PMID- 36015104 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20220830 IS - 1424-8247 (Print) IS - 1424-8247 (Electronic) IS - 1424-8247 (Linking) VI - 15 IP - 8 DP - 2022 Jul 31 TI - Efficacy and Safety of Trametinib in Neurofibromatosis Type 1-Associated Plexiform Neurofibroma and Low-Grade Glioma: A Systematic Review and Meta-Analysis. LID - 10.3390/ph15080956 [doi] LID - 956 AB - Trametinib has been used in neurofibromatosis type 1 (NF1) patients, especially those with unresectable nerve tumors, but no systematic review based on the latest studies has been published. We conducted this meta-analysis to evaluate the effectiveness and safety of trametinib in treating NF1-related nerve tumors. Original articles reporting the efficacy and safety of trametinib in NF1 patents were identified in PubMed, EMBASE, and Web of Science up to 1 June 2022. Using R software and the 'meta' package, the objective response rates (ORRs) and disease control rates (DCRs) were calculated to evaluate the efficacy, and the pooled proportion of adverse events (AEs) was calculated. The Grading of Recommendations, Assessment, Development and Evaluation system was used to assess the quality of evidence. Eight studies involving 92 patients were included, which had a very low to moderate quality of evidence. The pooled ORR was 45.3% (95% CI: 28.9-62.1%, I(2) = 0%), and the DCR was 99.8% (95% CI: 95.5-100%, I(2) = 0%). The most common AEs was paronychia, with a pooled rate of 60.7% (95% CI: 48.8-72.7%, I(2) = 0%). Our results indicate the satisfactory ability to stabilize tumor progression but a more limited ability to shrink tumors of trametinib in NF1-related nerve tumors. The safety profile of trametinib is satisfactory. FAU - Wang, Dun AU - Wang D AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. AD - Department of Burn and Plastic Surgery, West China Hospital of Sichuan University, No 37 Wainan Guoxue Road, Chengdu 610041, China. FAU - Ge, Lingling AU - Ge L AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Guo, Zizhen AU - Guo Z AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Li, Yuehua AU - Li Y AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Zhu, Beiyao AU - Zhu B AUID- ORCID: 0000-0002-6443-5586 AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Wang, Wei AU - Wang W AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Wei, Chengjiang AU - Wei C AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Li, Qingfeng AU - Li Q AUID- ORCID: 0000-0001-7822-618X AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. FAU - Wang, Zhichao AU - Wang Z AUID- ORCID: 0000-0002-5859-4193 AD - Department of Plastic and Reconstructive Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200011, China. LA - eng GR - 82102344; 82172228/National Natural Science Foundation of China/ GR - 20QA1405600/Shanghai Rising Star Program supported by Science and Technology Commission of Shanghai Municipality/ GR - 19JC1413/Science and Technology Commission of Shanghai Municipality/ GR - 22ZR1422300/Natural Science Foundation of Shanghai/ GR - 19CG18/"Chenguang Program" supported by Shanghai Education Development Foundation (SHEDF)/ GR - shslczdzk00901/Shanghai Municipal Key Clinical Specialty/ GR - SSMU-ZDCX20180700/Innovative research team of high-level local universities in Shanghai/ PT - Journal Article PT - Review DEP - 20220731 PL - Switzerland TA - Pharmaceuticals (Basel) JT - Pharmaceuticals (Basel, Switzerland) JID - 101238453 PMC - PMC9415905 OTO - NOTNLM OT - low-grade Glioma OT - meta-analysis OT - neurofibromatosis type 1 OT - plexiform neurofibroma OT - systematic review OT - trametinib COIS- There is no conflict of interests for all authors. EDAT- 2022/08/27 06:00 MHDA- 2022/08/27 06:01 PMCR- 2022/07/31 CRDT- 2022/08/26 01:41 PHST- 2022/06/30 00:00 [received] PHST- 2022/07/27 00:00 [revised] PHST- 2022/07/27 00:00 [accepted] PHST- 2022/08/26 01:41 [entrez] PHST- 2022/08/27 06:00 [pubmed] PHST- 2022/08/27 06:01 [medline] PHST- 2022/07/31 00:00 [pmc-release] AID - ph15080956 [pii] AID - pharmaceuticals-15-00956 [pii] AID - 10.3390/ph15080956 [doi] PST - epublish SO - Pharmaceuticals (Basel). 2022 Jul 31;15(8):956. doi: 10.3390/ph15080956.