Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2020 Nov;13(11):1165-1173.
doi: 10.1080/17474086.2020.1830370. Epub 2020 Oct 15.

What is the future of patient-reported outcomes in sickle-cell disease?

Sharon A Singh  1 , Nitya Bakshi  2   3 , Prashant Mahajan  4 , Claudia R Morris  3   5
Affiliations
Review

What is the future of patient-reported outcomes in sickle-cell disease?

Sharon A Singh et al. Expert Rev Hematol. 2020 Nov.

Abstract

Introduction: Sickle cell disease (SCD) is a complex, chronic disease caused by abnormal polymerization of hemoglobin, which leads to severe pain episodes, fatigue, and end-organ damage. Patient reported outcomes (PROs) have emerged as a critical tool for measuring SCD disease severity and response to treatment.

Areas covered: Authors review the key issues involved when deciding to use a PRO in a clinical trial. We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice.

Expert opinion: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries.

Keywords: ASCQ-Me; HRQOL; PRO; PROMIS; Patient reported outcome; Peds-QL; SF-36; hemoglobinopathy; quality of life; sickle cell disease.

PubMed Disclaimer

References

    1. Piel FB, Patil AP, Howes RE, et al. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013. January; 381(9861):142–51. - PMC - PubMed
    1. Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018. 03; 4:18010. - PubMed
    1. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010. December;376(9757):2018–31. - PubMed
    1. Ataga KI, Desai PC. Advances in new drug therapies for the management of sickle cell disease. Expert Opin Orphan Drugs. 2018;6(5):329–343. - PMC - PubMed
    1. Kalpatthi R, Novelli EM. Measuring success: utility of biomarkers in sickle cell disease clinical trials and care. Hematology Am Soc Hematol Educ Program. 2018. 11;2018(1):482–492. - PMC - PubMed

Publication types

MeSH terms