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

Expert Rev Hematol. 2020 Nov;13(11):1165-1173. doi: 10.1080/17474086.2020.1830370. Epub 2020 Oct 15.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Adult
  • Anemia, Sickle Cell / psychology
  • Anemia, Sickle Cell / therapy*
  • Child
  • Clinical Protocols
  • Clinical Trials as Topic / methods
  • Data Accuracy
  • Data Collection / methods
  • Databases, Factual
  • Decision Making, Shared
  • Humans
  • Information Systems / organization & administration
  • National Institutes of Health (U.S.)
  • Pain Measurement
  • Patient Reported Outcome Measures*
  • Patient-Centered Care / methods*
  • Quality of Life
  • Self Report
  • Severity of Illness Index
  • United States