Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Completed

• Conditions: Aplastic Anemia; Paroxysmal Nocturnal Hemoglobinuria

• Registration Number: NCT04128943
• Lead Sponsor: University Hospital, Basel, Switzerland

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-10-15
• Study First Submitted QC: 2019-10-15
• Study First Posted: 2019-10-16
• Study Start: 2019-11-01
• Primary Completion: 2020-07-06
• Study Completion: 2020-07-06
• Status Verified: 2020-09
• Last Update Submitted: 2020-09-30
• Last Update Posted: 2020-10-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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
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
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Division of Hematology, University Hospital Basel; 🇨🇭 Basel, Switzerland