Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis DOI Creative Commons
Abdullah Ashraf Hamad, Ibraheem M. Alkhawaldeh, Abdulqadir J. Nashwan

и другие.

Neurological Sciences, Год журнала: 2025, Номер unknown

Опубликована: Янв. 17, 2025

Abstract Objective Tofersen, an antisense oligonucleotide, has recently received FDA and EMA approval for treating amyotrophic lateral sclerosis (ALS) in adults with SOD1 gene mutations. This systematic review meta-analysis synthesized evidence on tofersen’s safety efficacy patients -related ALS. Methods A comprehensive search of three databases was conducted from inception through October 2024. Eligible studies included clinical trials, observational studies, case studies. Meta-analyses were using a random-effects model RevMan. Results Twelve involving 195 treated tofersen met the inclusion criteria, comprising two randomized controlled trials (RCTs), five cohort one series, four reports. Tofersen demonstrated promising effects, notably reducing levels cerebrospinal fluid neurofilament light chain (NfL) plasma, biomarker strongly correlated ALS progression survival. Meta-analysis RCTs showed significantly lower rate decline Functional Rating Scale-Revised (ALSFRS-R) scores baseline group compared to placebo (SMD = 0.44, 95% CI [0.05 0.83], P 0.03) significant reduction predicted Slow Vital Capacity ( 0.005). In pre-post decrease (ALSFRS-R rate) observed (MD -0.28, [-0.40 -0.15], < 0.0001). Reported adverse events consistent or procedural effects. Conclusion Current suggests that effectively reduces NfL slow disease ALS, showing promise as targeted therapeutic option.

Язык: Английский

Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis DOI Creative Commons
Abdullah Ashraf Hamad, Ibraheem M. Alkhawaldeh, Abdulqadir J. Nashwan

и другие.

Neurological Sciences, Год журнала: 2025, Номер unknown

Опубликована: Янв. 17, 2025

Abstract Objective Tofersen, an antisense oligonucleotide, has recently received FDA and EMA approval for treating amyotrophic lateral sclerosis (ALS) in adults with SOD1 gene mutations. This systematic review meta-analysis synthesized evidence on tofersen’s safety efficacy patients -related ALS. Methods A comprehensive search of three databases was conducted from inception through October 2024. Eligible studies included clinical trials, observational studies, case studies. Meta-analyses were using a random-effects model RevMan. Results Twelve involving 195 treated tofersen met the inclusion criteria, comprising two randomized controlled trials (RCTs), five cohort one series, four reports. Tofersen demonstrated promising effects, notably reducing levels cerebrospinal fluid neurofilament light chain (NfL) plasma, biomarker strongly correlated ALS progression survival. Meta-analysis RCTs showed significantly lower rate decline Functional Rating Scale-Revised (ALSFRS-R) scores baseline group compared to placebo (SMD = 0.44, 95% CI [0.05 0.83], P 0.03) significant reduction predicted Slow Vital Capacity ( 0.005). In pre-post decrease (ALSFRS-R rate) observed (MD -0.28, [-0.40 -0.15], < 0.0001). Reported adverse events consistent or procedural effects. Conclusion Current suggests that effectively reduces NfL slow disease ALS, showing promise as targeted therapeutic option.

Язык: Английский

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