PDF(Pubmed)
Abstract：
UNASSIGNED: There is a paucity of literature that comprehensively analyzes previous and current clinical trials targeting neurofibromatoses-related tumors. This article aims to provide readers with drug development efforts targeting these tumors by analyzing translational and clinical findings.
UNASSIGNED: This systematic review was written according to the PRISMA guidelines. Inclusion criteria were clinical trials involving patients with neurofibromatosis type 1, type 2, or schwannomatosis that were treated with therapies targeting neurofibromatoses-associated tumors and that were registered on clinicaltrials.gov. In addition, a search was performed in PubMed, Web of Science, Google Scholar, and Embase European for articles fully describing these clinical trials.
UNASSIGNED: A total of 265 clinical trials were registered and screened for eligibility. Ninety-two were included in this systematic review involving approximately 4636 participants. The number of therapies analyzed was more than 50. Drugs under investigation mainly act on the MAPK/ERK and PI3K/AKT/mTOR pathways, tumor microenvironment, or aberrantly over-expressed cell surface receptors. Selumetinib was the most effective medication for treating a neurofibromatosis type 1-associated tumor with approximately 68%-71% partial response for inoperable or progressive plexiform neurofibromas in children 2 years of age and older and bevacizumab for a neurofibromatosis type 2-related tumor with approximately 36%-41% partial response for vestibular schwannomas in patients 12 years of age and older.
UNASSIGNED: This systematic review presents the results of previous clinical investigations and those under development for neurofibromatoses-associated tumors. Clinicians may use this information to strategize patients to appropriate clinical trials.
UNASSIGNED: This systematic review was written according to the PRISMA guidelines. Inclusion criteria were clinical trials involving patients with neurofibromatosis type 1, type 2, or schwannomatosis that were treated with therapies targeting neurofibromatoses-associated tumors and that were registered on clinicaltrials.gov. In addition, a search was performed in PubMed, Web of Science, Google Scholar, and Embase European for articles fully describing these clinical trials.
UNASSIGNED: A total of 265 clinical trials were registered and screened for eligibility. Ninety-two were included in this systematic review involving approximately 4636 participants. The number of therapies analyzed was more than 50. Drugs under investigation mainly act on the MAPK/ERK and PI3K/AKT/mTOR pathways, tumor microenvironment, or aberrantly over-expressed cell surface receptors. Selumetinib was the most effective medication for treating a neurofibromatosis type 1-associated tumor with approximately 68%-71% partial response for inoperable or progressive plexiform neurofibromas in children 2 years of age and older and bevacizumab for a neurofibromatosis type 2-related tumor with approximately 36%-41% partial response for vestibular schwannomas in patients 12 years of age and older.
UNASSIGNED: This systematic review presents the results of previous clinical investigations and those under development for neurofibromatoses-associated tumors. Clinicians may use this information to strategize patients to appropriate clinical trials.
摘要：
UNASSIGNED：缺乏全面分析针对神经纤维瘤相关肿瘤的先前和当前临床试验的文献。本文旨在通过分析转化和临床发现，为读者提供针对这些肿瘤的药物开发工作。
UNASSIGNED：本系统综述是根据PRISMA指南编写的。纳入标准是涉及神经纤维瘤病1型，2型或神经鞘瘤病患者的临床试验，这些患者接受了针对神经纤维瘤相关肿瘤的治疗，并在临床试验中注册。此外,在PubMed中进行了搜索，WebofScience,谷歌学者，以及EmbaseEuropean的完整描述这些临床试验的文章。
UNASSIGNED：共有265项临床试验被注册并筛选合格。该系统评价包括92名，涉及约4636名参与者。分析的治疗方法超过50种。研究药物主要作用于MAPK/ERK和PI3K/AKT/mTOR通路,肿瘤微环境,或异常过度表达的细胞表面受体。Selumetinib是治疗1型神经纤维瘤病相关肿瘤的最有效药物，对2岁及以上儿童的不可手术或进行性丛状神经纤维瘤有约68％-71％的部分反应，对贝伐单抗治疗2型神经纤维瘤相关肿瘤，对12岁及以上的前庭神经鞘瘤有约36％-41％的部分反应。
UNASSIGNED：本系统综述介绍了神经纤维瘤相关肿瘤的既往临床研究和开发中的结果。临床医生可以使用这些信息为患者制定适当的临床试验策略。
UNASSIGNED：本系统综述是根据PRISMA指南编写的。纳入标准是涉及神经纤维瘤病1型，2型或神经鞘瘤病患者的临床试验，这些患者接受了针对神经纤维瘤相关肿瘤的治疗，并在临床试验中注册。此外,在PubMed中进行了搜索，WebofScience,谷歌学者，以及EmbaseEuropean的完整描述这些临床试验的文章。
UNASSIGNED：共有265项临床试验被注册并筛选合格。该系统评价包括92名，涉及约4636名参与者。分析的治疗方法超过50种。研究药物主要作用于MAPK/ERK和PI3K/AKT/mTOR通路,肿瘤微环境,或异常过度表达的细胞表面受体。Selumetinib是治疗1型神经纤维瘤病相关肿瘤的最有效药物，对2岁及以上儿童的不可手术或进行性丛状神经纤维瘤有约68％-71％的部分反应，对贝伐单抗治疗2型神经纤维瘤相关肿瘤，对12岁及以上的前庭神经鞘瘤有约36％-41％的部分反应。
UNASSIGNED：本系统综述介绍了神经纤维瘤相关肿瘤的既往临床研究和开发中的结果。临床医生可以使用这些信息为患者制定适当的临床试验策略。
