Abstract
Sickle cell disease significantly impacts one’s quality of life (QOL); thus, randomized controlled trials (RCTs) have integrated patient-reported outcomes (PROs) to assess patients’ health from their perspective. We aim to evaluate the completeness of reporting of PROs included in sickle cell disease RCTs. We searched MEDLINE, Embase and Cochrane Central Register of Controlled Trials (CENTRAL) for published sickle cell disease RCTs with at least one PRO measure from 2006 to 2021. In a masked, duplicate fashion, two investigators evaluated RCTs using the Consolidated Standards of Reporting in Trials (CONSORT)-PRO adaptation and Cochrane Collaboration Risk of Bias (RoB) 2.0 tool. The primary objective was mean percent completeness of the CONSORT-PRO adaptation. Additional relationships between trial characteristics and completeness of reporting were evaluated. Mean completeness of reporting of RCTs was 41.49% (SD = 20.90). Randomized controlled trials with primary outcomes were more complete (57.50%, SD = 8.33) than RCTs with secondary PROs (33.48%, SD = 20.91). We did not find a significant difference in completion between trials with primary PROs and secondary PROs (t1 = 2.07; p = 0.06). Our secondary objectives included factors that may be associated with completeness of PRO reporting. Of the 12 included studies, five were considered to be overall ‘high’ RoB (41.67%). In each of the five domains, the majority of studies received ‘low’ RoB evaluations. Incomplete PRO reporting was common within sickle cell RCTs. Therefore, we recommend future RCTs including PROs should take measures to increase completeness of reporting.
Acknowledgments
In memoriam of Abbey Renner (Oklahoma State University Center for Health Sciences Ringgold Standard Institution, College of Osteopathic Medicine, Tulsa, OK, USA); we are grateful for her leadership and expertise during the drafting of this manuscript. We are grateful to April Schweikhard (University of Oklahoma-Tulsa, Schusterman Library, Tulsa, OK, USA), who assisted in the development of our search string and the Oklakoma State University Medical Library, Tulsa, OK, USA, for their procurement of relevant literature.
Disclosure statement
No financial or other sources of support were provided during the development of this manuscript. Dr. M. Hartwell reports receiving funding from the National Institute of Justice for work unrelated to the current subject. Dr. M. Vassar reports receipt of funding from the National Institute on Drug Abuse, the National Institute on Alcohol Abuse and Alcoholism, the US Office of Research Integrity, Oklahoma Center for Advancement of Science and Technology, and internal grants from Oklahoma State University Center for Health Sciences, all outside of the present study.
Data availability statement
Data are available upon reasonable request from the corresponding author.