PhenX Toolkit Updated to Include the Burden of Pain in Sickle Cell Disease

Shortly after presenting data from her study on self-efficacy and stigma in adults with sickle cell disease (SCD) at the American Society of Hematology (ASH) Annual Meeting and Exposition, Stephanie Guarino, MD, MSHP, Nemours Dupont Hospital For Children, Center for Special Health Care Needs/Institute on Research in Equity and Community Health, Christiana Care, shared results from another study.

Guarino and a team of investigators aimed to expand the content in the PhenX toolkit to include practices for SCD-related pain. The National Heart, Lung, and Blood Institute (NHLBI) launched the PhenX (consensus measures for Phenotypes and eXposures) Measures for Sickle Cell Disease Research Project in 2014 to establish a framework for data sharing across different SCD research projects.

"One of the challenges when you're doing research in patients with sickle cell disease is the lack of standardized measures to collect data," she explained in an interview with HCPLive. "The populations are spread out across the country and many studies may only capture a small number of patients. So, when you try to generalize some of those findings across studies, it can be challenging."

According to Guarino, the goal of the toolkit is to bring experts together to vet the many metrics that are out there.

"It provides clinicians, or researchers who are developing studies, the very best evidence based guidelines and tools to collect data. It's a repository of measures that one might incorporate into a study with the goal of offering best practices and tools that have been validated in other studies, and then can be evaluated across studies to give us more meaningful data," she said.

Measuring and managing pain is a difficult task, and many investigations have been performed to further the collective understanding. Pain in sickle cell disease is often severe, and individuals experience it differently.

"The idea that we could collect more data to better understand pain will eventually lead to more effective treatments. What we know is that there are some metrics, or general pain, or pain in other conditions, that don't really apply to sickle cell disease," Guarino shared. "So, the goal was to create a set of metrics, or a toolbox for people that was sickle-cell-disease-specific, almost exclusively, so that we can get the very best information for this population."