The lived experience of people with Glanzmann's thrombasthenia
| ISRCTN | ISRCTN63686338 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN63686338 |
| IRAS number | 308011 |
| ClinicalTrials.gov number | NCT05315232 |
| Secondary identifying numbers | IRAS 308011, CPMS 52408 |
- Submission date
- 06/04/2022
- Registration date
- 07/04/2022
- Last edited
- 10/10/2024
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English Summary
Background and study aims
Glanzmann’s thrombasthenia (GT) is a rare inherited blood clotting (coagulation) disorder where platelets (small cells in the blood) do not stick together properly. This leads to bruising and abnormal bleeding, which may be severe and may be life-threatening. Treatment is given at specialist hospitals which often means a delay in access to treatment and is burdensome to the patient and family. Males and females are affected equally, but women have many more symptoms because of menstruation and childbirth, often resulting in anaemia and severe limitations on lifestyle. This study aims to investigate the impact of living with GT on the affected person and their family - this work has never been done before.
Who can participate?
Adults aged over 16 years and parents of children aged under 16 years with GT via UK hospitals and social media. This will allow non-UK participants.
What does the study involve?
Part one of the study is in two parts - the first is the completion of the online survey using questionnaires that are recognised as useful in measuring quality of life, mental health, impact of bleeding and self-management. From these survey respondents the researchers will invite 30 people with GT (10 women, 10 men and 10 parents of affected children) to take part in an in-depth interview about the impact of living with GT on their daily lives.
What are the possible benefits and risks of participating?
There are no immediate benefits of taking part. The risk is that discussing sensitive issues may cause emotional distress. The researchers recognise this and will give participants a 'debrief sheet' with details of where to get support. Most treatment centres have access to psychological support services for patients with bleeding disorders.
Where is the study run from?
Haemnet (UK)
When is the study starting and how long is it expected to run for?
October 2021 to May 2023
Who is funding the study?
Hemab (Denmark)
Who is the main contact?
Dr Kate Khair
Kate@haemnet.com
Contact information
Scientific
74 Glenwood Rd
London
N15 3JR
United Kingdom
 ORCID ID |
0000-0003-2001-5958 |
|---|---|
| Phone | +44 (0)7515900812 |
| Kate@haemnet.com |
Study information
| Study design | Mixed-methods observational study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Internet/virtual |
| Study type | Quality of life |
| Participant information sheet | 41517_PIS_V1.1_24Mar22.pdf |
| Scientific title | Glanzmann's 360. The lived experience of people with Glanzmann's thrombasthenia: a mixed-methods observational study |
| Study acronym | Glanzmann's 360 |
| Study hypothesis | Glanzmann’s thrombasthenia (GT) is a rare inherited platelet disorder characterized by impaired platelet function due to absent or reduced glycoprotein IIb/IIIa complex which is instrumental in platelet aggregation. The bleeding phenotype varies but is usually severe with most people being diagnosed in early childhood. Patients and families experience considerable psychosocial impact and treatment burden. There remains a need for a comprehensive understanding of the experience of people with GT in order to identify: 1. The nature and range of symptoms that people present with to services 2. The variability in pathways through which patients progress to access appropriate care 3. The impact of living with GT on the individual’s quality of life and that of their family |
| Ethics approval(s) | Approved 29/03/2022, South Central - Oxford B Research Ethics Committee (Ground Floor, Temple Quay House, 2 The Square, Bristol, BS1 6NP, UK; +44 (0)207 104 8178, +44 (0)207104 8360, +44 (0)207 104 8270; oxfordbrec@hra.nhs.uk), ref: 22/SC/0095 |
| Condition | Glanzmann's thrombasthenia |
| Intervention | Participants will be identified via their treatment centres and will be sent a postcard inviting them to undertake an online survey using validated quality of life, impact of bleeding, psychological health and well-being instruments. They will then be able to opt in to an in-depth interview which will be offered either face to face or via an online platform, at a time that is convenient to them. |
| Intervention type | Other |
| Primary outcome measure | The impact of Glanzmann's thrombasthenia on affected individuals and their families, measured using an array of validated questionnaires (EQ5D, Minnesota Importance Questionnaire [MIQ], Patient Health Questionnaire-9 [PHQ9], Patient-Reported Outcomes Measurement Information System [PROMIS], Rosenberg's self-efficacy scale) via an online survey at baseline |
| Secondary outcome measures | 1. Satisfaction with current treatments and management approaches 2. Identification of areas of unmet need among people with Glanzmann's thrombasthenia Both will be measured using one in depth (up to 1-hour duration) qualitative interview per participant at the end of the study |
| Overall study start date | 01/10/2021 |
| Overall study end date | 01/05/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 16 Years |
| Sex | Both |
| Target number of participants | 150 |
| Total final enrolment | 104 |
| Participant inclusion criteria | 1. Confirmed diagnosis of inherited Glanzmann's thrombasthenia 2. Adults aged >16 years 3. Parents of children aged <16 years 4. Ability to read/write/speak English for questionnaire and interview completion 5. Give informed consent |
| Participant exclusion criteria | 1. Acquired Glanzmann's thrombasthenia 2. Participants unable to read/write/speak English 3. Those who do not consent |
| Recruitment start date | 28/05/2022 |
| Recruitment end date | 01/05/2023 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centres
Old Road
Headington
Oxford
OX3 7LE
United Kingdom
Birmingham
B4 6NH
United Kingdom
Basingstoke
RG24 9NA
United Kingdom
Cosham
Portsmouth
PO6 3LY
United Kingdom
London
EC1A 7BE
United Kingdom
London
NW3 2QG
United Kingdom
Bristol
BS2 8BJ
United Kingdom
Sponsor information
Research organisation
74 Glenwood Rd
London
N15 3JR
England
United Kingdom
| Phone | +44 (0)7515900812 |
|---|---|
| Kate@haemnet.com |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 31/10/2024 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | Planned publications in high-impact peer-reviewed journals. |
| IPD sharing plan | The datasets generated during and/or analysed during the current study will be available upon request from Dr Kate Khair, Director of Research at Haemnet (kate@haemnet.com). The data will become available at study end (December 2023) and will be available for 5 years. Reasonable requests for access to anonymised data will be reviewed by Haemnet as long as the data is to be used for non-commercial analyses. Participant consent to share anonymised data will have been granted. Data will be anonymised to study participant number only. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Participant information sheet | version 1.1 | 24/03/2022 | 07/04/2022 | No | Yes |
| Protocol file | version 1.0 | 28/02/2022 | 07/04/2022 | No | No |
| HRA research summary | 28/06/2023 | No | No | ||
| Results article | 09/10/2024 | 10/10/2024 | Yes | No |
Additional files
Editorial Notes
10/10/2024: Publication reference added.
23/08/2024: The intention to publish date was changed from 31/08/2024 to 31/10/2024.
05/06/2024: The intention to publish date was changed from 30/06/2024 to 31/08/2024.
03/04/2024: The intention to publish date was changed from 30/04/2024 to 30/06/2024.
18/12/2023: The intention to publish date has been changed from 31/12/2023 to 30/04/2024.
03/05/2023: Total final enrolment added.
26/05/2022: The recruitment start date has been changed from 02/05/2022 to 28/05/2022.
05/05/2022: Internal review.
07/04/2022: Trial's existence confirmed by the South Central - Oxford B Research Ethics Committee.
