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| Autori principali: | , , , , , , , , , , , , , , , , , , , , , , , |
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| Natura: | Artículo Open Access |
| Pubblicazione: |
Wiley
2025
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| Soggetti: | |
| Accesso online: | https://onlinelibrary.wiley.com/doi/10.1111/ped.70002 |
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- Sirolimus treatment for intractable vascular anomalies ( SIVA ): An open‐label, single‐arm, multicenter, prospective trial Michio Ozeki Saori Endo Shiho Yasue Ryuta Asada Akiko M. Saito Hiroya Hashimoto Shigeru Ueno Shoji Watanabe Motoi Kato Kyoichi Deie Shunsuke Nosaka Mikiko Miyasaka Akihiro Umezawa Kentaro Matsuoka Mototoshi Kato Tatsuo Kuroda Takanobu Maekawa Satoshi Hirakawa Taizo Furukawa Shigehisa Fumino Tatsuro Tajiri Junkichi Takemoto Naonori Kawakubo Akihiro Fujino Pediatrics International Abstract Background Intractable vascular anomalies (VAs), including vascular tumors and venous, lymphatic, and mixed malformations, often have severe symptoms and a poor prognosis, highlighting the need for new treatments. We conducted a prospective trial of sirolimus (tablet and granule forms) for the treatment of VAs. Methods In this open‐label, single‐arm, multicenter trial across four Japanese institutions, patients with VAs received oral sirolimus daily, targeting a trough concentration of 5–15 ng/mL. We evaluated response rates (radiological volume changes in lesions), skin lesions, performance status, respiratory function, visceral symptoms (bleeding, pain), laboratory data, quality of life, and safety at 12, 24, and 52 weeks. Results Thirteen patients with VAs were treated with sirolimus. Seven patients (53.8%; 95% confidence interval: 25.1%–80.8%) showed a partial radiological response at 24 weeks, with no complete responses, and 61.5% had a partial response by 12 weeks, with little subsequent change in patients who had stable disease thereafter. Improvements in skin lesions, blood coagulation, and activities of daily living were noted. Common adverse events included stomatitis, dermatitis, diarrhea, and fever. Conclusions Sirolimus may reduce VA tissue volume and potentially improve symptoms and activities of daily living in patients with VAs. 10.1111/ped.70002 http://creativecommons.org/licenses/by/4.0/