A small pilot feasibility study for a possible randomised control trial comparing clinical outcomes and quality of life following two different transfusion strategies in children undergoing allogeneic hematopoietic stem cell transplant (HSCT)
| ISRCTN | ISRCTN17438123 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN17438123 |
| IRAS number | 246398 |
| Secondary identifying numbers | CPMS 41935 |
- Submission date
- 20/05/2019
- Registration date
- 23/05/2019
- Last edited
- 20/01/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Other
Plain English Summary
Background and study aims
This is a small pilot feasibility study for a possible randomised controlled trial comparing clinical outcomes and quality of life following two different transfusion strategies in children undergoing allogeneic hematopoietic stem cell transplant (HSCT). Children who undergo allogeneic HSCT usually receive red blood cell transfusions in the initial period after their transplant, due to reduced haemoglobin levels as a result of bone marrow suppression. In this situation, the red cell transfusions are commonly given when the child’s haemoglobin falls below 70-80g/L. The evidence for this practice is based largely on clinical trials which suggest that in most situations transfusing red cells at haemoglobin thresholds of 70g/L is sufficient, with no benefit of transfusing at higher haemoglobin thresholds. However, this evidence is
largely from adult trials in non-HSCT patients. Clinical studies are required to help us understand the best strategy for use of red cell transfusions in paediatric HSCT.
Who can participate?
Patients aged between and including 1 and 17 years who are due to undergo allogeneic Haematopoietic Stem Cell Transplant and are expected to require red cell transfusions can participate.
What does the study involve?
Patients aged between and including 1 and 17 years will participate in this study for the first 100 days after their transplant. Patients will be randomised between the two red cell transfusion strategies: transfusing at a haemoglobin threshold of ≤ 65 g/L (Arm A) or ≤ 80 g/L (Arm B).
Participants of 8 years and older will be asked to fill in short questionnaires about their quality of life at certain intervals during the period of their study. All parents will be asked to fill in an equivalent parent questionnaire
What are the possible benefits and risks of participating?
Benefits: Patients in Arm B (higher haemoglobin threshold) may receive a few additional transfusions than they would have had if they had not participated in the trial and patients in Arm A (lower haemoglobin threshold) may receive slightly fewer transfusions.
Risks: As for all blood transfusions, there are small risks associated with having a transfusion. These risks are described in the national transfusion leaflets produced by NHS Blood and Transplant and SNBTS. This includes the very small risks of catching an infection, such as hepatitis B or HIV (the virus that causes AIDS). Other risks include allergic reactions or developing antibodies to the blood transfused. However, all the paediatric patients undergoing allogeneic HSCT participating in the trial will be expected to receive red cell transfusions and are likely to have had multiple transfusions previously, and so these are not new risks for them. NHSBT has a rigorous system for testing all blood donations for viruses such as hepatitis B or HIV.
Where is the study run from?
NHSBT Clinical Trials Unit, Cambridge, UK.
When is the study starting and how long is it expected to run for?
June 2019 to June 2023
Who is funding the study?
NHS Blood and Transplant, UK
Who is the main contact?
