Michio Ozeki, Saori Endo, Shiho Yasue, Akifumi Nozawa, Ryuta Asada, Akiko M Saito, Hiroya Hashimoto, Takumi Fujimura, Yohei Yamada, Tatsuo Kuroda, Shigeru Ueno, Shoji Watanabe, Shunsuke Nosaka, Mikiko Miyasaka, Akihiro Umezawa, Kentaro Matsuoka, Takanobu Maekawa, Satoshi Hirakawa, Taizo Furukawa, Shigehisa Fumino, Tatsuro Tajiri, Junkichi Takemoto, Ryota Souzaki, Yoshiaki Kinoshita, Akihiro Fujino
INTRODUCTION: Intractable lymphatic anomalies (LAs) include cystic lymphatic malformation (LM; macrocystic, microcystic, or mixed), generalized lymphatic anomaly, and Gorham-Stout disease. LAs can present with severe symptoms and poor prognosis. Thus, prospective studies for treatments are warranted. We conducted a prospective clinical trial of sirolimus for intractable LAs. METHODS: This was an open-label, single-arm, multicenter, prospective trial involving five institutions in Japan...
2024: Frontiers in Medicine