Chronic graft-versus-host-disease
Conditions
Interventions
Experimental group:Sirolimus + Prednisone (or equivalent)
Control group:Prednisone (or equivalent)
Sponsors
Medical Center of Hematology, Xinqiao Hospital of Army Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 70 Years
Inclusion criteria
Inclusion criteria: 1. Age = 18, male or female; 2. 100 days after allogeneic transplantation, according to the 2014 NIH standard, newly diagnosed patients with moderate and severe chronic GVHD or overlapping syndrome (typical chronic plus acute graft-versus-host disease) (new diagnosis is defined as: 1) have not received any treatment before; 2) <14 days of prednisone treatment (or equivalent) before enrollment/randomization study treatment; 3) Previous treatment, but when the effective concentration of calcineurin inhibitor (CNI) is less than or equal to 16 weeks, the effect is not good); 3. The basic disease is stable without progression and recurrence; 4. Patients or guardians who are willing and able to provide informed consent; 5. Women with fertility potential are willing to use contraceptives; 6. Willing to comply with the research plan and research requirements.
Exclusion criteria
Exclusion criteria: 1. Progress or recurrence of basic diseases; 2. Patients with persistent acute GVHD or recurrent acute GVHD; 3. Can not tolerate the treatment of prednisone with a dose>0.5mg/kg/d; 4. Have received sirolimus treatment (to prevent or treat acute GVHD); Have received additional prednisone at a dose of at least 0.25 mg/kg/d; 5. Treat chronic graft-versus-host disease for more than 16 weeks; 6. Invasive fungal or viral infection, and antifungal or antiviral treatment is ineffective; 7. Renal insufficiency; 8. Inability to tolerate oral drugs; 9. Pregnancy (serum ß- HCG positive) or breast feeding; 10. Complicated with other malignant tumors and under treatment; 11. Hyperlipidemia; 12. Sirolimus allergy; 13. Serious cardiovascular disease (uncontrolled arrhythmia, congestive heart failure, NYHA III or IV or symptomatic ischemic heart disease); 14. Patients with neutrophils<1 * 10 ^ 9/L and/or platelets<20 * 10 ^ 9/L; 15. Infusion of donor lymphocytes or Car-T cells within the last 30 days; 16. Gastrointestinal ulcer, severe ulcerative colitis; 17. Severe osteoporosis; 18. Glaucoma (closed or open angle), corneal ulcer or corneal injury; 19. Severe diabetes and hypertension are poorly controlled.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall response rate after 6 months of treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| 1-year overall survival;1-year NRM;Proportion of starting secondary systemic immunosuppressive therapy;Incidence of infection;Lymphocyte subsets;Proportion of sirolimus discontinued due to toxicity or intolerance;Adverse drug reactions; | — |
Countries
China
Contacts
Public ContactZhang Xi
Medical Center of Hematology, Xinqiao Hospital of Army Medical University
Outcome results
None listed