Macrophage inclusions were more common in the cerebrospinal fluid of patients receiving tofersen than nusinersen
Original source
Macrophage inclusions in patients undergoing antisense oligonucleotide therapy for ALS or SMA: A retrospective and transversal study. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Related topics
A study found persistent macrophage inclusions in cerebrospinal fluid samples from most patients receiving tofersen for SOD1-associated amyotrophic lateral sclerosis (ALS). The inclusions were rare and temporary in patients receiving nusinersen for spinal muscular atrophy (SMA). Their presence was associated with cerebrospinal fluid inflammation, but patients with inclusions still showed favorable clinical responses.
Why this matters
The finding may help clinicians interpret cerebrospinal fluid results in people receiving tofersen or nusinersen. However, the inclusions’ origin and clinical significance remain unclear, and this study does not establish a need to change treatment or show that the inclusions cause benefit or harm.
Limitations and context
This was a retrospective, cross-sectional study involving 21 patients and 132 cerebrospinal fluid samples; inflammatory and clinical correlations were assessed in 18 patients. The cellular nature of the inclusions was not determined. The findings describe an association and do not establish causation or long-term treatment effects.