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ICHIHARA Atsuhiro
Department School of Medicine(Tokyo Women's Medical University Hospital), School of Medicine Position Professor and Division head |
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| Article types | Original article |
| Language | English |
| Peer review | Peer reviewed |
| Title | Efficacy and safety of metyrosine in pheochromocytoma/paraganglioma: a multi-center trial in Japan. |
| Journal | Formal name：Endocrine journal Abbreviation：Endocr J ISSN code：1348454009188959 |
| Volume, Issue, Page | 65(3),pp.359-371 |
| Author and coauthor | 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 |
| Publication date | 2018/03 |
| Summary | 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 |