Abstract：
BACKGROUND: The approval of selumetinib in patients with neurofibromatosis type 1(NF1) and inoperable plexiform neurofibromas (PN) has reshaped the landscape of clinical management of the disease, and further comprehensive evaluation of the drug\'s efficacy and safety is needed.
METHODS: Original articles reporting on the efficacy and safety of elumetinib in patients with NF1 were comprehensively searched in the Pubmed database, Embase database, Cochrane Library, and Web of Science database and screened for inclusion of studies that met the criteria. We pooled the objective response rate (ORR), disease control rate (DCR), disease progression rate (DPR), and the rate of improvement in PN-related complications using meta-analysis. The incidence of drug-related adverse events was also statistically analyzed.
RESULTS: This study included 10 clinical trials involving 268 patients. The pooled ORR was 68.0% (95% CI 58.0-77.3%), the DCR was 96.8% (95% CI 90.8-99.7%) and the DPR was only 1.4% (95% CI 0-4.3%). The pooled improvement rate was 75.3% (95% CI 56.2-90.9%) for pain and 77.8% (95% CI 63.1-92.5%) for motor disorders. Most adverse events were mild, with the most common being gastrointestinal reactions (diarrhea: 62.5%; vomiting: 54.5%).
CONCLUSIONS: Our study demonstrates that selumetinib is effective in patients with NF1 and PN, significantly improving the serious complications associated with PN as well as having tolerable toxicities. Our findings help to increase clinicians\' confidence in applying selumetinib and promote the clinical adoption and benefit of the new drug.
METHODS: Original articles reporting on the efficacy and safety of elumetinib in patients with NF1 were comprehensively searched in the Pubmed database, Embase database, Cochrane Library, and Web of Science database and screened for inclusion of studies that met the criteria. We pooled the objective response rate (ORR), disease control rate (DCR), disease progression rate (DPR), and the rate of improvement in PN-related complications using meta-analysis. The incidence of drug-related adverse events was also statistically analyzed.
RESULTS: This study included 10 clinical trials involving 268 patients. The pooled ORR was 68.0% (95% CI 58.0-77.3%), the DCR was 96.8% (95% CI 90.8-99.7%) and the DPR was only 1.4% (95% CI 0-4.3%). The pooled improvement rate was 75.3% (95% CI 56.2-90.9%) for pain and 77.8% (95% CI 63.1-92.5%) for motor disorders. Most adverse events were mild, with the most common being gastrointestinal reactions (diarrhea: 62.5%; vomiting: 54.5%).
CONCLUSIONS: Our study demonstrates that selumetinib is effective in patients with NF1 and PN, significantly improving the serious complications associated with PN as well as having tolerable toxicities. Our findings help to increase clinicians\' confidence in applying selumetinib and promote the clinical adoption and benefit of the new drug.
摘要：
背景：在1型神经纤维瘤病（NF1）和无法手术的丛状神经纤维瘤（PN）患者中批准司美替尼重塑了该疾病的临床管理格局，需要进一步综合评价该药的疗效和安全性。
方法：在Pubmed数据库中全面搜索了报告埃米替尼在NF1患者中的疗效和安全性的原始文章，Embase数据库,科克伦图书馆,和WebofScience数据库，并筛选符合标准的研究。我们汇总了客观反应率(ORR)，疾病控制率（DCR），疾病进展率（DPR），和使用meta分析的PN相关并发症的改善率。对药物相关不良事件的发生率进行统计学分析。
结果：本研究包括10项临床试验，涉及268名患者。合并的ORR为68.0%（95%CI58.0-77.3%），DCR为96.8%(95%CI90.8~99.7%),DPR仅为1.4%(95%CI0~4.3%).疼痛的合并改善率为75.3％（95％CI56.2-90.9％），运动障碍的合并改善率为77.8％（95％CI63.1-92.5％）。大多数不良事件是轻微的，最常见的是胃肠道反应（腹泻：62.5%；呕吐：54.5%）。
结论：我们的研究表明，司美替尼对NF1和PN患者有效，显着改善与PN相关的严重并发症，并具有可耐受的毒性。我们的发现有助于提高临床医生应用司美替尼的信心，并促进新药的临床采用和获益。
方法：在Pubmed数据库中全面搜索了报告埃米替尼在NF1患者中的疗效和安全性的原始文章，Embase数据库,科克伦图书馆,和WebofScience数据库，并筛选符合标准的研究。我们汇总了客观反应率(ORR)，疾病控制率（DCR），疾病进展率（DPR），和使用meta分析的PN相关并发症的改善率。对药物相关不良事件的发生率进行统计学分析。
结果：本研究包括10项临床试验，涉及268名患者。合并的ORR为68.0%（95%CI58.0-77.3%），DCR为96.8%(95%CI90.8~99.7%),DPR仅为1.4%(95%CI0~4.3%).疼痛的合并改善率为75.3％（95％CI56.2-90.9％），运动障碍的合并改善率为77.8％（95％CI63.1-92.5％）。大多数不良事件是轻微的，最常见的是胃肠道反应（腹泻：62.5%；呕吐：54.5%）。
结论：我们的研究表明，司美替尼对NF1和PN患者有效，显着改善与PN相关的严重并发症，并具有可耐受的毒性。我们的发现有助于提高临床医生应用司美替尼的信心，并促进新药的临床采用和获益。
