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

NCT ID: NCT04128943

Last Updated: 2020-10-05

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 9 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-11-01
• Study Completion Date: 2020-07-06

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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Interventions

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

Intervention Type: 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

Intervention Type: 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.

Intervention Type: 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

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ProPatient foundation of the University Hospital Basel

UNKNOWN

Sponsor Role: collaborator

Novartis

INDUSTRY

Sponsor Role: collaborator

University Hospital, Basel, Switzerland

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Beatrice Drexler, Dr. med

Role: PRINCIPAL_INVESTIGATOR

Division of Hematology, University Hospital Basel

Locations

Division of Hematology, University Hospital Basel

Basel, , Switzerland

Countries

Switzerland

References

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.

Reference Type: DERIVED

PMID: 36326854 (View on PubMed)

Other Identifiers

2019-01563; me18Drexler

Identifier Type: -

Identifier Source: org_study_id