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Efficacy and safety of selumetinib in patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: a systematic review and meta-analysis.
Han, Yahui; Li, Biyun; Yu, Xiaokun; Liu, Jianing; Zhao, Wei; Zhang, Da; Zhang, Jiao.
Affiliation
  • Han Y; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China.
  • Li B; Department of Pediatric Hematology Oncology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, China.
  • Yu X; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China.
  • Liu J; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China.
  • Zhao W; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China.
  • Zhang D; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China.
  • Zhang J; Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, Henan, China. zhangjiaomail@126.com.
J Neurol ; 271(5): 2379-2389, 2024 May.
Article in En | MEDLINE | ID: mdl-38502338
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%).

CONCLUSION:

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.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Benzimidazoles / Neurofibromatosis 1 / Neurofibroma, Plexiform Limits: Humans Language: En Journal: J Neurol / J. neurol / Journal of neurology Year: 2024 Type: Article Affiliation country: China

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Benzimidazoles / Neurofibromatosis 1 / Neurofibroma, Plexiform Limits: Humans Language: En Journal: J Neurol / J. neurol / Journal of neurology Year: 2024 Type: Article Affiliation country: China