期刊
JOURNAL OF NEUROLOGY
卷 265, 期 12, 页码 2861-2874出版社
SPRINGER HEIDELBERG
DOI: 10.1007/s00415-018-9057-7
关键词
l-Arginine; Mitochondrial disease; MELAS; Stroke-like episodes; Ictus
资金
- Ministry of Health, Labour and Welfare of Japan [H14-Shouni-006]
- Center for Clinical Trials, the Japan Medical Association Center [CCT-B-1803, CCT-C-2001]
ObjectiveTo examine the efficacy and safety of the therapeutic regimen using oral and intravenous l-arginine for pediatric and adult patients with mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes (MELAS).MethodsIn the presence and absence of an ictus of stroke-like episodes within 6h prior to efficacy assessment, we correspondingly conducted the systematic administration of oral and intravenous l-arginine to 15 and 10 patients with MELAS in two, 2-year, prospective, multicenter clinical trials at 10 medical institutions in Japan. Subsequently, patients were followed up for 7years. The primary endpoint in the clinical trial of oral l-arginine was the MELAS scale, while that for intravenous l-arginine was the improvement rates of headache and nausea/vomiting at 2h after completion of the initial intravenous administration. The relationships between the ictuses of stroke-like episodes and plasma arginine concentrations were examined.ResultsOral l-arginine extended the interictal phase (p=0.0625) and decreased the incidence and severity of ictuses. Intravenous l-arginine improved the rates of four major symptomsheadache, nausea/vomiting, impaired consciousness, and visual disturbance. The maximal plasma arginine concentration was 167mol/L when an ictus developed. Neither death nor bedriddenness occurred during the 2-year clinical trials, and the latter did not develop during the 7-year follow-up despite the progressively neurodegenerative and eventually life-threatening nature of MELAS. No treatment-related adverse events occurred, and the formulations of l-arginine were well tolerated.ConclusionsThe systematic administration of oral and intravenous l-arginine may be therapeutically beneficial and clinically useful for patients with MELAS.
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