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Sirolimus for vascular anomalies in the first year of life: a systematic review

Journal of Perinatology (2024)Cite this article

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Abstract

Evidence on effectiveness and safety of sirolimus in congenital vascular anomalies in infancy is lacking. We aim to systematically review the efficacy and safety of sirolimus in treating congenital VA in infancy. We searched for and included all studies evaluating sirolimus for VA in the first year of life. The primary outcome was effectiveness. The secondary outcome was safety. We included 84 case series and reports (172 participants). Sirolimus decreased the size of the VA in >50% of participants, most of whom had minor transient side effects, and 27% had no adverse effects at all. When categorized by age (<1 month, 1–5 months and 6–12 months), the effectiveness was similar in all age groups. Available evidence suggests that sirolimus is effective and well tolerated. The effectiveness of sirolimus should be evaluated in a well-designed randomized controlled or observational studies.

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Fig. 1: PRISMA flow diagram for inclusion of studies in the review.
Fig. 2: Improvement in clinical status after sirolimus therapy in the first year of life.
Fig. 3: Safety of sirolimus therapy in the first year of life.

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Data availability

All data generated or analyzed during this study are included in this published article and its supplementary information files.

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Funding

MP is funded by NIH grants, R03HD098482 and R21HD091718, not related to this manuscript. The NIH example had no role in the design and conduct of the study.

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Author notes

  1. These authors contributed equally: Rebeca Cavazos, Monika S. Patil.

Authors and Affiliations

  1. Department of Pediatrics, Section of Neonatology, Baylor College of Medicine, Houston, TX, USA

    Rebeca Cavazos, Monika S. Patil, Sharada H. Gowda, Caraciolo J. Fernandes & Mohan Pammi

  2. Department of Hematology Oncology, Baylor College of Medicine, Houston, TX, USA

    Ionela Iacobas

  3. Department of Otolaryngology, Baylor College of Medicine, Houston, TX, USA

    Tara Rosenberg

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Contributions

MSP and RC collected data, analyzed the data, drafted the initial manuscript, and reviewed and revised the manuscript. SC reviewed the data, contributed to the writing of the manuscript, and revised the manuscript. II reviewed the data, contributed to the writing and revising of the manuscript, and critically reviewed the manuscript for important intellectual content. TR reviewed the data, revised the manuscript and critically reviewed the manuscript for important intellectual content. CF reviewed the data, edited the manuscript, and directed the design of figures and tables along with critically reviewing the manuscript for important intellectual content. MP conceptualized the design of the study, contributed to the writing of the manuscript, reviewed and revised the manuscript, and critically reviewed the manuscript for important intellectual content. All authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.

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Correspondence to Rebeca Cavazos.

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Cavazos, R., Patil, M.S., Gowda, S.H. et al. Sirolimus for vascular anomalies in the first year of life: a systematic review. J Perinatol (2024). https://doi.org/10.1038/s41372-024-01868-9

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