Adding Centralized Electronic Patient-Reported Outcome Data Collection to an Established International Clinical Outcomes Registry

Abstract

The importance of patient-reported outcomes (PROs) in cellular therapies, including hematopoietic cell transplantation (HCT) is highlighted in this study. Longitudinal collection of PROs in a registry is recommended for several reasons, yet to date, PROs are not routinely collected from HCT patients to augment clinical registry data. The aim of this study was to determine the feasibility of electronic PRO data collection by a national clinical outcomes registry, by assessing differences between who does and does not report PROs. We conducted a cross-sectional pilot collection of PROs from HCT recipients after treatment using computer-adapted tests from the Patient-Reported Outcome Measurement Information System (PROMIS). We implemented centralized data collection through the Center for International Blood and Marrow Transplant Research (CIBMTR) among patients who underwent HCT for myelodysplastic syndromes (MDS), were at least 6 months post-HCT, and spoke English or Spanish. The main objective was identifying patient, disease, and transplant-related differences associated with completion of electronic PROs. Patients were excluded from analysis if they were determined to be ineligible (deceased, did not speak English or Spanish, refused to be contacted by the CIBMTR). A total of 163 patients were contacted and potentially eligible to participate; of these, 92 (56%) enrolled and 89 (55%) completed the PRO assessment. The most frequent reason for incomplete surveys was inability to contact patients (n = 88), followed by declining to participate in the study (n = 37). There were no sociodemographic or age differences between those who completed the PRO survey (n = 89) and eligible nonresponders (n = 155). Patient scores were within 3 points of the US average of 50 for all symptoms and functioning except physical functioning. Responders and nonresponders did not exhibit meaningfully different sociodemographic characteristics. Difficulty contacting patients posed the greatest barrier and also provided the greatest opportunity for improvement. Once enrolled, survey completion was high. These results support standardizing centralized PRO data collection through the CIBMTR registry.

Keywords: Cellular therapy; Clinical outcomes registry; Hematopoietic cell transplantation; Patient-Reported Outcome Measurement Information System (PROMIS); Patient-reported outcomes.

Figure 1.. Flow Diagram for Analytic Sample, indicating Refusals, Implicit Refusals, and Unknown Eligibility

Unknown eligibility = Patients were never able to be contacted to confirm eligibility; Refusal = Patients were contacted and verbally declined to be contacted about research or declined this study; Implicit Refusal = Patients eligibility was confirmed and initial contact was made, but patient did not respond to follow up contact or complete survey

Figure 2:

The average number of days between milestones made by the CIBMTR survey research group at each stage of activity within the ePRO