Prevention of Severe Acute Graft-versus-host Disease in Adult Patients Using a daGOAT Model
Study Details
Study Description
Brief Summary
To evaluate the efficacy and safety of ruxolitinib for prophylactic therapy of adult patients who are predicted to have a high risk for developing severe acute graftversus-host disease (aGVHD) by the dynamic aGVHD Onset Anticipation Tianjin (daGOAT) model.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Detailed Description
This study aims to prospectively evaluate the use of the daGOAT model in real-world clinical settings at the Institute of Hematology, Chinese Academy of Medical Sciences (IHCAMS).
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: The group of daGOAT model prevention Model-predicted high-risk patients: ruxolitinib 5mg bid po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (<0.1×10^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted moderate-risk patients: ruxolitinib 2.5mg bid p po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (<0.1×10^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted low risk: regular aGVHD prophylactic regimens (e.g., calcineurin inhibitors) without using ruxolitinib. |
Drug: Ruxolitinib
Model-predicted high-risk patients: ruxolitinib 5mg bid po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (<0.1×10^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery.
Model-predicted moderate-risk patients: ruxolitinib 2.5mg bid p po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (<0.1×10^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery.
Model-predicted low risk: regular aGVHD prophylactic regimens (e.g., calcineurin inhibitors) without using ruxolitinib.
|
Outcome Measures
Primary Outcome Measures
- Severe aGVHD during 100 days after transplantation according to the MAGIC criteria [100 days after transplantation]
Incidence of severe aGVHD after transplantation within 100 days. The medical records for each case wil be reviewed by two or three physicians to confirm the aGVHD diagnosis and grading (according to the MAGIC criteria)
Secondary Outcome Measures
- aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria [100 days after transplantation]
Incidence of aGVHD (any grade) in various target organs. The medical records for each case wil be reviewed by two or three physicians to confirm the aGVHD diagnosis and grading (according to the MAGIC criteria)
- Overall survival during 1 year after transplantation [Days 14, 28, 42, 60, 90, 180, 270, and 360 after transplantation]
Patients will be followed up at days 14, 28, 42, 60, 90, 180, 270, and 360 after transplantation; data on survival will be collected.
- Relapse-free survival rate and relapse rate during 1 year after transplantation [Days 14, 28, 42, 60, 90, 180, 270, and 360 after transplantation]
Patients will be followed up at days 14, 28, 42, 60, 90, 180, 270, and 360 after transplantation; data on relapse will be collected.
- Incidence of infections during 1 year after transplantation [1 year after transplantation]
Infection was defined as meeting one of the following criteria: culture-confirmed presence of bacteria or fungi in a sample collected from a sterile site; polymerase chain reaction-confirmed viremia at ≥ 5000 copies/ml for the cytomegalovirus or ≥ 10000 copies/ml for the Epstein-Barr virus; or body temperature ≥ 38 ℃ with culture-confirmed presence of pathogens from a non-sterile site.
- Safety of treatment during 1 year after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0 [1 year after transplantation]
Data on adverse events of treatment will be collected.
- Total cost of treatment during 1 year after transplantation [1 year after transplantation]
Data on total cost of treatment will be collected from the medical records.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Patients must be > 16 years of age;
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Patients receiving human leukocyte antigen mismatched and non-cord blood allogeneic hematopoietic stem cell transplantation;
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Patients who can take oral medication;
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Patients have to sign an informed consent form before the start of the research procedure.
Exclusion Criteria:
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Tandem transplantation or multiple transplantations;
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Patients who are allergic to or cannot tolerate ruxolitinib ;
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Mental or other medical conditions that make the patients unable to comply with the research treatment and monitoring requirements ;
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Patients who are pregnant or cannot take appropriate contraceptive measures during treatment;
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Patients who are ineligible for the study due to other factors, or will bear great risk if participating in the study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Institute of Hematology & Blood Diseases Hospital | Tianjin | Tianjin | China | 300020 |
Sponsors and Collaborators
- Institute of Hematology & Blood Diseases Hospital
Investigators
None specified.Study Documents (Full-Text)
More Information
Publications
None provided.- daGOAT-adult-001