The lived experience of people with von Willebrand disease

ISRCTN	ISRCTN14289653
DOI	https://doi.org/10.1186/ISRCTN14289653
IRAS number	328463
ClinicalTrials.gov number	NCT06064643
Secondary identifying numbers	IRAS 328463, CPMS 57207

Submission date: 21/06/2023
Registration date: 24/08/2023
Last edited: 18/12/2024

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Background and study aims
von Willebrand Disease (vWD) is an inherited blood clotting disorder that causes prolonged or spontaneous bleeding from birth. Affected individuals tend to bruise easily, may have frequent nosebleeds, bleeding from the gums, joints and sometimes stomach and intestinal bleeding (more common later in life). vWD also causes prolonged bleeding following injury, trauma, or surgery (including dental work). Women with vWD can have prolonged and heavy periods, they may also have an increased risk of excessive bleeding during pregnancy and childbirth. The severity and frequency of the bleeding episodes in vWD can vary greatly among affected individuals, even within the same family.

Treatment varies based on the diagnosis and rate and type of any bleeding experienced though is usually ‘on-demand’ (given after bleeding occurs) with some patients prophylaxis (treatment given to prevent bleeding from occurring) may be needed.
The lack of routine prophylaxis means that most patients are reliant on hospital-delivered care, which may involve frequent clinic appointments, causing prolonged bleeding due to a lack of timely administration of treatment. This can result in concurrent illnesses such as iron deficiency anaemia, which further impacts on the quality of life of affected individuals.
There remains a need for a comprehensive understanding of the experience of people with vWD in order to identify:

- The nature and range of symptoms that people experience and how these vary with the different disease subtypes.
- The variability in pathways through which people with vWD progress to access appropriate care.
- The impact of living with vWD on the individual’s quality of life.

Who can participate?
Adults over 16 years in the UK and Ireland & over 18 years in the US with a confirmed diagnosis of vWD.

What does the study involve?
Every participant will be asked to complete an online survey
Some will be asked if they would be willing to do an interview
some will be asked to complete a 30-day bleed diary

What are the benefits and risks of participating?
There are no risks in taking part and there are no direct medical benefits to you. However, participation may help others with the condition in the future.

Where is the study run from?
Oxford University Hospitals NHS Foundation Trust (UK)

When is the study starting and how long is it expected to run for?
June 2023 to December 2024

Who is funding the study?
The study is being funded by Hemab, a biotechnology company based in Denmark.

Who is the Main contact for the study?
Simon Fletcher, simon@haemnet.com

Contact information

Mr Simon Fletcher
Principal Investigator

74 Greenwood Road
London.
N15 3JR
United Kingdom

ORCID ID	0000-0001-9018-6176
Phone	+44 7891038065
Email	simon@haemnet.com

Study information

Study design	Qualitative mixed methods research project
Primary study design	Observational
Secondary study design	Cohort study
Study setting(s)	Home, Hospital
Study type	Quality of life
Participant information sheet	Not available in web format, please use contact details to request a participant information sheet
Scientific title	The lived experience of people with von Willebrand disease: a mixed methods study
Study acronym	vWD360
Study objectives	To identify the lived experience of people with von Willebrand Disease (vWD) and including: 1. Experiences of bleeding 2. Impact on quality of life 3. Access to therapeutic options 4. Satisfaction with current treatments and management 5. Areas of unmet need
Ethics approval(s)	Approved 27/10/2023, London - Brighton and Sussex Research Ethics Committee (Health Research Authority, 2 Redman Place, London, E20 1JQ, United Kingdom; +44 (0)20 7104 8202; approvals@hra.nhs.uk), ref: 23/PR/1013
Health condition(s) or problem(s) studied	von Willebrand Disease (any known subgroup)
Intervention	All participants will be asked to complete an online survey. 30 participants will also be asked to take part in a single semi-structured qualitative interview. 50 participants will be asked to complete a 30-day bleed diary
Intervention type	Other
Primary outcome measure	Bleeding rates, daily activities, pain/discomfort and anxiety/depression will be measured using a retrospective, self-reported, validated survey (EQ-5D, Menstrual Impact Questionaire (for women) PHQ8 and GAD 7). This data will also be recorded prospectively using a 30 day bleed dairy.
Secondary outcome measures	Treatment satisfaction and assessment of unmet needs will be measured through analysis of the descriptive interview narratives of the lives of people with vWD collected at a single time point
Overall study start date	22/06/2023
Completion date	31/12/2024

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	16 Years
Sex	Both
Target number of participants	Survey - 450, Interviews - 30, Bleed Diary - 50.
Key inclusion criteria	1. Adults aged 16 years and above (UK and Ireland) and adults aged over 18 years (in USA) with a confirmed diagnosis of inherited vWD of known diagnostic subtype and vWF level. 2. For the qualitative interview-based substudy, 30 adults who have completed the survey and who wish to be interviewed will be purposively selected for a broad range of ages and diagnostic subtype. 3. For the bleed diary substudy, 50 adults who have completed the survey and who wish to take part will be purposively selected for a broad range of ages and diagnostic subtype.
Key exclusion criteria	1. Have acquired vWD 2. Have other inherited bleeding disorders 3. Do not wish to participate in or to consent to the study. 4. Are under 16 years old (UK & Ireland) or 18 years old (US). 5. Those for whom written/spoken English would prohibit participation will also be excluded.
Date of first enrolment	18/12/2023
Date of final enrolment	31/12/2024

Locations

Countries of recruitment

England
Ireland
United Kingdom
United States of America

Study participating centre

Oxford University Hospitals NHS Foundation Trust

John Radcliffe Hospital
Headley Way
Headington
Oxford
OX3 9DU
United Kingdom

Sponsor information

Haemnet
Research organisation

74 Greenwood Road
London
N15 3JR
England
United Kingdom

Phone	+44 7711 654 839
Email	research@haemnet.com
Website	https://www.haemnet.com

Funders

Funder type

Industry

Hemab

No information available

Results and Publications

Intention to publish date	31/12/2025
Individual participant data (IPD) Intention to share	Yes
IPD sharing plan summary	Available on request
Publication and dissemination plan	The results of the study will be published in peer-reviewed journals as well as at conferences as either presentations or posters. All participants will be sent a copy of all of the publications.
IPD sharing plan	The datasets generated durang and/or analysed during the current study are/will be available upon reasonable request from Simon Fletcher (Principal Investigator) simon@haemnet.com

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Protocol file	version 1.5		27/10/2023	No	No

Additional files

ISRCTN14289653_PROTOCOL_V1.5.pdf

Editorial Notes

18/12/2024: The overall end date was changed from 31/12/2024 to 31/05/2025.
08/04/2024: ClinicalTrials.gov number added.
11/12/2023: The recruitment end date has been changed from 31/12/2023 to 31/12/2024.
27/10/2023: Protocol uploaded, ethics approval details added. The recruitment start date was changed from 01/10/2023 to 18/12/2023.
05/09/2023: Internal review.
21/08/2023: Trial's existence confirmed by NHS HRA.