Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (ePRO-AA-PNH) | Aplastic Anemia & MDS International Foundation
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (ePRO-AA-PNH)

Clinical Trial: NCT04128943

For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

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.

Status: 
Recruiting
Study Date: 
Fri, 11/01/2019 to Tue, 12/01/2020
Bone Marrow Disease(s): 
  • aplastic anemia
  • paroxysmal nocturnal hemoglobinuria (PNH)
Intervention: 
Other: symptom questionnaire 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 Other: recording of vital signs Record function for vital parameters (blood pressure, temperature and pulse); Data collection and processing via the ePRO application by Kaiku Health Ltd Other: European 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. Other: interview 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