Patient-Reported Outcomes and Economic Burden of Adults with Sickle Cell Disease in the United States: A Systematic Review

Soyon Lee; Diana K Vania; Menaka Bhor; Dennis Revicki; Seye Abogunrin; Grammati Sarri

doi:10.2147/IJGM.S257340

Patient-Reported Outcomes and Economic Burden of Adults with Sickle Cell Disease in the United States: A Systematic Review

Int J Gen Med. 2020 Jul 7:13:361-377. doi: 10.2147/IJGM.S257340. eCollection 2020.

Authors

Soyon Lee¹, Diana K Vania², Menaka Bhor¹, Dennis Revicki³, Seye Abogunrin², Grammati Sarri²

Affiliations

¹ Novartis, East Hanover, New Jersey, USA.
² Evidera, London W6 8BJ, UK.
³ Evidera, Bethesda, MD 20814, USA.

Abstract

Purpose: To systematically estimate the patient-reported outcomes (PROs) and economic burden of sickle cell disease (SCD) among adults in the United States (US).

Patients and methods: Two systematic literature reviews (SLRs), one each for the PROs and economic topics, were performed using MEDLINE and Embase to identify observational studies of adults with SCD. Included studies were published between 2007 and 2018 and evaluated health-related quality of life (HRQL), function, healthcare resource utilization (HCRU), or costs. Given the high degree of clinical and methodological heterogeneity, findings were summarized qualitatively.

Results: The SLRs identified 7 studies evaluating the PROs and 15 studies evaluating the economic burden meeting the pre-specified selection criteria. The PRO evidence showed the prevalence of depression and anxiety to be 21-33% and 7-36%, respectively, in adults with SCD. The mean SF-36 physical summary scores ranged from 33.6 to 59.0 and from 46.3 to 61.5 for the mental summary scores. Overall HRQL for adults with SCD was poor and significantly worse in those with opioid use. Adult SCD patients were found to have varying rates of emergency department (ED) utilization (0.3-3.5 annual ED visits), hospitalizations (0.5-27.9 per patient per year), and/or readmission (12-41%). Key factors associated with significant HCRU were age, dental infection, and SCD-related complications. SCD specialized care settings and SCD intensive management strategy were reported to significantly decrease the number of hospitalizations.

Conclusion: This systematic evidence synthesis found that disease burden measured by PROs and economic burden of SCD on adults in the US are substantial despite the availability of approved SCD treatments during 2007-2018. The use of hydroxyurea, optimal management with opioids, and employing intensive treatment strategies may help decrease the overall burden to patients and healthcare systems. Published data on costs associated with SCD are limited and highlight the need for more economic studies to characterize the full burden of the disease.

Keywords: economic burden; health-related quality of life; patient-reported outcomes; sickle cell disease.

Grants and funding

Novartis provided the funding for the study.