Twin-Twin Transfusion Syndrome Trial

双胎输血综合症试验

• 批准号: 6536423
• 负责人: TIMOTHY M CROMBLEHOLME
• 金额: $ 149.97万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-09-24 至 2006-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6536423
• 关键词: amniotic fluid; body fluid balance therapy; clinical trials; echocardiography; embryo /fetus therapy; human pregnant subject; human subject; human therapy evaluation; magnetic resonance imaging; patient oriented research; photocoagulation therapy; placenta disorders; twin /multiplet; ultrasonography

DESCRIPTION (provided by applicant): The twin twin transfusion syndrome (TTTS) is the most common serious complication of monochorionic twin gestations, affecting between 4 and 35% of monochorionic twin pregnancies each year in the United States. The TTTS accounts for 17% of all perinatal mortality associated with twin gestations. Standard therapy in the United States has been limited to serial amnioreduction which appears to improve the overall outcome but has had little impact on the more severe end of the spectrum in TTTS. In addition, survivors of TTTS treated by serial amnioreduction have an 18 to 26% incidence of significant neurologic and cardiac morbidity. Selective fetoscopic laser photocoagulation of chorioangiopagus has emerged as an alternative treatment strategy in TTTS with comparable survival to serial amnioreduction in non-randomized, non- prospective clinical studies. The superiority of fetoscopic laser treatment of TTTS remains unproven. In our preliminary data we have observed enhanced survival with selective fetoscopic laser photocoagulation in pregnancies with TTTS that had already failed serial amnioreduction or rnicroseptostomy. We hypothesize that treatment of the underlying chorioangiopagus by selective fetoscopic laser photocoagulation will not only improve the survival of twins but will reduce the incidence of neurologic, cardiac, and developmental sequelae of TTTS. We propose to test this hypothesis by a prospective randomized multicenter trial to compare serial amnioreduction with selective fetoscopic laser photocoagulation. In. the course of this study we will address the following specific aims: 1) To demonstrate that selective laser photocoagulation of chorioangiopagus in severe tETS results in significantly improved survival of twins compared to aggressive serial amnioreduction therapy. Survival of recipients and donors, both within the same pregnancy and overall survival to 30 days after birth will be examined; 2) To demonstrate that selective laser photocoagulation results in significantly improved cardiac, neurologic, and developmental outcomes in survivors of severe TTTS compared to survivors treated by aggressive serial amnioreduction. This will be a multicenter prospective randomized clinical trial with patients randomized either to serial amnioreduction at one of 16 participating centers or selective fetoscopic laser surgery at one or two participating centers. The fetal echocardiograms will be reviewed in a blinded fashion to evaluate cardiac morbidity and response to treatment.. Similarly, prenatal ultrasounds, as well as ultrasounds and MRI s obtained in the first week of life and after 4 weeks, will be reviewed in a blinded fashion to evaluate the neurologic morbidity and distinguish prenatal from perinatal events. Long-term neurodevelopmental outcome will be evaluated by the NIGH]) Neonatal Network at 18 to 22 months of age. The overall goal of the study is to improve the outcomes of twins with TTTS by determining which treatment for TTTS has a better survival as well as cardiac, neurologic, and developmental outcome.

描述（由申请人提供）：双胞胎输血综合征 TTTS是单绒毛膜双胎妊娠最常见的严重并发症 妊娠，影响4%至35%的单绒毛膜双胎妊娠， 年在美国。TTTS占所有围产儿的17 与双胞胎妊娠相关的死亡率。美国的标准治疗 国家一直局限于一系列的减少，这似乎改善了 总体结果，但对更严重的结局影响不大， TTTS中的频谱。此外，接受系列治疗的TTTS幸存者 脊髓减压术有18 - 26%的神经系统和 心脏病发病率选择性胎儿镜下激光光凝治疗胎儿宫内窘迫 脉络膜血管连胎已成为TTTS的替代治疗策略， 在非随机、 前瞻性临床研究。胎儿镜下激光治疗先天性胎儿畸形的优越性 TTTS仍然未经证实。在我们的初步数据中，我们观察到 选择性胎儿镜激光光凝治疗妊娠合并 TTTS已经失败了一系列的脊髓复位术或显微中隔造口术。我们 假设通过选择性血管造影术治疗潜在脉络膜血管连胎 胎儿镜激光光凝不仅可以提高双胞胎的存活率， 但会降低神经系统、心脏和发育方面的发病率 TTTS的后遗症。我们建议通过一个前瞻性的实验来验证这一假设。 一项随机多中心试验，比较连续性脊柱复位术与选择性脊柱复位术 胎儿镜激光凝固术In.在这项研究中，我们将 解决以下具体目标：1）证明选择性激光 在严重的TETS中，脉络膜血管连接的光凝导致显著的 与积极的连续减胎术相比，双胎存活率提高 疗法接受者和捐赠者的存活率，无论是在同一怀孕期间， 将检查出生后30天的总生存率; 2）证明 选择性激光光凝导致显著改善 严重TTTS幸存者的心脏、神经和发育结局 与接受积极的连续减容术的幸存者相比。这将 是一项多中心前瞻性随机临床试验， 在16个参与中心之一随机接受连续减胎术 或在一个或两个参与中心进行选择性胎儿镜激光手术。的 胎儿超声心动图将以盲法进行审查， 心脏病发病率和治疗反应。同样，产前超声波， 以及出生后第一周和之后的超声和MRI检查， 4周，将以设盲方式进行审查，以评价神经系统 发病率和区分产前和围产期事件。长期 神经发育结果将由NIGH评估]）新生儿网络， 18至22个月大。研究的总体目标是改善 通过确定TTTS的治疗方法， 更好的存活率以及心脏、神经和发育结果。