Background The introduction of new therapy modalities has significantly improved the outcome of aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. However, relatively little is known about the exact disease burden of AA/PNH since standardized assessments of symptoms including health-related quality of life (HRQoL) are frequently missing or inadequately designed for this rare patient group. We aimed to develop AA/PNH-specific questionnaires for self-reporting of symptoms, which could be included in electronic platforms for data collection and patient care. Methods By scoping review, we extracted any reported symptoms in AA/PNH and their prevalence from the literature (Phase I). Consensus rounds with patients and medical experts were conducted to identify core symptoms reported in the literature and to add missing items (Phase II). Ultimately, AA/PNH-specific patient-reported outcome (PRO) questionnaires including the selected measures were designed (Phase III). Results AA symptoms from 62 and PNH symptoms from 45 observational studies were extracted from the literature. Twenty-four patients and seven medical experts identified 11 core symptoms including HRQoL issues after three consensus rounds. Significant differences in the symptom ranking of patients versus medical experts could be observed. Therefore, patient- as well as expert-centered PRO questionnaires in AA and PNH were created following the concepts of validated instruments. Conclusion The development of symptom self-reporting questionnaires for AA and PNH was feasible and the disease-specific PRO questionnaires can now be validated within a web-based workflow in a subsequent feasibility study.

Background: The introduction of new therapy modalities has significantly improved the outcome of aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. However, relatively little is known about the exact disease burden of AA/PNH since standardized assessments of symptoms including health-related quality of life (HRQoL) are frequently missing or inadequately designed for this rare patient group. We aimed to develop AA/PNH-specific questionnaires for self-reporting of symptoms, which could be included in electronic platforms for data collection and patient care. Methods: By scoping review, we extracted any reported symptoms in AA/PNH and their prevalence from the literature (Phase I). Consensus rounds with patients and medical experts were conducted to identify core symptoms reported in the literature and to add missing items (Phase II). Ultimately, AA/PNH-specific patient-reported outcome (PRO) questionnaires including the selected measures were designed (Phase III). Results: AA symptoms from 62 and PNH symptoms from 45 observational studies were extracted from the literature. Twenty-four patients and seven medical experts identified 11 core symptoms including HRQoL issues after three consensus rounds. Significant differences in the symptom ranking of patients versus medical experts could be observed. Therefore, patient- as well as expert-centered PRO questionnaires in AA and PNH were created following the concepts of validated instruments.Conclusion: The development of symptom self-reporting questionnaires for AA and PNH was feasible and the disease-specific PRO questionnaires can now be validated within a web-based workflow in a subsequent feasibility study.

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.

Background: The introduction of new therapy modalities has significantly improved the outcome of aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. However, relatively little is known about the exact disease burden of AA/PNH since standardized assessments of symptoms including health-related quality of life (HRQoL) are frequently missing or inadequately designed for this rare patient group. We aimed to develop an AA/PNH-specific questionnaire for self-reporting of symptoms, which could be included in electronic platforms for data collection and patient care. Methods: By scoping review, we extracted any reported symptoms in AA/PNH and their prevalence from the literature (Phase I). Consensus rounds with patients and medical experts were conducted to identify core symptoms reported in the literature and to add missing items (Phase II). Ultimately, an AA/PNH-specific patient-reported outcome (PRO) questionnaire including the selected measures was designed (Phase III). Results: Sixty-one symptoms in AA and 45 symptoms in PNH were extracted from the literature. Twenty-four patients and seven medical experts identified 11 core symptoms including HRQoL issues after three consensus rounds. Significant differences in the symptom ranking of patients versus medical experts could be observed. We determined a compromise between experts’ opinions on physical constraints and patients’ frequent demands on HRQoL. These items built the basis for the development of two PRO questionnaires separate for AA and PNH following the concepts of validated instruments. Conclusion: The development of a symptom self-reporting questionnaire for AA and PNH was feasible and the PRO questionnaire can now be validated within a web-based workflow in a subsequent feasibility study.

Introduction: Electronic patient-reported outcome (ePRO) systems have demonstrated to improve patient outcome in cancer, but have not yet been evaluated in patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). This pilot study evaluated the feasibility of an ePRO monitoring, self-management advice and warning system customized for AA and PNH patients.Methods: After adapting an ePRO system to the needs of AA and PNH patients (ePRO-AA-PNH), the application was tested by patients and their medical team for 6 months. Based on a predefined algorithm symptom reporting triggered self-management advice and prompts to contact clinicians in case of severe symptoms. Meanwhile the medical team could monitor ePROs in real-time and receive alerts of severe symptoms electronically. Results: Nine patients completed the study, showing a high adherence rate to the weekly symptom-reporting (72%), which decreased from 91 % to 53 % over the study period. Satisfaction was high: ePRO-AA-PNH was found easy to use, to understand and to integrate 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 missed the integration to clinical workflows and the hospital electronic health records (EHR). Conclusion: An ePRO system customized for AA and PNH was feasible to patients and their medical team in terms of adherence, satisfaction and performance, showing a high potential for these rare and chronic conditions. However, the integration into clinical workflows and hospital EHR is crucial for routine use.Trial registration: ClinicalTrials.gov identifier: NCT04128943, October 16 2019, https://clinicaltrials.gov/ct2/show/NCT04128943