Similar Patient-Reported Quality of Life in the US and UK in Patients with Sickle Cell: A Comparative Grndad/Cousin Study

Background: The Globin Research Network for Data and Discovery, GRNDaD (2016), registry and its recently initiated partner, Collaborating to Understand Sickle Cell Disease Internationally, CoUSIn (2021), were developed by providers at sickle cell centers in the US and UK, respectively. The overall aim is a robust registry that will lead to an increased understanding of the natural history of sickle cell disease (SCD) and to use quality improvement tools to measure and improve guideline-based care for all individuals with this disease. In addition to collecting clinical and laboratory patient-level data for an accurate and detailed phenotype, the registries also collect patient-reported outcomes including data on quality of life (QOL) through the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) survey tool1. The purpose of this abstract is to compare QOL results from adult patients from the UK and US, and to describe clinical characteristics associated with QOL domains in available US data. Methods: GRNDaD is an ongoing prospective registry that is recruiting adult and pediatric patients at 19 sites across the US. CoUSIn is the UK equivalent, using the same variables which opened for enrollment for adults in September 2021. The full registry has over 300 unique variables extracted from medical records. In addition, survey instruments including ASCQ-Me and the brief pain inventory are available for patient direct data-entry into the REDCap registry. This analysis includes the first 106 participants from the UK and 486 participants in the US who have to date completed at least one ASCQ-Me questionnaire. All the ASCQ-Me metrics are created from standardized T-scores using an earlier SCD cohort, in which, for each measure, 50 is the average score, scores above which suggest better health or QOL. Different ASCQ-Me domains were collected in each country based on preference (Table). T-tests were used to compare differences in patient-reported outcomes from the UK and US. Wilcoxon rank sum tests were used to identify patient characteristics associated with the outcomes. Results: 486 of 1240 adult patients in the US and 106 of 236 UK patients have completed at least one ASCQ-Me survey. 58% of US and 62% of the UK respondents had hemoglobin SS/Sβ0thal. 55% of US and 60% of UK respondents were female. The median age of the US cohort was 35 (IQR-27-45) and the UK cohort was 39 (IQR-26-50). Results of ASCQ-Me by domain and country can be found in the Table and Figure. There were no statistically significant differences between patient-reported outcomes between UK and US respondents across the four common ASCQ-Me domains in which data was collected, when controlling for age and sex. For the US cohort, when comparing patients with SS/Sβ0thal on medication versus on chronic transfusion for their disease modifying therapy there was no statistically significant difference in any of the five ASCQ-Me domains. Discussion: While not yet fully mature, both the GRNDaD and CoUSIn registries demonstrate the feasibility of collecting patient reported outcomes from real world settings. Interestingly, despite vast differences in health care delivery, scores on ASCQ-Me are not different between countries and average scores are notable for being above the average score of the standardized subjects in whom the tool was developed, suggesting better QOL. In the US cohort, the lack of association of those on DMT requires further study to determine if these tools will be sensitive to changes in therapy in longitudinal follow-up and whether adherence to therapy might impact scores. In addition, planned registry improvements, to more concretely capture opioid and anti-inflammatory agent use may improve our patient-centered understanding of how best to decrease symptom burden in SCD. 1. Keller S, Yang M, Treadwell MJ, Hassell KL. Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-M CoUSIn is supported by a grant from bluebird bio Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal