The impact of vaso-occlusive crises and disease severity on quality of life and productivity among patients with sickle cell disease in the US

Abstract

Aim

Sickle cell disease (SCD) is a lifelong blood disorder affecting approximately 100,000 individuals in the United States (US). A number of new treatments have recently become available to improve SCD clinical outcomes, but it is unclear how treatment innovations that reduce disease severity could affect patients’ humanistic and economic outcomes.

Methods and materials

To answer this question, an online survey of US adult residents with a self-reported SCD diagnosis was conducted. Humanistic outcomes based on health-related quality of life (HRQoL)) were assessed during and outside of vaso-occlusive crises (VOCs). Economic outcomes were measured by annual household income and whether the respondent received disability insurance.

Results

Among the 301 respondents completing the survey, average age was 34.4 years and 73.4% were female. Average HRQoL, measured using health utilities, were 0.311 (95% CI: 0.286, 0.337) during a VOC and 0.738 (0.720, 0.756) not during a VOC. The likelihood of claiming disability insurance was correlated with more frequent VOCs (0 VOCs: 12% vs. ≥4 VOCs: 47%, p = .002) and disease severity (Severity Class II: 16% vs. Severity Class III: 39%, p = .03). There was a weak relationship between VOC frequency and household income (0 VOCs: $47,488 vs. ≥4 VOCs: $34,569, p = .06) and no evidence of a relationship between disease severity class and income (Severity Class II: $42,443 vs. Severity Class III: $36,842, p = .29).

Conclusion

In conclusion, disease severity, strongly predicted worse self-reported HRQoL, moderately predicted increased likelihood of collecting disability insurance, and weakly predicted lower household income levels.

Keywords:

• sickle cell disease
• vaso-occlusive crisis
• quality of life

Transparency

Declaration of funding

This manuscript was funded by Novartis Pharmaceuticals Corporation

Declaration of financial/other relationships

HT discloses personal consulting fees from Novartis Pharma AG, Hoffman La-Roche, Pfizer and Eli Lilly. NS reports consulting fees and funding from Novartis Pharmaceutical Co. JS reports equity in Precision Medicine Group. JS was an employee at PRECISIONheor at the time this research was conducted. MB reports employment by Novartis Pharmaceutical Co. at the time this study was conducted. LZ reports employment by PRECISIONheor at the time this study was conducted. AR reports employment by Optum at the time this study was conducted. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgements

None stated.