PMID- 36333279
OWN - NLM
STAT- MEDLINE
DCOM- 20230303
LR  - 20230316
IS  - 2470-9239 (Electronic)
IS  - 2470-9239 (Linking)
VI  - 8
IP  - 1
DP  - 2023 Mar
TI  - The efficacy and safety of ganaxolone for the treatment of refractory epilepsy: A 
      meta-analysis from randomized controlled trials.
PG  - 90-99
LID - 10.1002/epi4.12669 [doi]
AB  - OBJECTIVE: Epilepsy is one of the most common and refractory neurological 
      disorders globally. Ganaxolone, a neuroactive steroid that enhances GABAergic 
      inhibition, has been tested in many trials for the resolution of refractory 
      epilepsy. Based on these, our study implemented a meta-analysis to evaluate the 
      general benefit of ganaxolone for refractory epilepsy. METHODS: EMBASE, Medline, 
      Scopus, Cochrane Library, and Clinicaltrials.gov were searched for relevant 
      randomized controlled trials (RCTs) up to June 20, 2022. The risk ratio (RR) and 
      standard mean difference (SMD) were analyzed using dichotomous and continuous 
      outcomes, respectively with a random effect model. Trial sequential analysis 
      (TSA) was also performed to judge the reliability of results. RESULTS: We totally 
      collected 659 patients from four RCTs to evaluate the efficacy and safety of 
      ganaxolone. As results showed, >/=50% reduction in mean seizure frequency has 
      improved significantly compared with placebo (RR = 1.60, 95%CI: 1.02-2.49, 
      p = 0.04, I(2)  = 30%), which is supported by TSA. However, the percentage of 
      seizure-free days shows no statistical significance (p = 0.36). For safety 
      outcomes, adverse events (AEs), serious adverse events, and AE leading to study 
      drug discontinuation all revealed no obvious difference between ganaxolone and 
      placebo (p > 0.05). SIGNIFICANCE: Based on our research, we have observed that 
      ganaxolone is safe and has potential efficacy in the treatment of refractory 
      epilepsy, waiting for further studies.
CI  - (c) 2022 The Authors. Epilepsia Open published by Wiley Periodicals LLC on behalf 
      of International League Against Epilepsy.
FAU - Meng, Jiahao
AU  - Meng J
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
FAU - Yan, Zeya
AU  - Yan Z
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
FAU - Tao, Xinyu
AU  - Tao X
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
FAU - Wang, Wei
AU  - Wang W
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
FAU - Wang, Fei
AU  - Wang F
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
FAU - Xue, Tao
AU  - Xue T
AUID- ORCID: 0000-0002-1443-2475
AD  - Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, 
      Beijing, China.
FAU - Liu, Yanfei
AU  - Liu Y
AD  - Department of Neurosurgery, Suzhou Integrated Traditional Chinese and Western 
      Medicine Hospital, Suzhou, Jiangsu Province, China.
FAU - Wang, Zhong
AU  - Wang Z
AUID- ORCID: 0000-0002-7342-0845
AD  - Department of Neurosurgery & Brain and Nerve Research Laboratory, The First 
      Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China.
LA  - eng
PT  - Journal Article
PT  - Meta-Analysis
PT  - Research Support, Non-U.S. Gov't
DEP - 20221115
PL  - United States
TA  - Epilepsia Open
JT  - Epilepsia open
JID - 101692036
RN  - 0 (Anticonvulsants)
RN  - 98WI44OHIQ (ganaxolone)
SB  - IM
MH  - Humans
MH  - Anticonvulsants/therapeutic use
MH  - *Drug Resistant Epilepsy
MH  - Randomized Controlled Trials as Topic
MH  - *Epilepsy/drug therapy
PMC - PMC9978077
OTO - NOTNLM
OT  - GABAergic inhibition
OT  - ganaxolone
OT  - meta-analysis
OT  - refractory epilepsy
COIS- Neither of the authors has any conflict of interest to disclose. We confirm that 
      we have read the Journal's position on issues involved in ethical publication and 
      affirm that this report is consistent with those guidelines.
EDAT- 2022/11/06 06:00
MHDA- 2023/03/04 06:00
PMCR- 2022/11/15
CRDT- 2022/11/05 00:02
PHST- 2022/08/07 00:00 [received]
PHST- 2022/11/02 00:00 [accepted]
PHST- 2022/11/06 06:00 [pubmed]
PHST- 2023/03/04 06:00 [medline]
PHST- 2022/11/05 00:02 [entrez]
PHST- 2022/11/15 00:00 [pmc-release]
AID - EPI412669 [pii]
AID - 10.1002/epi4.12669 [doi]
PST - ppublish
SO  - Epilepsia Open. 2023 Mar;8(1):90-99. doi: 10.1002/epi4.12669. Epub 2022 Nov 15.