Supplemental Digital Content is available in the text. Afferent lesions of the arterial baroreflex occur in familial dysautonomia. This leads to excessive blood pressure variability with falls and frequent surges that damage the organs. These hypertensive surges are the result of excess peripheral catecholamine release and have no adequate treatment. Carbidopa is a selective DOPA-decarboxylase inhibitor that suppresses catecholamines production outside the brain. To learn whether carbidopa can inhibit catecholamine-induced hypertensive surges in patients with severe afferent baroreflex failure, we conducted a double-blind randomized crossover trial in which patients with familial dysautonomia received high dose carbidopa (600 mg/day), low-dose carbidopa (300 mg/day), or matching placebo in 3 4-week treatment periods. Among the 22 patients enrolled (13 females/8 males), the median age was 26 (range, 12–59 years). At enrollment, patients had hypertensive peaks to 164/116 (range, 144/92 to 213/150 mm Hg). Twenty-four hour urinary norepinephrine excretion, a marker of peripheral catecholamine release, was significantly suppressed on both high dose and low dose carbidopa, compared with placebo (P=0.0075). The 2 co-primary end points of the trial were met. The SD of systolic BP variability was reduced at both carbidopa doses (low dose: 17±4; high dose: 18±5 mm Hg) compared with placebo (23±7 mm Hg; P=0.0013), and there was a significant reduction in the systolic BP peaks on active treatment (P=0.0015). High- and low-dose carbidopa were similarly effective and well tolerated. This study provides class Ib evidence that carbidopa can reduce blood pressure variability in patients with congenital afferent baroreflex failure. Similar beneficial effects are observed in patients with acquired baroreflex lesions.

中文翻译：

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卡比多巴治疗家族性自主神经功能障碍传入压力反射衰竭

补充数字内容在文本中可用。动脉压力反射的传入损伤发生在家族性自主神经功能障碍中。这会导致血压波动过大，跌倒和频繁的波动会损害器官。这些高血压激增是外周儿茶酚胺释放过多的结果，并且没有足够的治疗。Carbidopa 是一种选择性多巴脱羧酶抑制剂，可抑制脑外儿茶酚胺的产生。为了了解卡比多巴是否可以抑制重度传入压力反射衰竭患者的儿茶酚胺诱导的高血压激增，我们进行了一项双盲随机交叉试验，其中家族性自主神经功能障碍患者接受高剂量卡比多巴（600 毫克/天）、低剂量卡比多巴（ 300 毫克/天），或在 3 个 4 周的治疗期内匹配安慰剂。在入组的 22 名患者中（13 名女性/8 名男性），中位年龄为 26 岁（范围，12-59 岁）。入组时，患者的高血压峰值为 164/116（范围，144/92 至 213/150 mmHg）。与安慰剂相比，高剂量和低剂量卡比多巴的 24 小时尿去甲肾上腺素排泄（外周儿茶酚胺释放的标志物）均被显着抑制（P=0.0075）。满足试验的 2 个共同主要终点。与安慰剂（23±7 mm Hg；P=0.0013）相比，两种卡比多巴剂量（低剂量：17±4；高剂量：18±5 mm Hg）的收缩压变异性 SD 均降低，并且显着降低在积极治疗时收缩压达到峰值 (P=0.0015)。高剂量和低剂量卡比多巴同样有效且耐受性良好。该研究提供了 Ib 类证据，证明卡比多巴可以降低先天性传入压力反射衰竭患者的血压变异性。在获得性压力反射病变的患者中观察到类似的有益效果。