The abstract has been submitted for presentation to the AANS 2026 meeting being held in San Antonio, TX, USA.Introduction:Oligodendrogliomas are an uncommon subset of gliomas that are molecularly defined by 1p/19q codeletion in the setting of an isocitrate dehydrogenase (IDH) 1/2 mutation. Standard-of-care management involves maximal safe resection followed by adjuvant chemoradiation with procarbazine, lomustine, and vincristine (PCV). Although PCV confers a durable survival advantage, treatment-limiting toxicity is common and often necessitates discontinuation. IDH inhibitors such as vorasidenib have demonstrated promising efficacy and more favorable tolerability profiles, but a paucity of comparative data across therapeutic classes limits optimal treatment decision-making.Methods:A systematic search was conducted through to 7 March 2025 in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). Eligible studies included adult patients (≥18 years) with IDH-mutant, 1p/19q-codeleted oligodendrogliomas treated with PCV chemotherapy or IDH inhibitors and with a minimum follow-up of 12 months. Outcomes of interest included overall survival (OS), progression-free survival (PFS), and grade ≥ 3 adverse events (AEs) that led to treatment discontinuation.Results:Twenty-eight studies met the inclusion criteria, with a total of 406 patients. All 406 patients carried a confirmed diagnosis of oligodendroglioma. For mixed-histology cohorts, only oligodendroglioma-specific data were extracted and analyzed. Among PCV cohorts, median PFS ranged from 24.3 months to 8.4 years and median OS was reported up to 14.7 years in long-term follow-up from RTOG 9402 and EORTC 26951. Grade ≥ 3 AEs resulted in treatment discontinuation in 65–70% of patients, primarily due to hematologic or neurologic events. In comparison, vorasidenib achieved a median PFS of 27.7 months in the phase III INDIGO trial (HR 0.39; 95% CI 0.27–0.56;p< 0.001), with median OS not yet reached at 14.2 months of follow-up. Grade ≥ 3 AEs occurred in 22.8% of patients and led to treatment discontinuation in only 1–3%, primarily due to asymptomatic transaminitis. Early real-world data from expanded-access programs similarly support these tolerability findings.Conclusions:While PCV chemotherapy remains the standard-of-care systemic therapy for oligodendroglioma supported by mature survival data, IDH inhibitors represent a mechanistically targeted alternative with encouraging early-phase outcomes and a significantly improved safety profile. Direct comparison across these regimens is constrained by differences in study design and limited long-term OS data for IDH inhibitors. Prospective head-to-head trials are essential for defining the optimal therapeutic sequence in this evolving treatment landscape. In the interim, we provide a recommend approach for current use.
本摘要已提交至将于美国德克萨斯州圣安东尼奥举行的2026年美国神经外科学会年会。 引言：少突胶质细胞瘤是胶质瘤中较少见的亚型，其分子定义为在异柠檬酸脱氢酶（IDH）1/2突变背景下存在1p/19q共缺失。标准治疗方案包括最大范围安全切除术后联合丙卡巴肼、洛莫司汀和长春新碱（PCV）方案的放化疗。尽管PCV方案能带来持久的生存获益，但其治疗限制性毒性常见，常导致治疗中止。IDH抑制剂（如伏西地尼）已显示出良好的疗效和更优的耐受性，但不同治疗类别间比较数据的缺乏限制了最佳治疗决策的制定。 方法：根据范围综述系统评价与荟萃分析报告规范扩展版（PRISMA-ScR）要求，截至2025年3月7日进行了系统性文献检索。符合条件的研究需纳入接受PCV化疗或IDH抑制剂治疗、经确诊为IDH突变型1p/19q共缺失少突胶质细胞瘤的成年患者（≥18岁），且随访时间至少12个月。关注结局指标包括总生存期（OS）、无进展生存期（PFS）以及导致治疗中止的≥3级不良事件（AEs）。 结果：共28项研究符合纳入标准，总计406例患者。所有患者均经病理确诊为少突胶质细胞瘤。对于混合组织学队列，仅提取并分析了少突胶质细胞瘤特异性数据。在PCV治疗队列中，中位PFS范围为24.3个月至8.4年；根据RTOG 9402和EORTC 26951的长期随访报告，中位OS最长可达14.7年。65-70%的患者因≥3级AEs（主要为血液学或神经系统事件）中止治疗。相比之下，伏西地尼在III期INDIGO试验中达到27.7个月的中位PFS（HR 0.39；95% CI 0.27–0.56；p<0.001），中位OS在14.2个月随访期内尚未达到。22.8%的患者出现≥3级AEs，但仅1-3%因此中止治疗，主要原因为无症状性转氨酶升高。扩大可及项目的早期真实世界数据同样支持该耐受性结论。 结论：尽管PCV化疗凭借成熟的生存数据仍是少突胶质细胞瘤的标准全身治疗方案，IDH抑制剂作为机制靶向的替代疗法，展现出令人鼓舞的早期疗效和显著改善的安全性特征。研究设计差异及IDH抑制剂长期OS数据的缺乏限制了不同方案间的直接比较。在当前快速演变的治疗格局中，前瞻性头对头试验对于确立最佳治疗顺序至关重要。基于现有证据，我们提出了当前临床应用的推荐方案。
肿瘤(癌症)患者之家