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This trial is a randomized (1:1) phase III open label study of frontline mini-MTX plus methylprednisolone 2mg/kg/day compared to methylprednisolone 2mg/kg in allogeneic stem cell transplant recipients with grade 2-4 aGVHD.
Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT. Corticosteroid is the standard first-line therapy for aGVHD. However, the response rate of corticosteroid was approximate 50%, and the clinical outcomes of patients with corticosteroid refractory GVHD were poor. Thus far, no combination therapy had been prove to be superior to corticosteroid alone as initial therapy for aGVHD. The study hypothesis: MTX combined corticosteroid treatment could help to control aGVHD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MTX and corticosteroid | Experimental | Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). MTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR. |
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| Corticosteroid | Active Comparator | Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MTX | Drug | MTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate (ORR) for aGVHD treatment after treatment | Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response | 14 days |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate (ORR) for aGVHD treatment at 28 days after treatment | Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response | 28 days |
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Inclusion Criteria:
Patients who are fully informed and sign informed consent by themselves or their guardians;
Patients receiving first allogeneic hematopoietic stem cell transplantation;
Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
KPS>60, Estimated survival >3 months;
No serious organ damage:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yu Wang, M.D. | Contact | 86-13552647384 | ywyw3172@sina.com | |
| Xiao-Jun Huang, M.D. | Contact |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking University Institute of Hematology, | Recruiting | Beijing | Beijing Municipality | 100044 | China |
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| ID | Term |
|---|---|
| D000305 | Adrenal Cortex Hormones |
| ID | Term |
|---|---|
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
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| Corticosteroid | Drug | Corticosteroid Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). |
|
| Overall response rate (ORR) for aGVHD treatment at 42 days after treatment |
Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response |
| 42 days |
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | Data collection including questionnaires at individual and group visits and physician interviews at individual visits will be used to assess participants for treatment-related adverse events. | 42 days |
| cGVHD | The cumulative incidence of chronic GVHD | 1 years |
| Infection and poor graft function | The cumulative incidence of severe infection and poor graft function | 1 year |
| Relapse | The cumulative incidence of relapse | 1 year |
| Non-relapse mortality | The cumulative incidence of non-relapse mortality | 1 year |
| Overall survival | The cumulative incidence of overall survival | 1 year |
| Disease free survival | The cumulative incidence of disease free survival | 1 year |
| Nanfang Hospital, Nanfang Medical University | Recruiting | Guangzhou | Guangdong | 510515 | China |
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