Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
electronic patient-reported outcomes, electronic health (eHealth) technology, app utilization, ePRO application
Brief summary
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective disease are available. As a consequence, patients may be left on their own between infrequent follow-ups at a specialist center. A web-based symptom-monitoring application can support selfmanagement and patient empowerment and promotes a patient- centered interdisciplinary team approach in the context of a disease management program. This pilot study is to investigate usability and feasibility of the electronic Patient-Reported Outcome (ePRO) application in AA/PNH by assessing recruitment, app utilization, data collection, functionality, acceptability after using and working with the ePRO application.
Interventions
A weekly filled in symptom questionnaire for reported symptoms of AA and/or PNH (yes/no, grade, date). Grading according to current grading systems (Common Terminology Criteria for Adverse Events,CTCAE). Data collection and processing via the ePRO application by Kaiku Health Ltd
Record function for vital parameters (blood pressure, temperature and pulse); Data collection and processing via the ePRO application by Kaiku Health Ltd
OTHEREuropean Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
The EORTC QLQ-C30 incorporates nine multi-item scales: five functional scales (Physical, Role, Cognitive, Emotional and Social Functioning); three symptom scales (Fatigue, Pain and Nausea/Vomiting); and a Global Health Status/QoL scale. Six single item scales are also included (Dyspnoea, Insomnia, Appetite Loss, Constipation, Diarrhoea and Financial Difficulties). All of the scales range in score from 0 to 100. A high score for a functional scale represents a high/healthy level of functioning whereas a high score for a symptom scale or item represents a high level of symptomatology or problems.
OTHERinterview
Interview with the patients and responsible medical team by phone or during hospital visit about functional problems regarding the application (duration: 15-30 min) to avoid incorrect use of the application, at week 3 and month 3 and month 6
Sponsors
ProPatient foundation of the University Hospital Basel
Novartis
University Hospital, Basel, Switzerland
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* AA (acquired and hereditary) and/or PNH patients * Minimal level of computer literacy with prior email experience and access to an Internet connection
Exclusion criteria
* Mental alteration or psychiatric disease that can compromise written informed consent or adherence to the protocol and monitoring of the trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| recruitment with the ePRO application (number) | Day 1= Day of inclusion | Analyses of usability of the ePRO application by assessing recruitment with the ePRO application |
| usability of the ePRO application | Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase | Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application |
| acceptability of the ePRO application | Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase | Descriptive analyses of acceptability of the ePRO application by assessing app utilization |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in quality of life assessed by the European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire (EORTC QLQ-C30 questionnaire) | at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase | Change in quality of life assessed by the EORTC QLQ-C30 questionnaire. The EORTC QLQ-C30 incorporates nine multi-item scales: five functional scales (Physical, Role, Cognitive, Emotional and Social Functioning); three symptom scales (Fatigue, Pain and Nausea/Vomiting); and a Global Health Status/QoL scale. Six single item scales are also included (Dyspnoea, Insomnia, Appetite Loss, Constipation, Diarrhoea and Financial Difficulties). All of the scales range in score from 0 to 100. A high score for a functional scale represents a high/healthy level of functioning whereas a high score for a symptom scale or item represents a high level of symptomatology or problems. |
Countries
Switzerland
Outcome results
None listed