Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (ePRO-AA-PNH)

Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study

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.

Study Overview

• Status: Completed
• Conditions: Aplastic Anemia; Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Other: symptom questionnaire; Other: recording of vital signs; Other: European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire; Other: interview

• Study Type: Observational
• Enrollment (Actual): 9

Contacts and Locations

Study Locations

• Switzerland
  • Basel, Switzerland, 4031
    • Division of Hematology, University Hospital Basel

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

All patients treated at the University Hospital Basel for AA and/or PNH

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
recruitment with the ePRO application (number)	Analyses of usability of the ePRO application by assessing recruitment with the ePRO application	Day 1= Day of inclusion
usability of the ePRO application	Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application	Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
acceptability of the ePRO application	Descriptive analyses of acceptability of the ePRO application by assessing app utilization	Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	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.	at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase

Collaborators and Investigators

• Sponsor: University Hospital, Basel, Switzerland
• Collaborators: Novartis; ProPatient foundation of the University Hospital Basel
• Investigators: Principal Investigator: Beatrice Drexler, Dr. med, Division of Hematology, University Hospital Basel

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 1, 2019
• Primary Completion (ACTUAL): July 6, 2020
• Study Completion (ACTUAL): July 6, 2020

Study Registration Dates

• First Submitted: October 15, 2019
• First Submitted That Met QC Criteria: October 15, 2019
• First Posted (ACTUAL): October 16, 2019

Study Record Updates

• Last Update Posted (ACTUAL): October 5, 2020
• Last Update Submitted That Met QC Criteria: September 30, 2020
• Last Verified: September 1, 2020

More Information

Terms related to this study

• Keywords: electronic patient-reported outcomes; electronic health (eHealth) technology; app utilization; ePRO application
• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Proteinuria; Anemia, Hemolytic; Bone Marrow Failure Disorders; Myelodysplastic Syndromes; Anemia; Anemia, Aplastic; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: 2019-01563; me18Drexler

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No