Macrophage Activation Syndrome
Conditions
Brief summary
The present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy .
Interventions
DRUGRuxolitinib
0.3mg/kg/d, iv.gtt, for at least 2 weeks
DRUGmethylprednisolone
2mg/kg, d1-d5, gradually reduced, for at least 2 weeks
Sponsors
Beijing Friendship Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS). 2. No HLH induction therapy was performed. 3. The expected survival time is more than 1 month. 4. Serum creatinine ≤ 1.5 times normal；Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；HBV copies less than 1E+03 copies/ml. 5. Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value 6. The left ventricular ejection fraction (LVEF) was normal. 7. No uncontrollable infection. 8. Contraception for both male or female. 9. Informed consent obtained.
Exclusion criteria
1. Pregnancy or lactating Women； 2. Allergic to ruxolitinib； 3. Active bleeding of the internal organs； 4. uncontrollable infection； 5. Serious mental illness; 6. Non-melanoma skin cancer history; 7. Patients unable to comply during the trial and/or follow-up phase; 8. Participate in other clinical research at the same time.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Response rate | Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy | A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of\<0.5 ×109/L, a response was defined as an increase by at least 100% to\>0.5× 109/L; for patients with a neutrophil count of 0.5 to 2.0 × 109/L, an increase by at least 100% to \>2.0 × 109/L was considered a response; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%. |
| Progression Free Survival | 2 years | from date of inclusion to date of progression, relapse, or death from any cause |
| Adverse events | 2 years | Adverse events including myelosuppression, infection, hemorrhage |
Contacts
Primary ContactYue Song
Outcome results
None listed