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NCT Number: NCT04128943

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

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.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Anemia, Hemolytic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes Paroxysmal Nocturnal Hemoglobinuria

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Division of Hematology, University Hospital Basel

Basel, 4031, Switzerland

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Primary outcomes

  1. recruitment with the ePRO application (number)

    Time frame: Day 1= Day of inclusion

    Analyses of usability of the ePRO application by assessing recruitment with the ePRO application

  2. usability of the ePRO application

    Time frame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase

    Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application

  3. acceptability of the ePRO application

    Time frame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase

    Descriptive analyses of acceptability of the ePRO application by assessing app utilization

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)

    Time frame: at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase

    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.

Sponsors and collaborators

Lead sponsor

University Hospital, Basel, Switzerland

Other

Collaborators

  • Novartis
  • ProPatient foundation of the University Hospital Basel

Registry information

Official study title

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

Acronym: ePRO-AA-PNH

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Oct 16, 2019
Registry last updated
Oct 5, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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