背景:G-POEM是治疗难治性胃轻瘫的一种有前途的技术。我们提出了第一项双盲随机研究,比较了G-POEM与幽门肉毒杆菌毒素注射液(BTI)的临床疗效。
方法:这项随机研究在两个专家中心进行。入选的患者患有难治性胃轻瘫,医学管理>6个月,并通过胃排空闪烁显像(GES)证实。随机分为两组:G-POEM和BTI,随访1年。主要终点是3个月和1年的临床疗效,定义为平均胃轻瘫躯体症状指数(GCSI)评分的降低。次要终点是GES进化,不良事件,生活质量(GIQLI和SF-12)。
结果:40名患者(22名女性,18名平均年龄为48.1±17.4岁的男性)被随机分组。平均症状持续时间为5.8±5.7年。病因为糖尿病(n=11),特发性(n=18),术后(n=6),或混合(n=4)。与BTI相比,G-POEM组的3个月临床成功率更高(65%对40%,分别,p=0.15)。1年的临床成功,在意向治疗分析中,也更高(60%对40%,分别),但不显著。两组GCSI在3个月和1年时均下降。G-POEM组仅发生1次轻微AE。G-POEM组的GES改善率为72%,而BTI组(NS)为50%。
结论:G-POEM似乎比BTI具有更高的临床相关成功率,但没有统计证明。不管怎样,这项研究证实了针对幽门的治疗的兴趣,无论是机械的还是化学的,用于治疗难治性胃轻瘫。
BACKGROUND: Gastric peroral endoscopic myotomy (G-POEM) is a promising technique for treating refractory gastroparesis. We present the first double-blind randomized study comparing the clinical efficacy of G-POEM versus pyloric botulinum toxin injection (BTI).
METHODS: This randomized study, conducted in two expert centers, enrolled patients with refractory gastroparesis, medically managed for >6 months and confirmed by gastric emptying scintigraphy (GES), into two groups, G-POEM versus BTI, with follow-up of 1 year. The primary end point was the 3-month clinical efficacy, defined as a >1-point decrease in the mean Gastroparesis Cardinal Symptom Index (GCSI) score. Secondary end points were: 1-year efficacy, GES evolution, adverse events, and quality of life.
RESULTS: 40 patients (22 women; mean age 48.1 [SD 17.4]), with mean symptom duration of 5.8 (SD 5.7) years, were randomized. Etiologies included idiopathic (n=18), diabetes (n=11), postoperative (n=6), and mixed (n=4). G-POEM showed a higher 3-month clinical success than BTI (65% vs. 40%, respectively; P=0.10), along with non-significantly higher 1-year clinical success (60% vs. 40%, respectively) on intention-to-treat analysis. The GCSI decreased in both groups at 3 months and 1 year. Only three minor adverse events occurred in the G-POEM group. The GES improvement rate was 72% in the G-POEM group versus 50% in the BTI group (non-significant).
CONCLUSIONS: G-POEM seems to have a higher clinically relevant success rate than BTI, but this was not statistically demonstrated. This study confirms the interest in treatments targeting the pylorus, either mechanically or chemically, for managing refractory gastroparesis.