PMID- 32602752
OWN - NLM
STAT- MEDLINE
DCOM- 20210222
LR  - 20210222
IS  - 1744-7682 (Electronic)
IS  - 1471-2598 (Linking)
VI  - 20
IP  - 9
DP  - 2020 Sep
TI  - Eculizumab for the treatment of myasthenia gravis.
PG  - 991-998
LID - 10.1080/14712598.2020.1786530 [doi]
AB  - INTRODUCTION: Acetylcholine receptor antibody-positive generalized myasthenia 
      gravis (gMG) is effectively treated with symptomatic and immunosuppressive drugs 
      but a proportion of patients has a persistent disease and severe adverse events 
      (AEs). The unmet medical needs are specific immunosuppression and AE lowering. 
      Eculizumab blocks C5 protecting neuromuscular junction from the destructive 
      autoantibody effects. Phase II (Study C08-001) and III (ECU-MG-301) studies, with 
      the open-label extension (ECU-MG-302), demonstrated eculizumab efficacy and 
      safety in refractory gMG patients. AREAS COVERED: We provide an overview of 
      eculizumab biological features, clinical efficacy, and safety in gMG patients, 
      highlighting our perspective on the drug positioning in the MG treatment 
      algorithm. EXPERT OPINION: Eculizumab has the potential to significantly change 
      the immunosuppressive approach in gMG offering the opportunity to avoid or delay 
      corticosteroids' use due to its speed and selective mechanism of action. 
      Eculizumab prescription will depend on: 1. ability to modify the natural disease 
      course; 2. sustainability in the clinical practice (cost/effectiveness ratio); 3. 
      drug-induced AE reduction. At present we are missing a controlled study on its 
      use as a first-line treatment. We think that immunosuppression in MG will change 
      significantly in the next years by adopting more focused 'Precision Medicine' 
      approaches, and Eculizumab seems to satisfy such a promise.
FAU - Mantegazza, Renato
AU  - Mantegazza R
AD  - Neurology IV Unit ‒ Neuroimmunology and Neuromuscular Diseases, Fondazione 
      I.R.C.C.S. Istituto Neurologico Carlo Besta , Milan, Italy.
FAU - Cavalcante, Paola
AU  - Cavalcante P
AD  - Neurology IV Unit ‒ Neuroimmunology and Neuromuscular Diseases, Fondazione 
      I.R.C.C.S. Istituto Neurologico Carlo Besta , Milan, Italy.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20200630
PL  - England
TA  - Expert Opin Biol Ther
JT  - Expert opinion on biological therapy
JID - 101125414
RN  - 0 (Antibodies, Monoclonal, Humanized)
RN  - 0 (Complement C5)
RN  - 0 (Complement Inactivating Agents)
RN  - A3ULP0F556 (eculizumab)
SB  - IM
MH  - Antibodies, Monoclonal, Humanized/immunology/metabolism/*therapeutic use
MH  - Clinical Trials as Topic
MH  - Complement C5/immunology
MH  - Complement Inactivating Agents/immunology/metabolism/*therapeutic use
MH  - Half-Life
MH  - Humans
MH  - Myasthenia Gravis/*drug therapy/pathology
MH  - Treatment Outcome
OTO - NOTNLM
OT  - Biological drugs
OT  - complement system
OT  - eculizumab
OT  - myasthenia gravis
EDAT- 2020/07/01 06:00
MHDA- 2021/02/23 06:00
CRDT- 2020/07/01 06:00
PHST- 2020/07/01 06:00 [pubmed]
PHST- 2021/02/23 06:00 [medline]
PHST- 2020/07/01 06:00 [entrez]
AID - 10.1080/14712598.2020.1786530 [doi]
PST - ppublish
SO  - Expert Opin Biol Ther. 2020 Sep;20(9):991-998. doi: 
      10.1080/14712598.2020.1786530. Epub 2020 Jun 30.