Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria—a pilot study (ePRO-AA-PNH)

Abstract

Introduction Electronic patient-reported outcomes (ePRO) are increasingly recognized in health care, as they have been demonstrated to improve patient outcomes in cancer, but have been less studied in rare hematological diseases. The aim of this study was to develop and test the feasibility of an ePRO system specifically customized for aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). Methods After performing a user-centered design evaluation an ePRO system for AA and PNH patients could be customized and the application was tested by patients and their medical teams for 6 months. Symptom-reporting triggered self-management advice for patients and prompts them to contact clinicians in case of severe symptoms, while the medical team received alerts of severe symptoms for patient care. Results All nine included patients showed a high adherence rate to the weekly symptom-reporting (72%) and reported high satisfaction. The system was rated high for usage, comprehensibility, and integration into daily life. Most patients (78%) would continue and all would recommend the application to other AA/PNH patients. Technical performance was rarely a barrier and healthcare providers saw ePRO-AA-PNH as a useful supplement, but the lacking integration into the hospital information system was identified as a major barrier to usage. Conclusion An ePRO system customized for AA and PNH was feasible in terms of adherence, satisfaction, and performance, showing a high potential for these rare conditions in terms of data collection and patient guidance. However, the integration into clinical workflows is crucial for further routine use. Trial registration ClinicalTrials.gov NCT04128943.

Perspective and Experience of Patients with Aplastic Anemia on Medication Adherence

“…Although some studies explore interventions to improve patient medication compliance, the status of medication implementation, medication persistence, and related factors are unclear, which is not conducive to the substantive progress of interventions. 7 , 8 …”

“…Although some studies explore interventions to improve patient medication compliance, the status of medication implementation, medication persistence, and related factors are unclear, which is not conducive to the substantive progress of interventions. 7,8 As demonstrated by multiple studies, medication adherence is a common but complex individual behavior faced by patients with chronic medical problems related to multiple factors. [9][10][11] When it comes to aplastic anemia patients, the oral medications mentioned, like androgens, often show significant side effects, such as severe acne, local skin reactions, mood swings, or sexual arousal.…”