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Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Abatacept and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) | Experimental | Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus | Drug | Orally (concentration 5-10ng/ml) from -1 to +100d post haplo-HSCT |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Grade 2-4 aGVHD within 100 days post transplantation | Participants will be followed for an expected average of 100 days post transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of chronic GVHD (cGVHD) within 1 year post transplantation | Participants will be followed for an expected average of 1 year | |
| Incidence of thrombotic microangiopathy within 1 year post transplantation | Participants will be followed for an expected average of 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative incidence of relapse | Participants will be followed for an expected average of 1 year | |
| Transplant-related mortality | Participants will be followed for an expected average of 1 year | |
Inclusion criteria:
Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
Contraindication or intolerance to CNI
Receiving haplo-HSCT for the first time
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yuqian Sun, M.D. | Contact | +86-10-88324577 | sunyuqian83@hotmail.com | |
| Yuqian Sun | Contact | +86-10-88324577 | sunyuqian83@hotmail.com |
| Name | Affiliation | Role |
|---|---|---|
| Xiao-Jun Huang | Institute of Hematology, Peking University | Principal Investigator |
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| Abatacept | Drug | 5mg/kg subcutaneous, -1/+5/+14/+28/+42/+56d post haplo-HSCT |
|
| MMF | Drug | 0.5g bid orally, from -3d to +60d post haplo-HSCT |
|
| ATG | Drug | 2.5 mg/kg, from -5d to -2d |
|
| Overall survival |
| Participants will be followed for an expected average of 1 year |
| Incidence of cytomegalovirus (CMV) and Epstein-Barr virus (EBV) | Participants will be followed for an expected average of 1 year |
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| D000741 | Anemia, Aplastic |
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000740 | Anemia |
| D000080983 | Bone Marrow Failure Disorders |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| D000069594 | Abatacept |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
| D018796 | Immunoconjugates |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D012712 | Serum Globulins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D005916 | Globulins |
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