PMID- 38300476
OWN - NLM
STAT- MEDLINE
DCOM- 20240222
LR  - 20240419
IS  - 1179-1934 (Electronic)
IS  - 1172-7047 (Linking)
VI  - 38
IP  - 2
DP  - 2024 Feb
TI  - The Efficacy and Safety of Different Targeted Drugs for the Treatment of 
      Generalized Myasthenia Gravis: A Systematic Review and Bayesian Network 
      Meta-analysis.
PG  - 93-104
LID - 10.1007/s40263-024-01062-7 [doi]
AB  - BACKGROUND: The treatment of generalized myasthenia gravis (gMG) has been 
      transformed by the development and approval of new targeted therapies. This 
      analysis aimed to rank and compare the new therapies for gMG using efficacy and 
      safety data from randomized controlled trials (RCTs). METHODS: We searched 
      PubMed, Embase, the Cochrane Central Register of Controlled Trials (CENTRAL), and 
      ClinicalTrials.gov (up to November 2022) for RCTs of targeted drugs for gMG. We 
      used a Bayesian random-effects network meta-analysis (NMA) model and a Markov 
      chain Monte Carlo (MCMC) model for statistical analysis. The primary outcome was 
      the change in quantitative myasthenia gravis score (QMGS) from baseline, while 
      the secondary outcome was the risk ratio (RR) of adverse events (AEs) during 
      treatment. The surface under the cumulative ranking curve (SUCRA) was used to 
      rank these targeted drugs, with higher SUCRA values indicating better efficacy or 
      lower likelihood of AEs. RESULTS: In total, 13 studies (872 subjects) were 
      included in this analysis evaluating 10 targeted drugs (batoclimab, belimumab, 
      CFZ533, eculizumab, efgartigimod, nipocalimab, rituximab, ravulizumab, 
      rozanolixizumab, and zilucoplan). With regards to the primary outcome, batoclimab 
      [standardized mean difference (SMD), - 1.61; 95% credible interval (CrI), - 2.78, 
      - 0.43] significantly reduced QMGS in patients with gMG when compared with 
      placebo and was ranked as the most efficacious drug. Ranked second and third were 
      eculizumab (SMD, - 0.67; 95% CrI, 1.43, 0.01) and zilucoplan (SMD, - 0.54; 95% 
      CrI, - 1.56, 0.46), respectively. Nipoclimab (SMD, - 0.02; 95% CrI, - 1.04, 1.00) 
      had the worst efficacy and ranked last among all targeted drugs. In our study, 
      except for batoclimab, there was no statistically significant difference in the 
      reduction of patient QMGS for the remaining targeted agents compared with 
      placebo. With regards to the secondary outcomes, only batoclimab (RR, 0.19; 95% 
      CrI, 0, 0.97) led to a significant reduction in the incidence of AEs when 
      compared with the placebo. Belimumab (RR, 0.85; 95% CrI, 0.57, 1.19), CFZ533 (RR, 
      0.95; 95% CrI, 0.72, 1.25), eculizumab (RR, 0.99; 95% CrI, 0.85, 1.21), and 
      efgartigimod (RR, 0.93; 95% CrI, 0.76, 1.15) also led to a lower incidence of 
      AEs, although these effects were not significantly different from the placebo. 
      CONCLUSIONS: Batoclimab had the best efficacy and safety for the treatment of gMG 
      and was ranked first out of the 10 targeted drugs included in this study. 
      Eculizumab was ranked second, and nipocalimab had the worst efficacy. With the 
      exception of batoclimab, the incidence of AEs for the remaining drugs was not 
      statistically significantly different from placebo. We note, however, that wide 
      CrIs reflect the uncertainty in this analysis owing to the small number of 
      available studies and low numbers of study participants; moreover, batoclimab had 
      the widest CrI of all drugs in this analysis. More well-designed studies with 
      long-term follow-up are needed to further evaluate and compare the efficacy and 
      safety of these drugs in the future.
CI  - (c) 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.
FAU - Ma, Yongbo
AU  - Ma Y
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China.
FAU - Nie, Xiangtao
AU  - Nie X
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China.
FAU - Zhu, Geke
AU  - Zhu G
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China.
FAU - Qi, Wenjing
AU  - Qi W
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China.
FAU - Hao, Lei
AU  - Hao L
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China. 
      haolei1102@163.com.
FAU - Guo, Xiuming
AU  - Guo X
AD  - Department of Neurology, The First Affiliated Hospital of Chongqing Medical 
      University, 1 Youyi Road, Yuzhong District, Chongqing, 400016, China. 
      xmguo813@163.com.
LA  - eng
PT  - Meta-Analysis
PT  - Systematic Review
DEP - 20240201
PL  - New Zealand
TA  - CNS Drugs
JT  - CNS drugs
JID - 9431220
SB  - IM
MH  - Humans
MH  - Network Meta-Analysis
MH  - *Drug Delivery Systems
MH  - *Myasthenia Gravis/drug therapy
EDAT- 2024/02/01 12:43
MHDA- 2024/02/22 06:42
CRDT- 2024/02/01 11:18
PHST- 2024/01/08 00:00 [accepted]
PHST- 2024/02/22 06:42 [medline]
PHST- 2024/02/01 12:43 [pubmed]
PHST- 2024/02/01 11:18 [entrez]
AID - 10.1007/s40263-024-01062-7 [pii]
AID - 10.1007/s40263-024-01062-7 [doi]
PST - ppublish
SO  - CNS Drugs. 2024 Feb;38(2):93-104. doi: 10.1007/s40263-024-01062-7. Epub 2024 Feb 
      1.