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Clinical Trial
. 2018 Mar 28;65(3):359-371.
doi: 10.1507/endocrj.EJ17-0276. Epub 2018 Jan 20.

Efficacy and Safety of Metyrosine in Pheochromocytoma/Paraganglioma: A Multi-Center Trial in Japan

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Clinical Trial

Efficacy and Safety of Metyrosine in Pheochromocytoma/Paraganglioma: A Multi-Center Trial in Japan

Mitsuhide Naruse et al. Endocr J. .
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Abstract

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.

Keywords: Catecholamines; Metyrosine; Multi-center clinical trial; Paraganglioma; Pheochromocytoma.

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