Mesenchymal stromal cells for the treatment of acute graft versus host disease
| ISRCTN | ISRCTN18091201 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN18091201 |
| Secondary identifying numbers | TPSL-LTU-2015 |
- Submission date
- 12/07/2017
- Registration date
- 18/08/2017
- Last edited
- 06/06/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Injury, Occupational Diseases, Poisoning
Plain English summary of protocol
Background and study aims
Acute graft-versus-host disease (aGVHD) is a possible complication of a bone marrow stem cell transplant from another person (allogeneic hematopoietic stem cell transplantation). It is caused by the donated immune cells reacting against the host tissues. It is potentially lethal and around half of stem cell recipients develop it. Acute GVHD typically occurs in the first 100 days after transplant and affects three organs: skin, liver and gastrointestinal (digestive) tract. The first treatment is to use glucocorticoid drugs but half of the patients do not respond to treatment (steroid refractory). The prognosis of patients who develop acute GVHD and do not respond to treatment is dismal. Numerous strategies to treat steroid-refractory aGVHD with a second treatment have been tested, but none have shown an effect. Therefore, there is no established second-line treatment for steroid-refractory acute GvHD. Mesenchymal stromal cells (MSCs) can decrease the activity of most cells of the immune system. The aim of this study is to assess the effectiveness of MSC treatment for steroid-refractory aGVHD.
Who can participate?
Patients aged 18 and older with steroid-refractory aGVHD after hematopoietic stem cell transplantation
What does the study involve?
All participants are treated with mesenchymal stromal cells. Blood samples are taken during treatment and at follow ups. Patients are followed up to assess their response to treatment, relapse and survival rates.
What are the possible benefits and risks of participating?
There are no benefits or risks for participants, but the results could be used to improve the treatment of patients with aGVHD in the future.
Where is the study run from?
Vilnius University Hospital Santaros Klinikos (Lithuania)
When is the study starting and how long is it expected to run for?
October 2013 to December 2023
Who is funding the study?
Hematology and Oncology Research Association of Lithuania
Who is the main contact?
Mr Adomas Bukauskas
Contact information
Scientific
Seliu 29-41
Vilnius
LT-08125
Lithuania
 ORCID ID |
0000-0001-5857-4638 |
|---|
Study information
| Study design | Observational prospective non-interventional single-center study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Longitudinal study |
| 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 | Mesenchymal stromal cells in patients with steroid refractory acute graft versus host disease after allogeneic hematopoietic stem cell transplantation or donor lymphocyte infusion |
| Study objectives | Mesenchymal stromal cells have been shown to have immunosuppressive effects. Through various mechanisms mesenchymal stromal cells exert an effect on most cells of the immune system and inhibit proliferation, activation, and cytokine release. Donor T cell activation and inflammatory cytokines play a major role in acute graft versus host disease (aGVHD) pathogenesis. The trialists hypothesize that by immunomodulatory properties mesenchymal stromal cells will be effective in controlling steroid refractory aGVHD and will prolong overall survival. |
| Ethics approval(s) | Vilnius regional Bioethics Committee, 03/11/2015, ref: 1582000-15-814-326 |
| Health condition(s) or problem(s) studied | Steroid refractory acute graft versus host disease after hematopoietic stem cell transplantation or donor lymphocyte infusion |
| Intervention | Patients with steroid refractory acute graft versus host disease were treated with mesenchymal stromal cells as compassionate use medicinal product (standard of care). Doses have not been predetermined in the protocol and follow standard practice. Additionally, blood samples were taken during treatment phase and at follow ups. |
| Intervention type | Biological/Vaccine |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Mesenchymal stromal cells |
| Primary outcome measure | Overall response rate (ORR) to mesenchymal stromal cells, defined as complete response (CR) or partial response (PR) of acute graft versus host disease, measured at D7, D14, D21, D28, D35, D42, D49, D56, 3 months |
| Secondary outcome measures | Measured at D7, D14, D21, D28, D35, D42, D49, D56, 3 months, 6 months, 9 months, 12 months, 18 months, 24 months and each year thereafter: 1. Overall survival (OS), defined as time from the start of treatment until death from any cause 2. Event free survival, defined as time from the start of treatment until the following events (whichever occurs first): death, hematologic malignancy relapse, no PR/CR by 3 months since the study entry, aGVHD relapse after PR/CR requiring next line treatment, progression to extensive chronic GVHD |
| Overall study start date | 09/10/2013 |
| Completion date | 31/12/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 45 |
| Total final enrolment | 93 |
| Key inclusion criteria | 1. Steroid refractory acute graft versus host disease after hematopoietic stem cell transplantation 2. 18 years old and older 3. Signed informed consent form |
| Key exclusion criteria | Does not meet inclusion criteria |
| Date of first enrolment | 09/10/2013 |
| Date of final enrolment | 10/04/2017 |
Locations
Countries of recruitment
- Lithuania
Study participating centre
Vilnius
LT-08406
Lithuania
Sponsor information
Research organisation
Savicikio 4
Vilnius
01108
Lithuania
Funders
Funder type
Research organisation
No information available
Results and Publications
| Intention to publish date | 01/06/2025 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Planned publication in a peer-reviewed journal |
| IPD sharing plan | The data sharing plans for the current study are unknown and will be made available at a later date. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | 13/05/2025 | 06/06/2025 | Yes | No |
Editorial Notes
06/06/2025: Publication reference added.
28/02/2025: The intention to publish date was changed from 01/03/2025 to 01/06/2025. Total final enrolment added.
14/11/2024: The intention to publish date was changed from 15/11/2024 to 01/03/2025.
21/08/2024: The intention to publish date was changed from 01/07/2024 to 15/11/2024.
08/03/2024: The intention to publish date was changed from 01/03/2024 to 01/07/2024.
28/11/2023: The following changes were made to the trial record:
1. The overall end date was changed from 01/11/2023 to 31/12/2023.
2. The intention to publish date was changed from 31/12/2023 to 01/03/2024.
3. The plain English summary was updated to reflect these changes.
13/07/2023: The following changes were made to the trial record:
1. The overall end date was changed from 01/06/2023 to 01/11/2023.
2. The intention to publish date was changed from 01/07/2023 to 31/12/2023.
3. The plain English summary was updated to reflect these changes.
01/12/2022: The following changes were made to the trial record:
1. The overall trial end date was changed from 30/11/2022 to 01/06/2023.
2. The intention to publish date was changed from 31/12/2018 to 01/07/2023.
