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Abstract Background Besides the hallmark of H3K27M mutation, aberrant amplifications of receptor tyrosine kinases (RTKs) are commonly observed in diffuse midline glioma (DMG), a highly malignant brain tumor with dismal prognosis. Here, we intended to evaluate the efficacy and safety of a multitarget RTK inhibitor anlotinib in patients with H3K27M‐DMG. Methods A total of 40 newly diagnosed H3K27M‐DMG patients including 15 with anlotinib and 25 without anlotinib treatment were retrospectively enrolled in this cohort. Progression‐free survival (PFS), overall survival (OS), and toxicities were assessed and compared. Results The median PFS and OS of all patients in this cohort were 8.5 months (95% CI, 6.5–11.3) and 15.5 months (95% CI, 12.6–17.1), respectively. According to the Response Assessment in Neuro‐Oncology (RANO) criteria, the disease control rate in the anlotinib group 93.3%, 95% confidence interval (CI), 70.2–98.8 was significantly higher than those without anlotinib (64%, 95% CI: 40.5–79.8, p = 0.039). The median PFS of patients with and without anlotinib was 11.6 months (95% CI, 7.8–14.3) and 6.4 months (95% CI, 4.3–10.3), respectively. Both the median PFS and OS of DMG patients treated with anlotinib were longer than those without anlotinib in the infratentorial patients (PFS: 10.3 vs. 5.4 months, p = 0.006; OS: 16.6 vs. 8.7 months, p = 0.016). Multivariate analysis also indicated anlotinib (HR: 0.243, 95% CI: 0.066–0.896, p = 0.034) was an independent prognosticator for longer OS in the infratentorial subgroup. In addition, the adverse events of anlotinib administration were tolerable in the whole cohort. Conclusions This study first reported that anlotinib combined with Stupp regimen is a safe and feasible regimen for newly diagnosed patients with H3K27M‐DMG. Further, anlotinib showed significant efficacy for H3K27M‐DMG located in the infratentorial region.
Liu et al. (Mon,) studied this question.