Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.

Veroniek E M Harbers, Lilly G J M Zwerink, Gerard A Rongen, Willemijn M Klein, Carine J M van der Vleuten, Ingrid M P van Rijnsoever, Lynda Gerdsen-Drury, Uta E Flucke, Bas H Verhoeven, Peter C J de Laat, Chantal M A M van der Horst, Leo J Schultze Kool, D Maroeska W M Te Loo

May 2023 Clin Transl Sci

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4 Posts

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Posts referencing the article

Sirolimus Treatment for Vascular Malformations: Pediatric and Adult Insights

Sirolimus Treatment for Vascular Malformations: Pediatric and Adult Insights

Low target levels of sirolimus effectively treat vascular malformations, improving symptoms in both children and adults. Adults experience more pain and side effects, but children respond faster. Long-term treatment may be necessary due to symptom recurrence.

Medicine RTCL TV

September 4, 2023

6 views

  • GoPI3Ks (Genetic Overgrowth PI3K Support)
    @GoPI3Ks (Twitter)

    This is from 2023 but may still be an interesting read for some: https://t.co/xHE54oB13p https://t.co/kKRq7WtVqj
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    March 28, 2025

  • V. Harbers
    @VHarbers1 (Twitter)

    RT @HecovanNijmegen: Good news! Our article received enough citations to be a Top Cited Article. Read it here https://t.co/M2kLElo8TC #Top…
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    March 21, 2025

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  • GoPI3Ks (Genetic Overgrowth PI3K Support)
    @GoPI3Ks (Twitter)

    RT @HecovanNijmegen: Good news! Our article received enough citations to be a Top Cited Article. Read it here https://t.co/M2kLElo8TC #Top…
    view full post

    March 20, 2025

    2

  • C. van der Vleuten
    @HecovanNijmegen (Twitter)

    Good news! Our article received enough citations to be a Top Cited Article. Read it here https://t.co/M2kLElo8TC #TopCitedArticle @wileyinresearch
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    March 20, 2025

    4

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Abstract Synopsis

  • The study found that low target levels of sirolimus (4-10 ng/mL) are effective in treating children and adults with vascular malformations, with most patients experiencing symptom improvement, though symptoms often recur after stopping treatment.
  • Adults tend to experience more pain and higher rates of side effects than children, but both groups respond similarly to the medication, with children responding faster and showing higher response rates.
  • The results suggest that early initiation of sirolimus in younger patients could be beneficial, and prolonged or lifelong treatment may be necessary due to symptom recurrence upon stopping the medication.