Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH)
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Kimmo Weisshaar1, Hannah Ewald2,3, Jörg Halter1
1Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.
Insights
New questionnaires were developed to assess symptoms and health-related quality of life in aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. These tools aim to improve understanding of the disease burden in these rare conditions.
Area of Science:
- Hematology
- Patient-Reported Outcomes
- Medical Informatics
Background:
- Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) outcomes have improved with new therapies.
- However, the disease burden, including health-related quality of life (HRQoL), is poorly understood due to inadequate symptom assessments.
- Standardized, patient-centered tools are needed for rare AA/PNH patient groups.
Purpose of the Study:
- To develop specific patient-reported outcome (PRO) questionnaires for AA and PNH.
- To create self-reporting tools for symptoms and HRQoL in AA/PNH.
- To facilitate data collection and patient care via electronic platforms.
Main Methods:
- A scoping review identified AA/PNH symptoms and prevalence from 62 and 45 studies, respectively (Phase I).
- Consensus rounds with 24 patients and 7 medical experts refined core symptoms and identified missing items (Phase II).
- AA/PNH-specific PRO questionnaires were designed based on selected measures and validated instrument concepts (Phase III).
Main Results:
- Eleven core symptoms, including HRQoL issues, were identified through consensus.
- Significant differences emerged between patient and expert symptom rankings.
- Patient- and expert-centered PRO questionnaires for AA and PNH were developed.
Conclusions:
- The development of symptom self-reporting questionnaires for AA and PNH is feasible.
- Disease-specific PRO questionnaires are ready for validation in a subsequent web-based feasibility study.
- These tools will aid in better assessing the disease burden and improving patient care for AA/PNH.
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.
