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Real-World Data on the Use of Sirolimus in Asian Children with Vascular Malformations

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Abstract

Objectives

The management of vascular malformations is complex and challenging. This study aimed to explore efficacy, plasma trough concentrations of sirolimus, post-withdrawal conditions, and adverse reactions of sirolimus in treating complex vascular malformations.

Methods

In our center, we analyzed vascular malformations treated with sirolimus (and corticosteroid) from August 2017 to June 2021. Meanwhile, we reviewed the medical records, the efficacy, side effects, and laboratory tests. Patients who had stopped taking sirolimus were followed up by telephone.

Results

A total of 25 patients with complicated vascular malformations in our center, including 7 females and 18 males aged 4 months to 15 years, were enrolled. In all, 19 patients (76.0%) responded to sirolimus, and the plasma concentration of sirolimus fluctuated between 0.97 and 27.15 ng/ml. In all, 24 patients (96.0%) were in follow-up. A total of 15 patients (62.5%) stopped taking sirolimus during follow-up, and 2 patients (13.3%) discontinued the sirolimus due to side effects. A total of 3 patients (20.0%) restarted sirolimus treatment.

Conclusion

Starting dose of 1.5–2 mg/m2 sirolimus is effective and safe in vascular malformation treatment. The best treatment regimen and discontinuation indications needed more investigation. Most should be done about targeted therapy to improve effectiveness and reduce side effects.

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Acknowledgements

The patients in this manuscript have given written informed consent to publication of their case details.

Author information

Authors and Affiliations

  1. Department of Dermatology, Beijing Children’s Hospital, National Center for Children’s Health, China, Capital Medical University, 56#, Nanlishi Road, Xi Cheng District, Beijing, 100045, China

    Lu Yu, Zigang Xu, Li Wei, Bin Zhang, Lei Qiu, Lin Ma & Li Li

  2. Department of Burns and Plastic Surgery, Beijing Jishuitan Hospital, Capital Medical University, Beijing, China

    Lu Yu

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  1. Lu Yu
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  2. Zigang Xu
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  3. Li Wei
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  4. Bin Zhang
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  5. Lei Qiu
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  6. Lin Ma
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  7. Li Li
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Contributions

LL conceptualized, designed the study, and reviewed and revised the manuscript. LY conceptualized and designed the study, drafted the initial manuscript, designed the data collection instruments, collected data, carried out the initial analyses, reviewed and revised the manuscript, and critically reviewed the manuscript for important intellectual content. ZX, LW, BZ, LQ, and LM collected data, contributed to analysis and interpretation of data, coordinated, supervised data collection, and critically reviewed it for important intellectual content. All authors contributed to the article and approved the submitted version.

Corresponding author

Correspondence to Li Li.

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Funding Sources

Beijing Municipal Natural Science Foundation (7232054). Chinese Association of Rehabilitation Medicine Scientific Research (KFKT-2022-003).

Conflict of Interest Statement

Lu Yu, Zigang Xu, Li Wei, Bin Zhang, Lei Qiu, Lin Ma, and Li Li declare no conflicts of interest that might be relevant to this work.

Statement of Ethics

This study protocol was reviewed and approved by the Medical Ethics Committee of Beijing Children’s Hospital, Capital Medical University. The ethical document approval number of our study is [2015]-Y-022-D.

Data Availability Statements

All data generated or analyzed during this study are included in this article. Further enquiries can be directed to the corresponding author.

Consent for Participation and Consent for Publication

Written informed consent was obtained from the individual(s), and/or minor(s)’ legal guardian/next of kin, for the publication of any potentially identifiable images or data included in this article.

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Not applicable.

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Cite this article

Yu, L., Xu, Z., Wei, L. et al. Real-World Data on the Use of Sirolimus in Asian Children with Vascular Malformations. Pediatr Drugs 26, 309–317 (2024). https://doi.org/10.1007/s40272-023-00605-7

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  • DOI: https://doi.org/10.1007/s40272-023-00605-7

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