Hyperdopaminergic crises in familial dysautonomia
Hyperdopaminergic crises in familial dysautonomia
复制标题
家族性自主神经功能障碍的多巴胺能亢进危象
作者：
L. Norcliffe;Jose Martinez;F. Axelrod;H. Kaufmann
目的：本研究的目的是确定卡比多巴(Lodosyn)是否是治疗家族性自主神经障碍(FD)和高多巴胺能恶心/干呕/呕吐发作的有效止吐药物。方法：我们在一项开放标记滴定和治疗研究中招募了12名FD患者，以评估卡比多巴的安全性。然后，我们进行了一项随机、双盲、安慰剂对照的交叉研究，以评估其止吐效果。结果：在研究的每个患者中，以前的胃底折叠术预防了呕吐，但所有的受试者都经历了严重的周期性恶心和无法控制的瘙痒，这是标准治疗所不能解决的。卡比多巴平均每日剂量为480毫克(范围为325-600毫克/天)，耐受性良好。在双盲期，服用卡比多巴的患者恶心和呕吐明显少于服用安慰剂的患者(分别为0.03和0.02)。服用卡比多巴的患者24小时尿多巴胺排泄量(147±32微克/克铬)显著低于服用安慰剂的患者(222±41微克/克铬，p<；0.05)。结论：卡比多巴是治疗FD患者的一种安全有效的止吐药物，其机制可能是通过减少脑外多巴胺的形成。证据分类：这项研究提供了二级证据，证明卡比多巴在减少FD患者的恶心/呕吐/呕吐方面是有效的。
Objective: The purpose of this study was to determine whether carbidopa (Lodosyn), an inhibitor of dopa-decarboxylase that blocks the synthesis of dopamine outside the brain, is an effective antiemetic in patients with familial dysautonomia (FD) and hyperdopaminergic nausea/retching/vomiting attacks. Methods: We enrolled 12 patients with FD in an open-label titration and treatment study to assess the safety of carbidopa. We then conducted a randomized, double-blind, placebo-controlled, crossover study to evaluate its antiemetic efficacy. Results: Previous fundoplication surgery in each patient studied prevented vomiting, but all of the subjects experienced severe cyclical nausea and uncontrollable retching that was refractory to standard treatments. Carbidopa at an average daily dose of 480 mg (range 325–600 mg/day) was well tolerated. In the double-blind phase, patients experienced significantly less nausea and retching while on carbidopa than on placebo (p < 0.03 and p < 0.02, respectively). Twenty-four-hour urinary dopamine excretion was significantly lower while on carbidopa (147 ± 32 µg/gCr) than while on placebo (222 ± 41µg/gCr, p < 0.05). Conclusions: Carbidopa is a safe and effective antiemetic in patients with FD, likely by reducing the formation of dopamine outside the brain. Classification of evidence: This study provides Class II evidence that carbidopa is effective in reducing nausea/retching/vomiting in patients with FD.
影响因子：
9.8
作者：
Slaugenhaupt, SA;Blumenfeld, A;Gusella, JF
通讯作者：
Gusella, JF