PhenX Measures for Sickle Cell Disease (SCD) Research

In 2014, the National Heart, Lung, and Blood Institute (NHLBI) launched the PhenX Measures for Sickle Cell Disease Research Project to establish a framework for data sharing across different SCD research projects. As a result of this project, a Core Collection and two Specialty Collections (Cardiovascular, Pulmonary, and Renal; Neurology, Quality of Life, and Health Services) of standard measures were selected by the Sickle Cell Disease Research and Scientific Panel (SRSP) and SCD Working Groups. These Collections were added to the Toolkit in July of 2015. Consistent use of the standard measures in these Collections will establish a common currency to help researchers to better understand the etiology, progression, and treatment of SCD.

The SCD Collections are the result of a collaborative, consensus effort by the extramural scientific community and staff members at NHLBI, National Human Genome Research Institute, National Institute of Neurological Disorders and Stroke, Centers for Disease Control and Prevention, National Institute of Diabetes and Digestive and Kidney Diseases, Health Resources & Services Administration, National Institute of Child Health and Human Development, and the National Institute of Arthritis and Musculoskeletal and Skin Diseases. Further details about the PhenX Measures for Sickle Cell Disease Research project can be found here.

Core Collections

The Sickle Cell Disease Research and Scientific Panel (SRSP) selected a core collection of measures recommended for use by all NHLBI-funded investigators performing human subjects research in SCD. These measures are designed to create a common framework to facilitate cross-study data aggregation and to address questions related to the effects of a key set of SCD-related factors on relevant outcomes.

The Core: Tier 1 collection includes 11 measures that are deemed relevant and essential to all areas of SCD research. Six of the Core: Tier 1 measures are focused on demographic (age, gender, race, ethnicity) and anthropometrics-related (height, weight) measures. Core: Tier 1 Collection also includes measures covering pulse oximetry, hemoglobin characterization, history of transfusion, and episodes of SCD-related pain episodes.

The Core: Tier 2 includes 16 measures that are relevant to many areas of SCD research but are more specialized and require a greater time commitment. Core: Tier 2 measures are complementary to Core: Tier 1 and address additional areas relevant to the SCD-related studies including demographics and socioeconomic status, use of alcohol, tobacco and other substances, and ancestry.

Specialty Collections

The measures in Cardiovascular, Pulmonary, and Renal and Neurology, Quality of Life, and Health Services Specialty Collections are complementary to Core: Tier 1 and Core: Tier 2 and are deemed relevant and essential within these more specialized research domains. The Cardiovascular, Pulmonary, and Renal Specialty Collection includes 14 measures that address heart disease, lung function, and biomarkers for hemolysis, anemia, iron overload, and renal function. The Neurology, Quality of Life, and Health Services Specialty Collection includes 8 measures that address developmental delays, risk factors and outcomes for stroke, quality of life, and quality of care.