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Short-term, low-dose etoposide in refractory adult-onset Still’s disease-associated macrophage activation syndrome

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Abstract

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.

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Funding

Shanghai Municipal Science and Technology Fund 21ZR1438800.

Shanghai Talents Development fund 2019092.

Shanghai Pujiang Young Rheumatologists Training Program SPROG201907.

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Author notes

  1. Ran Wang and Ting Li contributed equally to this work.

Authors and Affiliations

  1. Department of Rheumatology, School of Medicine, Renji Hospital, Shanghai Jiaotong University, Shanghai, 200001, China

    Ran Wang, Ting Li, Shuang Ye, Liangjin Lv, Sheng Chen, Xiaodong Wang, Chun-de Bao & Qiong Fu

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  1. Ran Wang
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Correspondence to Chun-de Bao or Qiong Fu.

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Wang, R., Li, T., Ye, S. et al. Short-term, low-dose etoposide in refractory adult-onset Still’s disease-associated macrophage activation syndrome. Clin Rheumatol 41, 2817–2823 (2022). https://doi.org/10.1007/s10067-022-06184-1

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  • DOI: https://doi.org/10.1007/s10067-022-06184-1

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