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Author Notes:

Correspondence: Carol M. Hamilton, RTI International, 3040 Cornwallis Rd, Research Triangle Park, NC 27709; e-mail: chamilton@rti.org

Contribution: J.E.R., K.L.H., E.M.W, W.H., and C.M.H. participated in the drafting and writing of the manuscript; W.H. created Tables 1-​-4;4; E.S.K. participated in writing the sections about the cardiovascular, pulmonary, and renal WG (WG 1) and provided a critical review of the manuscript; and R.J.A. and J.A.P. participated in writing the sections about the neurology, quality-of-life, and health services WG (WG 2) and provided a critical review of the manuscript.

The authors wish to acknowledge all Sickle Cell Disease Research and Scientific Panel (SRSP) and Working Group (WG) members: SRSP members included J.R.E. (co-chair), K.L.H. (co-chair), Jon A. Detterich, Jeffrey Glassberg, Allison A. King, Zora R. Rogers, Kim Smith-Whitley, John J. Strouse, James Taylor, Marilyn J. Telen, and E.M.W. WG 1 members included E.S.K. (chair), Carol Blaisdell, Jon A. Detterich, Antonio Guasch, Johnson Haynes, Vandana Sachdev, and John Wood. WG 2 members included R.J.A. (co-chair), J.A.P. (co-chair), F. Daniel Armstrong, David C. Brousseau, Judith A. Paice, Steven Pavlakis, and Marsha J. Treadwell.

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Subject:

Research Funding:

Funding for the PhenX Toolkit was provided by National Institutes of Health, National Human Genome Research Institute (via Cooperative Agreement U41 HG007050) with cofunding from the National Institute on Drug Abuse.

The sickle cell disease Administrative Supplement to the U41 was supported with funding from the National Heart, Lung, and Blood Institute.

Standard measures for sickle cell disease research: the PhenX Toolkit sickle cell disease collections.

Tools:

Journal Title:

Blood Advances

Volume:

Volume 1, Number 27

Publisher:

, Pages 2703-2711

Type of Work:

Article | Final Publisher PDF

Abstract:

Standard measures and common data elements for sickle cell disease (SCD) will improve the data quality and comparability necessary for cross-study analyses and the development of guidelines that support effective treatments and interventions. In 2014, the National Institutes of Health, National Heart, Lung, and Blood Institute (NHLBI) funded an Administrative Supplement to the PhenX Toolkit (consensus measures for Phenotypes and eXposures; https://www.phenxtoolkit.org/) to identify common measures to promote data comparability across SCD research. An 11-member Sickle Cell Disease Research and Scientific Panel provided guidance to the project, establishing a core collection of SCD-related measures and defining the scope of 2 specialty collections: (1) cardiovascular, pulmonary, and renal complications, and (2) neurology, quality-of-life, and health services. For each specialty collection, a working group of SCD experts selected high-priority measures using a consensus process that included scientific community input. The SCD measures were released into the Toolkit in August 2015. The 25 measures included in the core collection are recommended for use by all NHLBI-funded investigators performing human-subject SCD research. The 10 neurology, quality-of-life, and health services measures and 14 cardiovascular, pulmonary, and renal measures are recommended for use within these specialized research areas. For SCD and other researchers, PhenX measures will promote collaborations with clinicians and patients, facilitate cross-study analysis, accelerate translational research, and lead to greater understanding of SCD phenotypes and epigenetics. For clinicians, using PhenX measures will help elucidate the etiology, progression, and treatment of SCD, leading to improved patient care and quality of life.
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