Efficacy and safety of metyrosine in pheochromocytoma/paraganglioma: a multi-center trial in Japan



    • Naruse Mitsuhide
    • Clinical Research Institute for Endocrinology and Metabolic Diseases, National Hospital Organization Kyoto Medical Center, Kyoto 612-8555, Japan
    • Matsuda Tadashi
    • Department of Urology and Andrology, Kansai Medical University Hospital, Hirakata 573-1191, Japan
    • Imai Tsuneo
    • National Hospital Organization, Higashinagoya National Hospital, Nagoya 465-8620, Japan
    • Yamada Masanobu
    • Department of Medicine and Molecular Science, Gunma University Graduate School of Medicine, Maebashi 371-8511, Japan
    • Satoh Fumitoshi
    • Division of Nephrology, Endocrinology and Vascular Medicine, Tohoku University Hospital, Sendai 980-8574, Japan
    • Tanabe Akiyo
    • Department of Diabetes, Endocrinology and Metabolism, National Center for Global Health and Medicine, Tokyo 162-8655, Japan
    • Okamoto Takahiro
    • Department of Surgery II, Tokyo Women's Medical University, Tokyo 162-8666, Japan
    • Ichihara Atsuhiro
    • Department of Endocrinology and Hypertension, Tokyo Women's Medical University, Tokyo 162-8666, Japan
    • Tsuiki Mika
    • Department of Endocrinology and Metabolism, National Hospital Organization Kyoto Medical Center, Kyoto 612-8555, Japan
    • Katabami Takuyuki
    • Department of Metabolism and Endocrinology, St. Marianna University School of Medicine, Yokohama City Seibu Hospital, Yokohama 241-0811, Japan
    • Nomura Masatoshi
    • Department of Endocrine and Metabolic Diseases, Kyushu University Hospital, Fukuoka 812-8582, Japan
    • Tanaka Tomoaki
    • Department of Molecular Diagnosis, Chiba University Graduate School of Medicine, Chiba 260-8670, Japan


<p>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.</p>


  • Endocr J

    Endocr J 65(3), 359-371, 2018

    一般社団法人 日本内分泌学会


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