PMID- 35511347
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220801
IS  - 2193-8253 (Print)
IS  - 2193-6536 (Electronic)
IS  - 2193-6536 (Linking)
VI  - 11
IP  - 3
DP  - 2022 Sep
TI  - The European Lambert-Eaton Myasthenic Syndrome Registry: Long-Term Outcomes 
      Following Symptomatic Treatment.
PG  - 1071-1083
LID - 10.1007/s40120-022-00354-8 [doi]
AB  - INTRODUCTION: Lambert-Eaton myasthenic syndrome (LEMS) is characterized by 
      autoantibodies against voltage-gated calcium channels (VGCC) at the neuromuscular 
      junction causing proximal muscle weakness, decreased tendon reflexes, and 
      autonomic changes. The European LEMS registry aimed to collate observational 
      safety data for 3,4-diaminopyridine phosphate (3,4-DAPP) and examine long-term 
      outcomes for patients with LEMS. METHODS: Thirty centers across four countries 
      participated in the non-interventional European LEMS registry. Any patients 
      diagnosed with LEMS by means of clinical assessment and abnormal 
      neurophysiological testing, or clinical assessment and positive for VGCC 
      antibodies were eligible to participate. Patients were monitored using standard 
      assessments for LEMS-related clinical manifestations. RESULTS: Among 96 evaluable 
      participants, 50 (52.1%) were being treated with 3,4-DAPP, 21 (21.9%) with 
      3,4-diaminopyridine (3,4-DAP), and 25 (26.0%) with other treatments (e.g., 
      pyridostigmine, corticosteroids, immunoglobulins, and azathioprine); 74 
      participants (77.1%) were exposed to 3,4-DAPP at any time. Quantitative 
      myasthenia gravis scores were similar across treatment groups. Muscle strength 
      was generally good and maintained during follow-up. Cerebellar ataxia, defined as 
      a negative Romberg's test and at least one other positive ataxia test, was 
      observed in 30 (56.6%) patients. Most participants had reduced reflex tone and 
      limited functioning. Sustained or improved functioning was observed in 
      participants administered 3,4-DAPP. Inconsistent and sporadic functional 
      improvement and regression was observed with 3,4-DAP and other treatments. 
      Fifty-five treatment-related adverse events (AEs) were reported by 32 (33.3%) 
      participants. Eight (8.3%) participants reported nine treatment-related serious 
      AEs. No new safety signals were identified. CONCLUSION: No new safety signals 
      were observed following long-term management of LEMS with 3,4-DAPP.
CI  - (c) 2022. The Author(s).
FAU - Meisel, Andreas
AU  - Meisel A
AUID- ORCID: 0000-0001-7233-5342
AD  - Charite Universitatsmedizin Berlin, Chariteplatz 1, 10117, Berlin, Germany. 
      andreas.meisel@charite.de.
FAU - Sieb, Jorn P
AU  - Sieb JP
AD  - HELIOS Hanseklinikum Stralsund, Stralsund, Germany.
FAU - Le Masson, Gwendal
AU  - Le Masson G
AD  - Hopital Pellegrin, Bordeaux, France.
FAU - Postila, Ville
AU  - Postila V
AD  - SERB SA, Brussels, Belgium.
FAU - Sacconi, Sabrina
AU  - Sacconi S
AD  - Service Systeme Nerveux Peripherique et Muscles, Centre Hospitalier Universitaire 
      de Nice, Universite Cote d'Azur, Nice, France.
LA  - eng
PT  - Journal Article
DEP - 20220505
PL  - New Zealand
TA  - Neurol Ther
JT  - Neurology and therapy
JID - 101637818
PMC - PMC9338181
OTO - NOTNLM
OT  - Amifampridine
OT  - Lambert-Eaton myasthenic syndrome
OT  - Observational study
OT  - Paraneoplastic
OT  - Quality of life
OT  - Real world
OT  - Safety
EDAT- 2022/05/06 06:00
MHDA- 2022/05/06 06:01
PMCR- 2022/05/05
CRDT- 2022/05/05 12:00
PHST- 2022/02/23 00:00 [received]
PHST- 2022/04/07 00:00 [accepted]
PHST- 2022/05/06 06:00 [pubmed]
PHST- 2022/05/06 06:01 [medline]
PHST- 2022/05/05 12:00 [entrez]
PHST- 2022/05/05 00:00 [pmc-release]
AID - 10.1007/s40120-022-00354-8 [pii]
AID - 354 [pii]
AID - 10.1007/s40120-022-00354-8 [doi]
PST - ppublish
SO  - Neurol Ther. 2022 Sep;11(3):1071-1083. doi: 10.1007/s40120-022-00354-8. Epub 2022 
      May 5.