Clinical trial NCT04128943: Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Official title: Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Aplastic Anemia
Paroxysmal Nocturnal Hemoglobinuria
Keywords: electronic patient-reported outcomes
electronic health (eHealth) technology
app utilization
ePRO application
Lead sponsor: University Hospital, Basel, Switzerland
Sponsor class: OTHER
Collaborators: name: ProPatient foundation of the University Hospital Basel
name: Novartis
First posted: 2019-10-16
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: All patients treated at the University Hospital Basel for AA and/or PNH

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.


Eligibility criteria: 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

Primary outcomes:
measure: recruitment with the ePRO application (number)
timeFrame: Day 1= Day of inclusion
measure: usability of the ePRO application
timeFrame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
measure: acceptability of the ePRO application
timeFrame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
Secondary outcomes:
measure: 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)
timeFrame: at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: Division of Hematology, University Hospital Basel
Study officials:
affiliation: Division of Hematology, University Hospital Basel
name: Beatrice Drexler, Dr. med
role: PRINCIPAL_INVESTIGATOR
References:
citation: Banziger S, Weisshaar K, Arokoski R, Gerull S, Halter J, Rovo A, Bargetzi M, Goede JS, Senft Y, Valenta S, Passweg JR, Drexler B. Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria-a pilot study (ePRO-AA-PNH). Ann Hematol. 2023 Jan;102(1):199-208. doi: 10.1007/s00277-022-05012-5. Epub 2022 Nov 3.