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Sirolimus can promote the disappearance of renal angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study

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Abstract

Background

Renal angiomyolipoma (RAML) is the most common kidney lesion in patients with tuberous sclerosis complex (TSC), affecting about 80% of patients. It is a benign tumor that grows over time, usually bilaterally, and can easily lead to kidney complications such as acute hemorrhage. Herein, we investigated the efficacy and safety of sirolimus in children with TSC-associated RAML and explored the factors affecting tumor disappearance under sirolimus treatment through subgroup analysis.

Methods

A prospective cohort study was conducted. Sirolimus was initiated at 1 mg/(m2 × day), and dose adjustments were made by a 2-week titration period to attain a trough blood concentration of 5–10 ng/mL. The disappearance of RAML in children after sirolimus treatment was observed, and Cox regression was used to screen the factors affecting tumor disappearance.

Results

One hundred and twenty-six patients who met the criteria were analyzed. After 3 months, 6 months, 12 months, and 24 months of follow-up, tumors disappeared in 18 (14.3%), 30 (23.8%), 39 (31.0%), and 42 (33.3%) children, respectively. Tumors disappeared in 50 (39.7%) children by the last visit of each individual, and 30 (60%) of them occurred within 6 months. The multivariate Cox regression analysis showed that patients with a smaller maximum tumor diameter at baseline had a higher tumor disappearance rate. Thirty-six (29%) patients had stomatitis during the entire treatment period, and no serious adverse reactions were observed.

Conclusions

Sirolimus could promote the disappearance of TSC-related RAML. The disappearance rate was correlated with the maximum diameter at baseline, and the smaller the tumor was, the higher the disappearance rate. It is well tolerated in the treatment of RAML associated with TSC.

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Data availability

The data of this study are available on reasonable request to the corresponding author.

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Acknowledgements

We thank all enrolled patients and their parents or guardians.

Funding

This study was funded by the Capital's Funds for Health Improvement and Research (No. 2022-1-5081) and National Key Research and Development Program of China (No. 2016YFC1000707).

Author information

Authors and Affiliations

  1. Medical School of Chinese PLA, Beijing 100853, China

    Shuo Dun & Lu-Peng Qiu

  2. Department of Pediatrics, The First Medical Center, Chinese PLA General Hospital, Beijing 100853, China

    Shuo Dun, Yang-Yang Wang, Lin Wan, Qiu-Hong Wang, Qian Lu, Xiao-Yan Yang, Qi Zhang, Hui-Min Chen & Li-Ping Zou

  3. Center of Epilepsy, Beijing Institute for Brain Disorders, Beijing 100069, China

    Li-Ping Zou

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  1. Shuo Dun
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Contributions

DS and WYY conceived and designed the study, collected data, analyzed the data and drafted the initial manuscript, and revised the manuscript. WL conceived and designed the study, analyzed the data and drafted the initial manuscript, and revised the manuscript. WQH, LQ, YXY, ZQ, and CHM collected data and reviewed the manuscript. QLP analyzed the data and drafted the initial manuscript, and revised the manuscript. ZLP conceived and designed the study, and reviewed the manuscript. All the authors approved the final manuscript and agreed to be accountable for all aspects of the work.

Corresponding author

Correspondence to Li-Ping Zou.

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Ethical approval

This study has been performed in line with the principles of the Declaration of Helsinki. Ethical approval was obtained from the Ethics Committee of the PLA General Hospital (No. S2013-028-01). Written informed consent was obtained from legal guardians of all enrolled participants included in the study.

Conflict of interest

The authors declare no competing interests. No financial or non-financial benefits have been received or will be received from any party related directly or indirectly to the subject of this article.

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Dun, S., Wang, YY., Wan, L. et al. Sirolimus can promote the disappearance of renal angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study. World J Pediatr (2023). https://doi.org/10.1007/s12519-023-00751-5

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