Pediatric Heart Network University of Utah

犹他大学儿科心脏网络

• 批准号: 8692577
• 负责人: L. LuAnn Minich
• 金额: $ 44.6万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-01 至 2016-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8692577
• 关键词: Address; Adverse effects; Adverse event; Age; Age-Months; Anabolic steroids; Burn injury; C-reactive protein; Cardiac Surgery procedures; Cardiovascular system; Cessation of life; Child; Childhood; Chronic; Clinical; Clinical Research; Collection; Complex; Congenital Abnormality; Constitutional; Data Analyses; Data Coordinating Center; Disease; Effectiveness; Energy Intake; Failure; Feeding Methods; Goals; Growth; HIV Infections; Head circumference; Health Care Costs; Heart; Heart Transplantation; Height; Hepatic; Hospital Costs; Hospitalization; Hospitals; Hour; Hypoplastic Left Heart Syndrome; Infant; Intensive Care; Intervention; Laboratories; Lead; Life; Mechanical ventilation; Medical; Metabolic stress; Morbidity - disease rate; National Heart, Lung, and Blood Institute; Neonatal; Nitrogen; Nutritional; Operative Surgical Procedures; Outcome; Outcome Assessment; Oxandrolone; Performance; Perioperative; Perioperative Care; Pharmaceutical Preparations; Pharmacotherapy; Physical Examination; Placebos; Postoperative Complications; Prealbumin; Principal Investigator; Prior Therapy; Procedures; Protein Biosynthesis; Protocols documentation; Pulmonary artery structure; Randomized; Resources; Risk; Risk Factors; Roentgen Rays; Safety; Serum; Shunt Device; Surgeon; Techniques; Testing; Time; Transplantation; Turner' s Syndrome; Universities; Urine; Utah; Ventricular Function; Weight; Weight Gain; aortic arch; compare effectiveness; design; double-blind placebo controlled trial; feeding; girls; hemodynamics; improved; innovation; modifiable risk; neurodevelopment; nitrogen balance; operation; palliative; primary outcome; reconstruction; secondary outcome; skeletal muscle growth; success; wasting; willingness

DESCRIPTION (provided by applicant): Since 2001, the Utah Pediatric Heart Network's accomplishments in performing clinical research, including protocol design, recruitment, study performance, data analysis and interpretation, and dissemination of results, demonstrate their willingness to collaborate with the other clinical centers, the Data Coordinating Center, and NHLBI. This study addresses the significant problem of growth failure in infants with hypoplastic left heart syndrome (HLHS). As the first study to evaluate the effectiveness of a pharmacologic intervention that does not target the cardiovascular system to improve growth, it also demonstrates innovation. The improved survival of infants with HLHS is accompanied by considerable morbidity and resource utilization. Their hospital costs are among the highest of any type of birth defect, exceeding $180,000,000/year. Infants with HLHS undergo at least two complex palliative procedures during their first few months: aortic arch reconstruction with pulmonary artery shunt (Norwood procedure) often before one week of age, and superior cavopulmonary connection (SCPC) usually at four to six months of age. Neonatal cardiac surgery places a significant metabolic stress on these infants, many who are already malnourished at the time of their operation. After the Norwood procedure, infants have a drastic decline in weight for age and continue to grow poorly between Nonwood discharge and the SCPC surgery. Poor growth is one of the most important risk factors for prolonged hospitalization, death or transplant, and impaired neurodevelopment. Medications aimed at improving hemodynamics and specific feeding protocols have failed to improve growth in these infants. Oxandrolone is an anabolic steroid that has been in routine clinical use for over 30 years. The drug has few side effects and has improved weight gain in children with extensive burns and improved growth in children with short stature, including girls with Turner's syndrome. We propose a multicenter, randomized, double- blind, placebo-controlled trial to assess the impact of a short course (30 days) of oxandrolone therapy to promote growth in infants with HLHS. The specific aims are to: 1) evaluate the effectiveness of oxandrolone therapy on growth, hospital course, and neurodevelopmental outcome and 2) describe the safety profile of oxandrolone therapy in infants with HLHS. RELEVANCE: Surviving infants with HLHS have ongoing medical problems and high health care costs. These infants grow poorly in the first months of life, increasing their risk for longer hospitalizations, poor neurodevelopment, and heart transplant or death. Growth is not improved by medications aimed at improving hemodynamics or by specific feeding methods. Oxandrolone is a drug that has helped other children gain weight If successful, oxandrolone therapy would represent a major breakthrough impacting the field by improving outcomes and decreasing the resource utilization for infants with HLHS.

描述（由申请人提供）： 自 2001 年以来，犹他州儿科心脏网络在临床研究方面取得的成就，包括方案设计、招募、研究绩效、数据分析和解释以及结果传播，表明他们愿意与其他临床中心、数据协调中心和 NHLBI 合作。 这项研究解决了左心发育不全综合征 (HLHS) 婴儿生长障碍的重大问题。作为第一项评估不针对心血管系统以促进生长的药物干预有效性的研究，它也展示了创新。 HLHS 婴儿生存率的提高伴随着相当大的发病率和资源利用率。他们的住院费用是所有类型出生缺陷中最高的，每年超过 1.8 亿美元。患有 HLHS 的婴儿在最初几个月内至少要经历两次复杂的姑息手术：通常在一周大之前进行主动脉弓重建和肺动脉分流术（诺伍德手术），以及通常在四到六个月大时进行上腔静脉肺动脉连接术（SCPC）。新生儿心脏手术给这些婴儿带来了显着的代谢压力，许多婴儿在手术时已经营养不良。 Norwood 手术后，婴儿的体重与年龄相比急剧下降，并且在 Nonwood 出院和 SCPC 手术之间继续生长不良。生长不良是导致长期住院、死亡或移植以及神经发育受损的最重要的危险因素之一。旨在改善血流动力学的药物和特定的喂养方案未能改善这些婴儿的生长。 氧雄龙是一种合成代谢类固醇，已在临床常规使用 30 多年。该药物几乎没有副作用，可以改善大面积烧伤儿童的体重增加，并改善身材矮小的儿童（包括患有特纳氏综合症的女孩）的生长。我们提出了一项多中心、随机、双盲、安慰剂对照试验，以评估短期（30 天）氧雄龙治疗对促进 HLHS 婴儿生长的影响。具体目标是：1) 评估氧雄龙治疗对生长、住院病程和神经发育结果的有效性；2) 描述氧雄龙治疗 HLHS 婴儿的安全性。 相关性：患有 HLHS 的幸存婴儿存在持续的医疗问题和高昂的医疗费用。这些婴儿在生命的最初几个月生长不良，增加了他们住院时间较长、神经发育不良、心脏移植或死亡的风险。旨在改善血流动力学的药物或特定的喂养方法并不能改善生长。氧雄龙是一种帮助其他儿童增加体重的药物。如果成功，氧雄龙疗法将通过改善 HLHS 婴儿的治疗结果并减少资源利用率，代表着该领域的重大突破。