Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome

Beijing Friendship Hospital40 enrolled

Overview

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 .

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Macrophage Activation Syndrome

Interventions

RuxolitinibDRUG

0.3mg/kg/d, iv.gtt, for at least 2 weeks

methylprednisoloneDRUG

2mg/kg, d1-d5, gradually reduced, for at least 2 weeks

Eligibility

Sex: ALLMin age: 1 YearMax age: 75 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Response rate

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%.

Time frame: Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy

Progression Free Survival

from date of inclusion to date of progression, relapse, or death from any cause

Time frame: 2 years

Adverse events

Adverse events including myelosuppression, infection, hemorrhage

Time frame: 2 years

Central Contacts

Yue Song

CONTACT

86-010-63139862 xueqifeng1992@163.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.