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

NCT04128943 | Completed

• 1 other identifier: 2019-01563; me18Drexler
• Study Type: observational
• Target: 9
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia; Paroxysmal Nocturnal Hemoglobinuria

Keywords

electronic patient-reported outcomes; electronic health (eHealth) technology; app utilization; ePRO application

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• 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

Interventions

symptom questionnaire OTHER

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

recording of vital signs OTHER

Record function for vital parameters (blood pressure, temperature and pulse); Data collection and processing via the ePRO application by Kaiku Health Ltd

European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire OTHER

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.

interview OTHER

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• AA (acquired and hereditary) and/or PNH patients
• Minimal level of computer literacy with prior email experience and access to an Internet connection

You may not qualify if:

• Mental alteration or psychiatric disease that can compromise written informed consent or adherence to the protocol and monitoring of the trial

Sponsors & Collaborators

• University Hospital, Basel, Switzerland: lead
• ProPatient foundation of the University Hospital Basel: collaborator
• Novartis: collaborator

Study Sites (1)

Division of Hematology, University Hospital Basel

Basel, 4031, Switzerland

Location

Related Publications (1)

• 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.

  PMID: 36326854 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic; Hemoglobinuria, Paroxysmal

Interventions

Interviews as Topic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Anemia, Hemolytic; Myelodysplastic Syndromes

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Beatrice Drexler, Dr. med

  Division of Hematology, University Hospital Basel

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2019
• First Posted: October 16, 2019
• Study Start: November 1, 2019
• Primary Completion: July 6, 2020
• Study Completion: July 6, 2020
• Last Updated: October 5, 2020

Record last verified: 2020-09

Locations

CH (1)