Valerie Hopkins,
Valerie.Hopkins@nhsbt.nhs.uk
Contact information
Scientific
Clinical Trials Unit
NHS Blood & Transplant
Long Road
Cambridge
CB2 0PT
United Kingdom
| Phone | 01223 588188 |
|---|---|
| Valerie.hopkins@nhsbt.nhs.uk |
Scientific
NHS Blood and Transplant
Clinical Trials Unit
Cambridge Donor Centre
Long Road
Cambridge
CB2 0PT
United Kingdom
 ORCID ID |
0000-0002-7631-9275 |
|---|---|
| Phone | +44 (0)7385 964361 |
| Claire.Rourke2@nhsbt.nhs.uk |
Study information
| Study design | Interventional; Design type: Process of Care, Management of Care |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Red Cell Transfusion in Paediatric Allogeneic HSCT |
| Study acronym | RePAST |
| Study hypothesis | The principal research question is to see if it is feasible to recruit and achieve adherence to a transfusion protocol when randomising patients to one of two haemoglobin thresholds. The study will be considered feasible and worthy of further development into a larger randomised trial if at least half of eligible patients are randomised, and if there is no evidence that the adherence to the protocol (such that transfusions are given appropriately in accordance with the randomised haemoglobin threshold policy) is lower than 70% in either arm. The results will help design a larger multi-centre randomised, controlled trial to be conducted in the future, or may show that it is not possible to carry out such a trial. |
| Ethics approval(s) |
Approved 24/06/2019, London – Camden and King’s Cross REC (Friends House, Margaret Fell Room, 173 - 177 Euston Road, London, NW1 2BJ, United Kingdom; +44 (0)2071048238; camdenandkingscross.rec@hra.nhs.uk), ref: 19/LO/0714 |
| Condition | Red cell transfusion |
| Intervention | This is a small pilot feasibility study for a possible randomised controlled trial comparing clinical outcomes and quality of life following two different transfusion strategies in children undergoing allogeneic hematopoietic stem cell transplant (HSCT). Children who undergo allogeneic HSCT usually receive red blood cell transfusions in the initial period after their transplant, due to reduced haemoglobin levels as a result of bone marrow suppression. In this situation, the red cell transfusions are commonly given when the child’s haemoglobin falls below 70-80g/L. The evidence for this practice is based largely on clinical trials which suggest that in most situations transfusing red cells at haemoglobin thresholds of 70g/L is sufficient, with no benefit of transfusing at higher haemoglobin thresholds. However, this evidence is largely from adult trials in non-HSCT patients. Clinical studies are required to help us understand the best strategy for use of red cell transfusions in paediatric HSCT. Patients aged between and including 1 and 17 years will participate in this study for the first 100 days after their transplant. Patients will be randomised between the two red cell transfusion strategies: transfusing at a haemoglobin threshold of < = 65 g/L (Arm A) or < = 80 g/L (Arm B). Participants of 8 years and older will be asked to fill in short questionnaires about their quality of life at certain intervals during the period of their study. All parents will be asked to fill in an equivalent parent questionnaire. |
| Intervention type | Other |
| Primary outcome measure | Current primary outcome measures as of 20/01/2025: Recruitment: The percentage of eligible children recruited and randomised into the study Adherence: The percentage of Hb measurements where appropriate action was taken in accordance with the randomised policy Previous primary outcome measures: Adherence outcomes: 1. The proportion of enrolled participants for whom the transfusion policy was successfully followed 2. The proportion of red cell transfusions given in accordance with the randomisation policy where the correct dose was given 3. The mean pre-transfusion, post-transfusion and overall haemoglobin concentration (g/L) up to day 100 of HSCT and the difference between the two arms 4. The percentage of pre-transfusion haemoglobin concentrations falling at or below, or above the threshold haemoglobin of the red cell transfusion threshold assigned 5. The percentage of post-transfusion haemoglobin concentrations falling below, or at and above the target haemoglobin of the red cell transfusion threshold assigned 6. Drop out: the proportion of randomised participants who were withdrawn from the study 7. Quality of Life (QoL) questionnaire compliance: the proportion of QoL questionnaires completed at each time point |
