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000280914 1001_ $$0P:(DE-2719)9000485$$aBernsen, Sarah$$b0$$eFirst author$$udzne
000280914 245__ $$aSerum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen-Treated ALS.
000280914 260__ $$aNew York, NY [u.a.]$$bWiley$$c2025
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000280914 520__ $$aCardiac 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%) patients treated with tofersen experienced a reduction in cTnT levels.In this study, we describe a response signal of cTnT to tofersen treatment, which supports the value of cTnT as an independent biomarker in ALS. These results contribute to the notion that cTnT may provide additional value as a progression and treatment response biomarker in ALS complementary to NfL and warrant further investigation.
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000280914 650_7 $$2Other$$aSOD1
000280914 650_7 $$2Other$$aamyotrophic lateral sclerosis
000280914 650_7 $$2Other$$abiomarker
000280914 650_7 $$2Other$$atofersen
000280914 650_7 $$2Other$$atroponin
000280914 650_7 $$2NLM Chemicals$$aTroponin T
000280914 650_7 $$2NLM Chemicals$$aBiomarkers
000280914 650_7 $$2NLM Chemicals$$aOligonucleotides
000280914 650_7 $$2NLM Chemicals$$aNeurofilament Proteins
000280914 650_7 $$0EC 1.15.1.1$$2NLM Chemicals$$aSuperoxide Dismutase-1
000280914 650_7 $$0EC 2.7.3.2$$2NLM Chemicals$$aCreatine Kinase
000280914 650_7 $$2NLM Chemicals$$aneurofilament protein L
000280914 650_2 $$2MeSH$$aHumans
000280914 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: blood
000280914 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: drug therapy
000280914 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: genetics
000280914 650_2 $$2MeSH$$aTroponin T: blood
000280914 650_2 $$2MeSH$$aMale
000280914 650_2 $$2MeSH$$aFemale
000280914 650_2 $$2MeSH$$aMiddle Aged
000280914 650_2 $$2MeSH$$aAged
000280914 650_2 $$2MeSH$$aBiomarkers: blood
000280914 650_2 $$2MeSH$$aOligonucleotides: therapeutic use
000280914 650_2 $$2MeSH$$aTreatment Outcome
000280914 650_2 $$2MeSH$$aNeurofilament Proteins
000280914 650_2 $$2MeSH$$aSuperoxide Dismutase-1: genetics
000280914 650_2 $$2MeSH$$aCohort Studies
000280914 650_2 $$2MeSH$$aAdult
000280914 650_2 $$2MeSH$$aCreatine Kinase: blood
000280914 7001_ $$aFabian, Rachel$$b1
000280914 7001_ $$aKoc, Yasemin$$b2
000280914 7001_ $$aSchumann, Peggy$$b3
000280914 7001_ $$0P:(DE-2719)2812030$$aKörtvélyessy, Peter$$b4$$udzne
000280914 7001_ $$0P:(DE-2719)9002230$$aCastro-Gomez, Mario Sergio$$b5$$udzne
000280914 7001_ $$00000-0002-2736-7350$$aMeyer, Thomas$$b6
000280914 7001_ $$0P:(DE-2719)9001116$$aWeydt, Patrick$$b7$$eLast author$$udzne
000280914 773__ $$0PERI:(DE-600)1476641-3$$a10.1002/mus.28453$$gVol. 72, no. 3, p. 509 - 514$$n3$$p509 - 514$$tMuscle & nerve$$v72$$x0148-639X$$y2025
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