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Updated: Sep 22, 2025
Depletion and Reconstitution of Macrophages in Mice
Published on: August 1, 2012
Short-term, low-dose etoposide in refractory adult-onset Still's disease-associated macrophage activation syndrome
Ran Wang1, Ting Li1, Shuang Ye1
1Department of Rheumatology, School of Medicine, Renji Hospital, Shanghai Jiaotong University, Shanghai, 200001, China.
Introduction:
In this study, we modified the classical regimen of the hemophagocytic lymphohistiocytosis-04 protocol and evaluated the efficacy and safety of short-term, low-dose etoposide in patients with refractory macrophage activation syndrome (MAS) associated with adult-onset Still's disease (AOSD).
Methods:
A total of 17 patients with refractory AOSD-associated MAS were enrolled and received short-term, low-dose etoposide (100 mg twice a week for four times). Another 11 patients, who were not treated with etoposide, were included as historical controls. Patient information, such as clinical manifestations, laboratory results, treatments, and short-term prognosis, were recorded and analyzed.
Results:
In this case series, 88.24% of the patients with MAS who were treated with short-term, low-dose etoposide had a favorable response in 3 weeks, which was significantly higher (p = 0.017) than that in the patients with MAS who were treated without etoposide (45.45%). The 90-day survival rate after the onset of MAS was significantly higher (p = 0.0029) among the patients in the short-term etoposide group (16/17, 94.12%) than in the control group (5/11, 45.45%).
Conclusion:
The regimen of short-term (2 weeks), low-dose etoposide was highly effective in the treatment for patients with refractory AOSD-associated MAS with an acceptable safety profile. Key Points • There is no high level evidence to guide the management of refractory MAS-associated AOSD patients. • This study was the first to propose and confirm the efficacy and safety of short-term, low-dose etoposide in the treatment of refractory MAS-associated AOSD patients.
Insights
Short-term, low-dose etoposide effectively treated refractory macrophage activation syndrome (MAS) in adult-onset Still
Area of Science:
- Rheumatology
- Hematology
- Clinical Pharmacology
Background:
- Macrophage activation syndrome (MAS) is a severe complication of adult-onset Still's disease (AOSD).
- Refractory MAS in AOSD lacks established treatment guidelines.
- Modified hemophagocytic lymphohistiocytosis-04 protocol was used.
Purpose of the Study:
- To evaluate the efficacy and safety of short-term, low-dose etoposide for refractory MAS in AOSD.
- To assess the treatment response and survival rates compared to historical controls.
Main Methods:
- A case series of 17 patients with refractory AOSD-associated MAS treated with etoposide.
- 11 patients served as historical controls without etoposide treatment.
- Data collected included clinical, laboratory, treatment, and prognosis information.
Main Results:
- 88.24% of patients receiving etoposide showed a favorable response within 3 weeks.
- This response rate was significantly higher than the 45.45% in the control group (p=0.017).
- The 90-day survival rate was 94.12% in the etoposide group versus 45.45% in the control group (p=0.0029).
Conclusions:
- Short-term, low-dose etoposide (2 weeks) is highly effective for refractory MAS-associated AOSD.
- The treatment demonstrated an acceptable safety profile.
- This study provides novel evidence for treating refractory MAS-associated AOSD.
