Impact of DL-3-n-Butylphthalide on Progression in Alzheimer's Disease: A Retrospective Cohort Analysis

To evaluate the efficacy of DL-3-n-butylphthalide (NBP), a synthetic compound that has shown neuroprotective effects, on cognitive function, psychiatric-behavioral symptoms, and daily activities in patients with Alzheimer's disease (AD).

This retrospective cohort study included patients with AD treated with or without NBP. Disease deterioration and decline were defined by changes in Clinical Dementia Rating-Sum of Boxes (CDR-SB) over six months. Multivariate logistic regression, inverse probability of treatment weighting (IPTW) and overlap-weighted propensity score matching (PSM) were used to adjust for confounding.

Totally 100 were included in this study, with 39 classified as the NBP group and 61 as the non-NBP group. NBP was associated with lower odds of deterioration (adjusted odds ratio [OR] = 0.19, 95% confidence interval [CI]: 0.04-0.88, p = 0.034) and decline (adjusted OR = 0.10, 95% CI: 0.03-0.49, p = 0.001). In IPTW and PSM analyses, deterioration occurred in 4.31% vs 22.10% and 4.06% vs 24.27%, and decline in 4.31% vs 39.38% and 4.06% vs 44.28% for the NBP and non-NBP groups, respectively.

NBP was associated with reduced risks of clinical worsening and helped preserve cognitive and behavioral functions in patients with AD. These results highlight the potential of NBP as a promising therapeutic option in AD management. Future randomized controlled trials are necessary to validate these findings and assess the long-term efficacy of NBP in clinical settings.

This real-world study suggests that NBP may slow disease progression and preserve cognitive and behavioral function in AD.