Hoshino Kyoko, Hayashi Masaharu, Ishizaki Asayo, Kimura Kazue, Kubota Masaya, Nezu Atsuo, Yasuhara Akihiro
Segawa Memorial Neurological Clinic for Children, Tokyo, Japan.
The Very-Low-Dose Levodopa Therapy Research Group http://www.segawa-research.com/ldopa-therapy/index.html, Tokyo, Japan.
Front Pediatr. 2021 Mar 4;9:569594. doi: 10.3389/fped.2021.569594. eCollection 2021.
Post-synaptic dopamine receptor supersensitivity (DARSS) has been extensively researched by Dr. Masaya Segawa, who has investigated the efficacy of very-low-dose levodopa therapy (VLDT; 0.5-1 mg/kg/day). Considerable Japanese research supports the possibility that VLDT could be used to treat pediatric neurological disorders. We conducted an on-line survey in 2014 to collect real-world data on the use of VLDT to treat DARSS. A two-step survey, including a screening test and questionnaire, was posted on a private internet site that could be accessed via the VLDT Research Group home page, and 1,165 pediatric neurologists across Japan were invited to complete it. A total of 25 respondents reported prescribing VLDT; 19 used VLDT to treat autism spectrum disorder, 14 for tics, 12 for speech delay, 9 for Rett syndrome, 7 for attention-deficit/hyperactivity disorder, intellectual disability, and 6 for sleep problems. Twelve respondents reported prescribing a dose of 0.5 mg/kg. Twenty-two reported that VLDT was effective for treating behavioral problems, and twenty reported a good efficacy for treating motor symptoms. Adverse events had a low incidence. Notably, respondents chose VLDT for its possible action in DARSS and for its safety. VLDT was commonly used for behavioral problems in patients younger than 5 years, and for motor symptoms in aged 5-9 years. VLDT could safely treat behavioral and motor symptoms in pediatric neurological disorders. In contrast, dopamine antagonists are associated with potent efficacy, but with adverse effects such as sleepiness and obesity. Further surveys should be conducted with a broader participants.
突触后多巴胺受体超敏反应（DARSS）已由濑川正哉博士进行了广泛研究，他研究了极低剂量左旋多巴疗法（VLDT；0.5 - 1毫克/千克/天）的疗效。大量日本研究支持VLDT可用于治疗小儿神经疾病的可能性。我们在2014年进行了一项在线调查，以收集关于使用VLDT治疗DARSS的真实世界数据。一项包括筛查测试和问卷的两步调查发布在一个可通过VLDT研究小组主页访问的私人互联网网站上，邀请了日本各地的1165名儿科神经科医生完成该调查。共有25名受访者报告开具了VLDT处方；19人使用VLDT治疗自闭症谱系障碍，14人用于治疗抽动症，12人用于治疗语言发育迟缓，9人用于治疗雷特综合征，7人用于治疗注意力缺陷/多动障碍、智力障碍，6人用于治疗睡眠问题。12名受访者报告开具的剂量为0.5毫克/千克。22人报告VLDT对治疗行为问题有效，20人报告对治疗运动症状疗效良好。不良事件发生率较低。值得注意的是，受访者选择VLDT是因其在DARSS中可能的作用及其安全性。VLDT常用于5岁以下患者的行为问题，以及5 - 9岁患者的运动症状。VLDT可以安全地治疗小儿神经疾病中的行为和运动症状。相比之下，多巴胺拮抗剂疗效显著，但会产生嗜睡和肥胖等不良反应。应进行更广泛参与者的进一步调查。
