cGVHD
Conditions
Brief summary
The study plan aims to include patients who have been diagnosed with steroid-refractory chronic GVHD in the liver following allogeneic hematopoietic stem cell transplantation. After obtaining informed consent, the patients will receive a treatment regimen consisting of the Anti-CD25 rhMAb in combination with prednisone, cyclosporine, and ruxolitinib.The objective is to assess the effectiveness and safety of Anti-CD25 rhMAb in the treatment of severe chronic GVHD affecting the liver.
Interventions
DRUGanti-CD25 rhMAb
1 mg/kg/day administered IV day 1, 4, and 8, then weekly for 6 doses. For patients achieving partial remission, an extra dose of Anti-CD25 rhMAb can be given on days 39 and 49.
DRUGPrednisone
Maintain pre-screening dose
DRUGRuxolitinib
10mg, BID PO
DRUGCyclosporine
1.25mg/kg, BID PO/IV, target:150-250ng/ml
Sponsors
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Age 16 and 65 years 2. Received allogeneic hematopoietic stem cell transplantation 3. Developed chronic GVHD in the liver after transplantation 4. Ineffective prednisone treatment prior to screening 5. Received ≤4 lines of systemic therapy prior to screening 6. After informed consent, the patient agreed to receive anti-CD25 rhMAb treatment
Exclusion criteria
1. Elevation of bilirubin, ALT, or alkaline phosphatase due to reasons other than chronic GVHD 2. No prior treatment with prednisone 3. Overlap syndrome 4. Uncontrolled active infection 5. Organ failure 6. Early progression or recurrence of hematologic diseases 7. Allergy to anti-CD25 rhMAb 8. Received other interleukin-2 receptor monoclonal antibody treatment due to various reasons within one month after transplantation 9. Participated in other clinical studies within one month
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| overall response rate (ORR) | 56 days | ORR is defined as the percentage of complete response (CR) and partial response (PR). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| duration of response（DOR） | 1 year | DOR is defined as the duration calculated from the time of achieving PR or CR until the progression of GVHD, the addition of other systemic immunosuppressive therapy, or death. |
| patient-reported outcomes (PRO) | 1 year | the unabbreviated scale title is the Lee Chronic GVHD Symptom Scale.the minimum and maximum are 0 and 100, and higher scores mean a worse outcome. |
| disease-free survival (DFS) | 1 year | DFS is defined as the duration of survival after treatment in which the original hematologic disease is in a state of complete remission. |
| failure-free survival (FFS) | 1 year | Events that are considered as failures include the onset of new chronic GVHD, relapse, and death. |
| non-relapse mortality (NRM) | 1 year | NRM is defined as death due to reasons other than progression/relapse of hematologic disease. |
| overall survival （OS） | 1 year | OS is defined as the time from treatment until death from any cause or the last follow-up. |
| adverse drug reactions (ADR) | 1 year | The occurrence of various organ toxicities related to treatment that emerge following treatment. |
| Best Overall Response Rate (ORR) of Overall cGVHD | 1 year | CR + PR Rate of Overall cGVHD |
| Immune Reconstitution | 1 year | Humoral immune reconstitution (IgM, IgA, and IgG)；Cellular immune reconstitution parameters (CD3⁺, CD4⁺, CD8⁺, and CD19⁺ cell subsets) |
| Quality of Life Assessment | 1 year | SF-36 Questionnaire；FACT-BMT Questionnaire |
Outcome results
None listed