| Secondary outcome measures | Current secondary outcome measures as of 20/01/2025: Adherence outcomes: 1. The proportion of enrolled participants for whom the transfusion policy was successfully followed 2. The proportion of RBC transfusions given in accordance with the randomised policy where the correct dose was given 3. The mean pre-transfusion, post-transfusion and overall Hb concentration up to Day 100 of HSCT and the difference between the two arms 4. The percentage of pre-transfusion Hb concentrations falling at or below, or above the threshold Hb of the red cell transfusion threshold assigned 5. The percentage of post-transfusion Hb concentrations falling below, or at and above the target Hb of the red cell transfusion threshold assigned 6. Drop-out: the proportion of randomised participants who were withdrawn from the study 7. Quality of life (QoL) questionnaire compliance: the proportion of QoL questionnaires completed at each time point Clinical outcomes: 1. Death: All-cause mortality at Day 100 of HSCT 2. Clinically significant bleeding (WHO Grade 3-4) 3. Red Blood Cell Exposure: The RBC transfusion volume per recipient weight (ml/Kg), number of red blood cell units administered and number of RBC transfusion episodes up to death/Day 100 of HSCT (whichever comes first). Number of patients requiring an additional transfusion within 24 hours, and reason 4. Proportion of participants experiencing thromboembolic and ischaemic events 5. Transfusion reactions 6. Grade of acute graft versus host disease and Bearman toxicity score 7. Veno-occlusive disease 8. Admission to Paediatric Intensive Care (reason and duration of stay) 9. Number of platelet transfusions 10. Health-related QoL scores Previous secondary outcome measures: Clinical outcomes: 1. Death: all-cause mortality at Day 100 of HSCT 2. Clinically significant bleeding (WHO Grade 3-4) 3. Red cell exposure: the red cell transfusion volume per recipient weight (ml/Kg), number of red cell units administered and number of red cell transfusion episodes up to death/Day 100 of HSCT (whichever comes first) 4. Number of patients requiring an additional transfusion within 24 hours, and reason 5. Proportion of participants experiencing thromboembolic and ischaemic events 6. Transfusion reactions 7. Grade of acute graft versus host disease and Bearman toxicity score 8. Veno-occlusive disease 9. Admission to Paediatric Intensive Care (reason and duration of stay) 10. Number of platelet transfusions 11. Health-related Quality of Life Scores. |
| Overall study start date | 01/01/2017 |
| Overall study end date | 30/06/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 1 Year |
| Upper age limit | 18 Years |
| Sex | Both |
| Target number of participants | Planned Sample Size: 34; UK Sample Size: 34 |
| Total final enrolment | 34 |
| Participant inclusion criteria | 1. Children undergoing allogeneic HSCT 2. Aged at least 1 year and under 18 years at time of consent 3. Expected to require red cell transfusions |
| Participant exclusion criteria | 1. Patients for whom the attending haematologist feels allocation to either a restrictive or liberal policy of red cell transfusion is not appropriate (e.g. acutely unwell, bleeding or ‘unstable’. 2. Children undergoing HSCT for haemoglobinopathy or red cell aplasia. |
| Recruitment start date | 01/06/2019 |
| Recruitment end date | 01/12/2020 |
Locations
Countries of recruitment
- England
- Scotland
- United Kingdom
Study participating centres
Bristol
BS1 3NU
United Kingdom
Gartnavel Royal Hospital
1055 Great Western Road
Glasgow
G12 0XH
United Kingdom
Sheffield
S10 2TH
United Kingdom
Oxford Road
Manchester
M13 9WL
United Kingdom
Sponsor information
Hospital/treatment centre
Clinical Trials Unit
NHS Blood & Transplant
Long Road
Cambridge
CB2 0PT
England
United Kingdom
| Phone | 01223 588920 |
|---|---|
| heather.smethurst@nhsbt.nhs.uk | |
"ROR" |
https://ror.org/0227qpa16 |
Funders
Funder type
Government
No information available
Results and Publications
| Intention to publish date | 31/12/2024 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal. |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are/will be available upon request from NHSBT Clinical Trials Unit (CTU@nhsbt.nhs.uk). |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| HRA research summary | 28/06/2023 | No | No |
Editorial Notes
20/01/2025: The primary and secondary outcome measures were updated.
17/01/2025: IRAS number added.
16/07/2024: The following changes were made to the study record:
1. Sheffield Childrens Hospital and Manchester University NHS Foundation Trust were added as study participating centres.
2. The intention to publish date was changed from 30/06/2024 to 31/12/2024.
18/01/2024: The intention to publish date was changed from 30/01/2024 to 30/06/2024.
19/09/2023: The intention to publish date was changed from 30/09/2023 to 30/01/2024.
29/03/2023: The following changes were made to the trial record and the plain English summary updated accordingly:
1. The overall trial end date was changed from 31/03/2023 to 30/06/2023.
2. The intention to publish date was changed from 30/06/2023 to 30/09/2023.
26/01/2023: The following changes were made to the trial record:
1. The overall trial end date was changed from 01/12/2022 to 31/03/2023.
2. The intention to publish date was changed from 31/03/2023 to 30/06/2023.
3. Total final enrolment and IPD sharing statement added.
12/01/2023: Contact details updated.
13/12/2022: The intention to publish date was changed from 31/12/2022 to 31/03/2023.
11/03/2020: The following changes were made to the trial record:
1. The age group was corrected from 'adult' to 'child'
2. The intention to publish date was changed from 03/09/2019 to 31/12/2022.
09/09/2019: The recruitment start date was changed from 31/12/2022 to 03/09/2019.
01/07/2019: Ethics approval details added.
22/05/2019: Trial's existence confirmed by the NIHR.
