Safety Monitoring for Use of Propranolol for Hemangiomas

普萘洛尔治疗血管瘤的安全性监测

• 批准号: 8096354
• 负责人: BETH A DROLET
• 金额: $ 17.49万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-04-01 至 2013-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8096354
• 关键词: Adherence; American; Anatomy; Behavior; Benign; Blindness; Blood Vessels; Cardiac; Cardiology; Caring; Cells; Cessation of life; Child; Childhood; Cicatrix; Classification; Clinical; Clinical Research; Clinical Treatment; Clinical Trials; Collaborations; Combined Modality Therapy; Complex; Complication; Congestive Heart Failure; Consensus; Critical Care; Data; Development; Diagnosis; Discipline; Dose; Elements; Ensure; Epidemiology; FDA approved; Funding; Future; Goals; Grant; Growth; Guidelines; Hemangioma; Hemorrhage; Incidence; Individual; Infant; Institution; Institutional Review Boards; Intestines; Judgment; Lead; Left; Life; Liver; Lung; Manuals; Measures; Methods; Monitor; Multi-Institutional Clinical Trial; National Institute of Arthritis and Musculoskeletal and Skin Diseases; Neuraxis; Neurology; Oral; Organ; Outcome Measure; Patient-Focused Outcomes; Patients; Pediatric Neurology; Pharmaceutical Preparations; Phase III Clinical Trials; Physicians; Preparation; Procedures; Process; Proliferating; Propranolol; Protocols documentation; Quality of life; Randomized Controlled Clinical Trials; Recording of previous events; Recovery; Reporting; Research; Research Design; Research Methodology; Research Personnel; Safety; Sample Size; Severities; Severity of illness; Skin; Specialist; Spleen; Steroids; Strawberry nevus; System; Toxic effect; Training and Education; Treatment Protocols; Ulcer; United States; airway obstruction; comparative effectiveness; compare effectiveness; data integrity; data management; design; effectiveness trial; evidence base; health related quality of life; instrument; investigator training; meetings; member; multidisciplinary; novel; operation; patient population; prednisolone; rapid growth; response; success; tool; treatment response; tumor; tumor growth

DESCRIPTION (provided by applicant): Infantile hemangiomas are benign tumors composed of blood vessel cells that grow in the first months of life. Most infantile hemangiomas are found in the skin, but sometimes they occur in other organs in the body such as the liver, spleen, intestine, airway, lungs and the central nervous system. While most are benign in their behavior, some may interfere with vital functions and leave permanent scarring. Severe hemangiomas require treatment to control the growth of this tumor. Rapid growth may lead to complications such as ulceration, bleeding, loss of vision, airway obstruction, congestive heart failure, and permanent disfigurement. We lack uniform guidelines for treatment and methods to measure response to treatment of infants with hemangiomas. The objective of this R34 proposal is to establish a planning process and develop novel interdisciplinary strategies to overcome existing barriers to the implementation of a multi-institutional clinical trial. The overall goal of the future clinical trial is to determine the safety and efficacy of combination therapy of prednisolone plus propranolol compared to propranolol alone for complicated infantile hemangiomas. The proposed R34 planning project builds upon the success of the Hemangioma Investigator Group by incorporating complementary expertise to comprehensively construct the study design and protocol, and develop tools for data management, methods of research oversight, and other essential elements of the study including a manual of operations/procedures. The proposed research will rapidly advance the field by facilitating an investigator-initiated, randomized-controlled clinical trial targeted at providing evidence-based care for the critical clinical issues related to treatment of infants with complicated hemangiomas. PUBLIC HEALTH RELEVANCE: Hemangiomas are benign tumors composed of blood vessel cells that grow in the first months of life, and while most are uncomplicated, some may interfere with vital functions and leave permanent scarring. Severe hemangiomas require treatment to control the growth of this tumor, but physicians lack uniform guidelines for the treatment of infants with hemangiomas. The proposed research will quickly advance the field by providing the necessary data and expertise to implement research to evaluate the efficacy of new medications in the treatment of infants with complicated hemangiomas.

描述（由申请人提供）：婴儿血管瘤是由生命最初几个月生长的血管细胞组成的良性肿瘤。大多数婴儿血管瘤都在皮肤中发现，但有时它们发生在体内其他器官中，例如肝脏，脾，肠，气道，肺和中枢神经系统。虽然大多数人的行为都良性，但有些人可能会干扰重要的功能并留下永久性的疤痕。严重的血管瘤需要治疗以控制该肿瘤的生长。快速生长可能导致并发症，例如溃疡，出血，视力丧失，气道阻塞，充血性心力衰竭和永久性毁容。我们缺乏治疗方法统一的指南和测量对血管瘤治疗的反应的方法。该R34提案的目的是建立计划过程并制定新的跨学科策略，以克服实施多机构临床试验的现有障碍。未来临床试验的总体目标是确定泼尼松龙和普萘洛尔组合疗法的安全性和功效，而单独的普萘洛尔对复杂的婴儿血管瘤。拟议的R34计划项目是基于血管瘤研究者组成功的基础，该项目通过纳入补充专业知识来全面构建研究设计和协议，并为数据管理，研究监督方法以及研究的其他基本要素开发工具，包括手册/程序。拟议的研究将通过促进研究者发动的，随机控制的临床试验来迅速发展该领域，该试验针对与复杂血管瘤治疗有关的关键临床问题提供基于证据的护理。 公共卫生相关性：血管瘤是由血管细胞组成的良性肿瘤，这些肿瘤在生命的头几个月中生长，虽然大多数是简单的，但有些人可能会干扰重要的功能并留下永久性的疤痕。严重的血管瘤需要治疗以控制该肿瘤的生长，但医生缺乏治疗血管瘤婴儿的统一准则。拟议的研究将通过提供必要的数据和专业知识来实施研究以评估新药物在治疗复杂血管瘤的婴儿中的功效，从而迅速推进该领域。

项目成果

Retrospective review of adverse effects from propranolol in infants.

普萘洛尔对婴儿的不良反应的回顾性审查。

• DOI: 10.1001/jamadermatol.2013.2230
• 发表时间: 2013
• 期刊: JAMA dermatology
• 影响因子: 10.9
• 作者: Kwon,Eun-KyungM;Joachim,Shawna;Siegel,DawnH;Drolet,BethA;Holland,Kristen
• 通讯作者: Holland,Kristen