Prepare Datasheets

Final Measures Released in

Toolkit

Community Outreach

PhenX Sickle Cell Disease Research and Scientific Panel

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Name and Affiliation

James R. Eckman, MD, Co-ChairEmory University School of Medicine

Kim Smith-Whitley, MDChildren’s Hospital of Philadelphia 

Kathryn L. Hassell, MD, Co-Chair University of Colorado, Denver

John J. Strouse, MD, PhDJohns Hopkins University 

Jon A. Detterich, MD Children’s Hospital Los Angeles

James Taylor, MDNational Heart, Lung, and Blood Institute 

Jeffrey Glassberg, MD Mount Sinai Medical Center  

Marilyn J. Telen, MDDuke University School of Medicine

Allison A. King, MD, MPHWashington University School of Medicine

Ellen M. Werner, PhD, MANational Heart, Lung, and Blood Institute

Zora R. Rogers, MDUniversity of Texas, Southwestern

3 Components to PhenX-SCD Measures

1. Core Measures (Panel) 2. Cardiovascular, Renal and Pulmonary WG 3. Neurology, QoL Health Services WG

Examine sickle cell disease-related measures in the PhenX Toolkit to ensure that new measures complement existing Toolkit content

Identify measures and protocols currently in use in the field- PROMIS and ASCQ-Me- NINDS CDE Project- NIH CDE Project- Sickle Cell Disease Research Studies and Clinical Trials

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Cardiovascular, Renal and Pulmonary WG

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Name and Affiliation

Elizabeth S Klings, MD, ChairBoston University School of Medicine  

Antonio Guasch, MDEmory University 

Carol Blaisdell, MDNational Heart, Lung, and Blood Institute  

Vandana Sachdev, MDNational Heart, Lung, and Blood Institute  

Jon Detterich, MD Children’s Hospital Los Angeles 

John Wood, MD, PhDChildren’s Hospital Los Angeles  

Johnson Haynes, MDUniversity of South Alabama 

 

Neuro, QoL, Health Services WG

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Name and AffiliationRobert J. Adams, MD, MS, Co-ChairMedical University of South Carolina

Judith A. Paice, PhD, RNNorthwestern University

Julie A. Panepinto, MD, MSPH, FAAP, Co-ChairMedical College of Wisconsin 

Steven Pavlakis, MD Maimonides Medical Center 

F. Daniel Armstrong, PhDUniversity of Miami 

Marsha J. Treadwell, PhDChildren’s Hospital Oakland Research Institute

David C. Brousseau, MD, MS Medical College of Wisconsin 

 

Core: Tier 1 Relevant and essential to all areas of sickle cell disease research. NHLBI grantees/applicants conducting human-subject SCD studies are strongly

encouraged to use the Core Tier 1 measures

Core: Tier 2 Relevant to many areas of sickle cell disease research, but more specialized

than the Core Tier 1 measures. NHLBI grantees/applicants conducting human-subject SCD studies are

encouraged to incorporate the Core Tier 2 whenever possible and appropriate. NHLBI grantees are discouraged from using alternative measures in lieu of the

Core Tier 2 measures to collect similar data.

Specialty Collections The measures in those collections are deemed relevant within these more

specialized research domains. NHLBI grantees/applicants conducting human-subject studies in these specific

areas are strongly encouraged to incorporate the specialty measures into research protocols and are discouraged from using alternative measures to collect similar data.

Planned Use of PhenX SCD Measures forDiseasResearch

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13

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PROs at www.healthmeasures.net

Use and build on existing resources Plan for outreach to assure (to extent

possible) consensus among target stakeholders

Take advantage of in-reach Keep it simple (at first)

3. Lessons Learned

Several “gold-standard” measures have high participant/provider burden. Brain Morphology (CT and MRI)

Protocols that are not specific to sickle cell disease Medication adherence Access to and utilization of health care Quality of care indicators Immunization compliance

Acute vs. chronic pain

3. Lessons Learned: Challenges

Implementation Research FOAs Adoption by target users Wish list of gaps

G&D, sexual maturation and reproduction Orthopedic complications GI Ophthalmology

4. Plans for SCD CDEs

Health and Human Services (HHS) Liaisons

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Name and Affiliation

Kevin Abbott, MD, MPH The National Institute of Diabetes and Digestive and Kidney Diseases  

Joanne C. Odenkirchen, MPHNational Institute of Neurological Disorders and Stroke

Vence L. Bonham, Jr., J.D National Human Genome Research Institute 

Emmanuel K. Peprah, PhDNational Heart, Lung, and Blood Institute

Susan M. Czajkowski, PhD National Heart, Lung, and Blood Institute

Denise Stredrick, PhD Office of Disease Prevention

Michael M. Engelgau, MD, MS National Heart, Lung, and Blood Institute 

Mike Soucie, PhDCenters for Disease Control and Prevention 

Edward Donnell Ivy, MD, MPHHealth Resources & Services Administration

William P Tonkins Jr, Dr.PHNational Institute of Arthritis and Musculoskeletal and Skin Diseases

Harvey Luksenburg, M.D National Heart, Lung, and Blood Institute 

Tiina Urv, PhDNational Institute of Child Health and Human Development 

Joylene I. John-Sowah, MD, MPHNational Heart, Lung, and Blood Institute

James Witter, M.D., Ph.D. FACRNational Institute of Arthritis and Musculoskeletal and Skin Diseases