TY  - JOUR
AU  - Bernsen, Sarah
AU  - Fabian, Rachel
AU  - Koc, Yasemin
AU  - Schumann, Peggy
AU  - Körtvélyessy, Peter
AU  - Castro-Gomez, Mario Sergio
AU  - Meyer, Thomas
AU  - Weydt, Patrick
TI  - Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen-Treated ALS.
JO  - Muscle & nerve
VL  - 72
IS  - 3
SN  - 0148-639X
CY  - New York, NY [u.a.]
PB  - Wiley
M1  - DZNE-2025-00997
SP  - 509 - 514
PY  - 2025
AB  - Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)-ALS patients treated with the antisense oligonucleotide tofersen show a decrease in NfL. In this study, we assess cTnT levels in SOD1-ALS at baseline and during tofersen treatment.cTnT was analyzed at baseline and during tofersen treatment in 23 SOD1-ALS patients at two specialized ALS centers in Germany and compared to a control cohort of 74 ALS patients without SOD1 variants.cTnT levels increased in the control ALS cohort over time (p < 0.0001) but not in the tofersen group (p = 0.36). Creatine kinase (CK) and CK-MB levels did not show significant changes over time. The median monthly increase of cTnT was 0.045 points (IQR 0.02-0.08) in the control ALS cohort and 0.01 points (IQR -0.01-0.03) in the tofersen group (p = 0.0013). A significantly lower fold change in cTnT levels was observed in the tofersen-treated cohort (median 1.2; IQR 0.77-1.59) relative to the control group (median 1.89; IQR 1.35-2.75) (p = 0.0003). Nine (39
KW  - Humans
KW  - Amyotrophic Lateral Sclerosis: blood
KW  - Amyotrophic Lateral Sclerosis: drug therapy
KW  - Amyotrophic Lateral Sclerosis: genetics
KW  - Troponin T: blood
KW  - Male
KW  - Female
KW  - Middle Aged
KW  - Aged
KW  - Biomarkers: blood
KW  - Oligonucleotides: therapeutic use
KW  - Treatment Outcome
KW  - Neurofilament Proteins
KW  - Superoxide Dismutase-1: genetics
KW  - Cohort Studies
KW  - Adult
KW  - Creatine Kinase: blood
KW  - SOD1 (Other)
KW  - amyotrophic lateral sclerosis (Other)
KW  - biomarker (Other)
KW  - tofersen (Other)
KW  - troponin (Other)
KW  - Troponin T (NLM Chemicals)
KW  - Biomarkers (NLM Chemicals)
KW  - Oligonucleotides (NLM Chemicals)
KW  - Neurofilament Proteins (NLM Chemicals)
KW  - Superoxide Dismutase-1 (NLM Chemicals)
KW  - Creatine Kinase (NLM Chemicals)
KW  - neurofilament protein L (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:40491248
C2  - pmc:PMC12338008
DO  - DOI:10.1002/mus.28453
UR  - https://pub.dzne.de/record/280914
ER  -