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@ARTICLE{Bjelica:281844,
author = {Bjelica, Bogdan and Wohnrade, Camilla and Osmanovic, Alma
and Schreiber-Katz, Olivia and Koerner, Sonja and Kollewe,
Katja and Haeckl, Sebastian and Freigang, Maren and Cordts,
Isabell and Becker, Benedikt and Vogt, Charlotte and
Muhandes, Mohamad Tareq and Günther, René and Deschauer,
Marcus and Koch, Jan C and Tuerk, Matthias and Regensburger,
Martin and Neuwirth, Christoph and Petri, Susanne},
title = {{C}ompound {M}uscle {A}ction {P}otential ({CMAP})
{A}mplitude {T}rajectories and {P}attern in {A}dults with
5q-{S}pinal {M}uscular {A}trophy {R}eceiving {N}usinersen
{T}herapy: {A} {M}ulticenter, {B}inational {O}bservational
{S}tudy.},
journal = {European journal of neurology},
volume = {32},
number = {11},
issn = {1351-5101},
address = {Oxford [u.a.]},
publisher = {Wiley-Blackwell},
reportid = {DZNE-2025-01223},
pages = {e70405},
year = {2025},
abstract = {This study aimed to evaluate changes in compound muscle
action potential (CMAP) amplitude in adults with spinal
muscular atrophy (SMA) undergoing nusinersen treatment and
its association with motor function improvements.This
multicenter study assessed median, ulnar, and peroneal CMAP
over a follow-up of up to 4.5 years using linear mixed
models. Motor function was measured using the Revised Upper
Limb Module (RULM) and the Hammersmith Functional Motor
Scale Expanded (HFMSE). Correlations between CMAP and motor
function scores were analyzed.Seventy-eight patients (27
ambulatory, 51 non-ambulatory) were included. Baseline ulnar
CMAP ≥ 2.0 mV distinguished SMA type 3 from type 2 with
$91.3\%$ sensitivity and $88.9\%$ specificity (AUC 0.96,
$95\%$ CI 0.92-1.0), while baseline median nerve CMAP ≥
6.5 mV showed $91.7\%$ sensitivity and $77.3\%$ specificity
(AUC 0.84, $95\%$ CI 0.72-0.96). No significant changes over
time were observed in median, ulnar, and peroneal CMAP
amplitudes (p > 0.05). CMAP trajectories did not differ
between SMA types 2 and 3 (p > 0.05). No significant
difference in the change in RULM or HFMSE at any time point
was observed between SMA patients with baseline median nerve
CMAP < 5 mV and those with CMAP of ≥ 5 mV (p > 0.05). No
significant correlations were found between changes in
median nerve CMAP and HFMSE or RULM (p > 0.05).CMAP
amplitudes remained stable during nusinersen treatment, with
no differences in trajectories between SMA types 2 and 3.
Our findings suggest that while CMAP amplitude correlates
with disease severity, it may not serve as a sensitive
biomarker of treatment response in adult SMA patients.},
keywords = {Humans / Female / Oligonucleotides: therapeutic use /
Oligonucleotides: pharmacology / Male / Adult / Action
Potentials: drug effects / Action Potentials: physiology /
Middle Aged / Ulnar Nerve: physiopathology / Muscular
Atrophy, Spinal: drug therapy / Muscular Atrophy, Spinal:
physiopathology / Young Adult / Median Nerve:
physiopathology / Spinal Muscular Atrophies of Childhood:
drug therapy / Spinal Muscular Atrophies of Childhood:
physiopathology / Peroneal Nerve: physiopathology /
biomarker (Other) / compound muscle action potential (CMAP)
(Other) / neurophysiology (Other) / nusinersen (Other) /
spinal muscular atrophy (SMA) (Other) / nusinersen (NLM
Chemicals) / Oligonucleotides (NLM Chemicals)},
cin = {AG Falkenburger},
ddc = {610},
cid = {I:(DE-2719)1710012},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41174958},
pmc = {pmc:PMC12579276},
doi = {10.1111/ene.70405},
url = {https://pub.dzne.de/record/281844},
}