イチハラ　アツヒロ   ICHIHARA Atsuhiro   市原　淳弘    所属   医学部 医学科（東京女子医科大学病院）    職種   教授・基幹分野長
論文種別	原著
言語種別	英語
査読の有無	査読あり
表題	Efficacy and safety of metyrosine in pheochromocytoma/paraganglioma: a multi-center trial in Japan.
掲載誌名	正式名：Endocrine journal 略　 称：Endocr J ISSNコード：1348454009188959
巻・号・頁	65(3),pp.359-371
著者・共著者	NARUSE Mitsuhide†*, SATOH Fumitoshi, TANABE Akiyo, OKAMOTO Takahiro, ICHIHARA Atsuhiro, TSUIKI Mika, KATABAMI Takuyuki, NOMURA Masatoshi, TANAKA Tomoaki, MATSUDA Tadashi, IMAI Tsuneo, YAMADA Masanobu, HARADA Tomohiro, KAWATA Nobuyuki, TAKEKOSHI Kazuhiro
発行年月	2018/03
概要	To assess the efficacy, safety, and pharmacokinetics of metyrosine (an inhibitor of catecholamine synthesis) in patients with pheochromocytoma/paraganglioma (PPGL), we conducted a prospective, multi-center, open-label study at 11 sites in Japan. We recruited PPGL patients aged ≥12 years requiring preoperative or chronic treatment, receiving α-blocker treatment, having baseline urinary metanephrine (uMN) or normetanephrine (uNMN) levels ≥3 times the upper limit of normal values, and having symptoms associated with excess catecholamine. Metyrosine treatment was started at 500 mg/day and modified according to dose-adjustment criteria up to 4,000 mg/day. The main outcome measure was the proportion of patients who achieved at least 50% reduction in uMN or uNMN levels from baseline. Sixteen patients (11 males/5 females) aged 12-86 years participated. After 12 weeks of treatment and at the last evaluation of efficacy, the primary endpoint was achieved in 31.3% of all patients, including 66.7% of those under preoperative treatment and 23.1% of those under chronic treatment. Sedation, anemia, and death were reported in 1 patient each as serious adverse drug reactions during the 24-week treatment. Metyrosine was shown to be tolerated and to relieve symptoms by reducing excess catecholamine in PPGL patients under both preoperative and chronic treatment.
DOI	10.1507/endocrj.EJ17-0276
PMID	29353821