Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
Study Details
Study Description
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 .
Condition or Disease | Intervention/Treatment | Phase |
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Phase 3 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: Ruxolitinib+methylprednisolone Ruxolitinib and methylprednisolone administered as the first-line therapy |
Drug: Ruxolitinib
0.3mg/kg/d, iv.gtt, for at least 2 weeks
Drug: methylprednisolone
2mg/kg, d1-d5, gradually reduced, for at least 2 weeks
|
Outcome Measures
Primary Outcome Measures
- 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
Eligibility Criteria
Criteria
Inclusion Criteria:
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Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
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No HLH induction therapy was performed.
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The expected survival time is more than 1 month.
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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.
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Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
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The left ventricular ejection fraction (LVEF) was normal.
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No uncontrollable infection.
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Contraception for both male or female.
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Informed consent obtained.
Exclusion Criteria:
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Pregnancy or lactating Women;
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Allergic to ruxolitinib;
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Active bleeding of the internal organs;
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uncontrollable infection;
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Serious mental illness;
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Non-melanoma skin cancer history;
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Patients unable to comply during the trial and/or follow-up phase;
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Participate in other clinical research at the same time.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Beijing Friendship Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- Ruxolitinib-steroids-MAS