OBJECTIVEPostoperative neurological deficits impair the overall outcome of revascularization surgery for patients with moyamoya disease (MMD). dl-3-n-butylphthalide (NBP) is approved for the treatment of ischemic stroke in China. This pilot study evaluated the effect of NBP on perioperative stroke and neurological deficits in patients with MMD.METHODSThe authors studied cases in which patients underwent combined revascularization surgery for MMD at their institution, with or without NBP administration. The overall study group included 164 patients (213 surgically treated hemispheres), including 49 patients who received NBP (25 mg twice daily) for 7 postoperative days. The incidence of perioperative stroke and transient neurological deficit (TND) and the severity of neurological deficits were compared between 49 propensity score-matched case pairs with or without NBP treatment. Subgroup analyses by type of onset and preoperative neurological status were also performed to determine specific characteristics of patients who might benefit from NBP administration.RESULTSIn the overall cohort, baseline characteristics differed with respect to preoperative stroke and modified Rankin Scale (mRS) score between patients who received NBP and those who did not receive it. In the 49 propensity score-matched pairs, postoperative stroke was observed in 11 patients and TND occurred in 21 patients, with no significant difference in incidence between the 2 groups. However, the TND was less severe in the NBP-treated group (p = 0.01). At 1 month after surgery, the neurological outcome was more favorable (p = 0.001) and the disability-free recovery rate was higher in patients with NBP treatment (p < 0.001). The number of patients who experienced an improved neurological function, compared to preoperative function, as measured by mRS, was greater in the NBP group than in the no-NBP group (p < 0.001). Multivariable analysis revealed that NBP administration was associated with decreased severity of TND (OR 0.28, p = 0.02), improved neurological function (OR 65.29, p = 0.04), and lower postoperative mRS score (OR 0.06, p < 0.001). These beneficial effects of NBP remained significant in ischemic type MMD and patients with preoperative mRS scores of 2 or greater.CONCLUSIONSPostoperative administration of NBP may alleviate perioperative neurological deficits after revascularization surgery for MMD, especially in patients with ischemic MMD and unfavorable preoperative status. The results of this study suggest that randomized controlled trials to assess the potential benefit of NBP in patients with MMD may be warranted.