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.
Article highlights
Sickle cell disease (SCD) is a common inherited hemoglobinopathy, and is a complex, chronic disease caused by abnormal polymerization of hemoglobin in red blood cells
SCD disease manifestations are variable, but most patients have recurrent pain episodes, hemolysis, fatigue from severe anemia, end-organ damage, and early mortality
PROs (patient reported outcomes) are a report of the status of the patients health condition that comes directly from the patient without interpretation of the patient’s response by clinician or anyone else’.
Many SCD disease symptoms are subjective so PROs can complement clinician reported outcomes by measuring the patient experience
PRO measures in pediatrics may be self-reported or by the parent-proxy report, although the two types of data are not interchangeable and may not correlate in some situations
PRO measures are increasingly being used in clinical trials and there are several excellent generic and disease specific tools validated for use in sickle cell disease as primary or secondary outcomes
Significant challenges remain in adapting PROs to clinical practice and for use in developing countries
Declaration of interest
C Morris is the inventor or co-inventor of several UCSF-Benioff Children’s Hospital Oakland patents/patent-pending applications that include nutritional supplements, and biomarkers of cardiovascular disease related to arginine bioavailability, is an inventor of an Emory University School of Medicine patent application for a nutritional supplement, is a consultant for Pfizer and has received research support from MAST Therapeutics, the United States Food and Drug Administration, the Health Resources and Service Administration, and the National Institutes of Health. This project was supported in part by NIH/NCCIH K24AT009893 to Dr. Morris. P Mahajan’s effort is partially supported by grants awarded by the Agency for Healthcare Research and Quality (1R18HS026622, 1R01HS024953) and Eunice Kennedy Shriver National Institute of Child Health and Human 1R01HD08523. SA. Singh, MD has been a consultant for Emmaus Medical, Inc. 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.
Reviewer disclosures
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.