PMID- 35017312
OWN - NLM
STAT- MEDLINE
DCOM- 20220406
LR  - 20220406
IS  - 1526-632X (Electronic)
IS  - 0028-3878 (Linking)
VI  - 98
IP  - 9
DP  - 2022 Mar 1
TI  - Efficacy and Safety of Selumetinib in Pediatric Patients With Neurofibromatosis 
      Type 1: A Systematic Review and Meta-analysis.
PG  - e938-e946
LID - 10.1212/WNL.0000000000013296 [doi]
AB  - BACKGROUND AND OBJECTIVES: Although the recent approval of selumetinib is 
      expected to transform the management of children with neurofibromatosis type 1 
      (NF1), particularly those with symptomatic and inoperable plexiform 
      neurofibromas, no systematic review has summarized its efficacy and safety based 
      on the latest studies. This study was conducted to systematically evaluate the 
      efficacy and safety of selumetinib in children with NF1. METHODS: Original 
      articles reporting the efficacy and safety of selumetinib in patients with NF1 
      were identified in PubMed and EMBASE up to January 28, 2021. The pooled objective 
      response rates (ORRs) and disease control rates (DCRs) were calculated using the 
      DerSimonian-Laird method based on random-effects modeling. The pooled proportion 
      of adverse events (AEs) was also calculated. The quality of the evidence was 
      assessed using the Grading of Recommendations, Assessment, Development and 
      Evaluation system. RESULTS: Five studies involving 126 patients were included in 
      our analysis. The studies had a very low to moderate quality of the evidence. The 
      pooled ORR was 73.8% (95% CI 57.3%-85.5%) and the DCR was 92.5% (95% CI 
      66.5%-98.7%). The 2 most common AEs were diarrhea, which had a pooled rate of 
      63.8% (95% CI 52.9%-73.4%), and an increase in creatine kinase levels, which had 
      a pooled rate of 63.3% (95% CI 35.6%-84.3%). DISCUSSION: Our results indicate 
      that selumetinib is an effective and safe treatment for pediatric patients with 
      symptomatic, inoperable plexiform neurofibromas. Further larger-scale randomized 
      controlled studies are needed to confirm the long-term outcome of patients 
      treated with this drug.
CI  - (c) 2022 American Academy of Neurology.
FAU - Hwang, Jisun
AU  - Hwang J
AUID- ORCID: 0000-0002-7593-2246
AD  - From the Department of Radiology (J.H.), Dongtan Sacred Heart Hospital, Hallym 
      University Medical Center, Gyeonggi-do; and Department of Radiology and Research 
      Institute of Radiology (H.M.Y., P.H.K., K.W.K.) and Department of Pediatrics 
      (B.H.L.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, 
      South Korea.
FAU - Yoon, Hee Mang
AU  - Yoon HM
AUID- ORCID: 0000-0001-6491-5734
AD  - From the Department of Radiology (J.H.), Dongtan Sacred Heart Hospital, Hallym 
      University Medical Center, Gyeonggi-do; and Department of Radiology and Research 
      Institute of Radiology (H.M.Y., P.H.K., K.W.K.) and Department of Pediatrics 
      (B.H.L.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, 
      South Korea. espoirhm@gmail.com.
FAU - Lee, Beom Hee
AU  - Lee BH
AUID- ORCID: 0000-0001-9709-2631
AD  - From the Department of Radiology (J.H.), Dongtan Sacred Heart Hospital, Hallym 
      University Medical Center, Gyeonggi-do; and Department of Radiology and Research 
      Institute of Radiology (H.M.Y., P.H.K., K.W.K.) and Department of Pediatrics 
      (B.H.L.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, 
      South Korea.
FAU - Kim, Pyeong Hwa
AU  - Kim PH
AD  - From the Department of Radiology (J.H.), Dongtan Sacred Heart Hospital, Hallym 
      University Medical Center, Gyeonggi-do; and Department of Radiology and Research 
      Institute of Radiology (H.M.Y., P.H.K., K.W.K.) and Department of Pediatrics 
      (B.H.L.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, 
      South Korea.
FAU - Kim, Kyung Won
AU  - Kim KW
AD  - From the Department of Radiology (J.H.), Dongtan Sacred Heart Hospital, Hallym 
      University Medical Center, Gyeonggi-do; and Department of Radiology and Research 
      Institute of Radiology (H.M.Y., P.H.K., K.W.K.) and Department of Pediatrics 
      (B.H.L.), Asan Medical Center, University of Ulsan College of Medicine, Seoul, 
      South Korea.
LA  - eng
PT  - Journal Article
PT  - Meta-Analysis
PT  - Research Support, Non-U.S. Gov't
PT  - Systematic Review
DEP - 20220111
PL  - United States
TA  - Neurology
JT  - Neurology
JID - 0401060
RN  - 0 (AZD 6244)
RN  - 0 (Benzimidazoles)
SB  - IM
MH  - Benzimidazoles/adverse effects
MH  - Child
MH  - Diarrhea
MH  - Humans
MH  - *Neurofibroma, Plexiform
MH  - *Neurofibromatosis 1/complications/drug therapy
EDAT- 2022/01/13 06:00
MHDA- 2022/04/07 06:00
CRDT- 2022/01/12 06:12
PHST- 2021/08/04 00:00 [received]
PHST- 2021/12/27 00:00 [accepted]
PHST- 2022/01/13 06:00 [pubmed]
PHST- 2022/04/07 06:00 [medline]
PHST- 2022/01/12 06:12 [entrez]
AID - WNL.0000000000013296 [pii]
AID - 10.1212/WNL.0000000000013296 [doi]
PST - ppublish
SO  - Neurology. 2022 Mar 1;98(9):e938-e946. doi: 10.1212/WNL.0000000000013296. Epub 
      2022 Jan 11.