Sirolimus treatment for intractable vascular anomalies (SIVA): An open-label, single-arm, multicenter, prospective trial.

IF 1 4区医学 Q3 PEDIATRICS

Pediatrics International Pub Date : 2025-01-01 DOI:10.1111/ped.70002

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

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引用次数: 0

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.

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来源期刊

Pediatrics International 医学-小儿科

CiteScore

2.00

自引率

7.10%

发文量

519

审稿时长

12 months

期刊介绍： Publishing articles of scientific excellence in pediatrics and child health delivery, Pediatrics International aims to encourage those involved in the research, practice and delivery of child health to share their experiences, ideas and achievements. Formerly Acta Paediatrica Japonica, the change in name in 1999 to Pediatrics International, reflects the Journal''s international status both in readership and contributions (approximately 45% of articles published are from non-Japanese authors). The Editors continue their strong commitment to the sharing of scientific information for the benefit of children everywhere. Pediatrics International opens the door to all authors throughout the world. Manuscripts are judged by two experts solely upon the basis of their contribution of original data, original ideas and their presentation.