Nimustine Hydrochloride

Temozolomide combined with radiotherapy has been widely recognized as the first-line treatment for glioblastoma. However, a standard treatment regimen for recurrent glioblastoma after treatment with temozolomide plus radiotherapy is lacking at present. This network meta-analysis aimed to assess and rank the therapeutic efficacy and safety of temozolomide plus radiotherapy combined with 11 other anti-cancer treatments in glioblastoma so as to provide valuable evidence for appropriate clinical decision-making.

Eligible studies investigating the efficacy and safety of temozolomide plus radiotherapy (standard treatment) compared to combination therapies were obtained by searching PubMed, Cochrane Library, and EMBASE. Outcomes included progression-free survival, overall survival, and grade 3-5 adverse events.

A total of 16 studies were included in this meta-analysis. Except for autologous lymphoid effector cells specific against tumor, galunisertib, and interferon-β, other anti-cancer treatments used in combination with standard treatment resulted in a survival advantage over standard treatment alone. The combination of nimustine with standard treatment performed best in improving overall survival and progression-free survival and exhibited a favorable safety profile, making it the optimal treatment option for recurrent glioblastoma.

After a comprehensive safety analysis, nimustine and lomustine were identified as effective drugs that could be combined with standard treatment.

www.crd.york.ac.uk/prospero identifier is CRD420251004329.

Diffuse intrinsic pontine glioma (DIPG) is a very challenging‐to‐treat pediatric malignant tumor, with a median survival time of < 12 months. Convection‐enhanced delivery (CED) allows for direct drug administration into the tumor site, showing potential as a novel therapeutic approach. This study evaluated the efficacy of CED of nimustine hydrochloride (ACNU) in children with DIPG. This phase 2, single‐arm, multicenter study enrolled patients aged 3–21 years and diagnosed with DIPG. The investigational treatment commenced 1 month after completing radiotherapy (local 50–60 Gy). The treatment involved stereotactic brain surgery for catheter placement, followed by ACNU administration via a CED catheter at a concentration of 0.75 mg/mL for 2–3 days until a cumulative dose of 7 (±0.3) mL was achieved. The primary endpoint was the 1‐year survival rate. From April 2018 to March 2020, 21 children were enrolled in the trial and treated, with 20 evaluable for the primary endpoint. The 1‐year survival rate from the start of radiotherapy was 60%, and the median survival time was 15 months. The response rate was analyzed in 20 patients, with one complete response (CR), six partial responses (PR), nine stable diseases, and four progressive diseases, resulting in a response rate of 35% (CR + PR). The CED of ACNU in the brainstem of children with DIPG after radiotherapy appears to be an effective therapeutic strategy. This approach warrants further development as a treatment for children with DIPG. This study is registered with jRCT (No. jRCT2021190003).