散发性和冯·希佩尔-林道病患者脊髓血管母细胞瘤手术后的局部肿瘤控制和神经学结果：一项多中心研究
Local tumor control and neurological outcomes after surgery for spinal hemangioblastomas in sporadic and von Hippel-Lindau disease: A multicenter study.
作者信息
Wach Johannes, Basaran Alim Emre, Vychopen Martin, Tihan Tarik, Wostrack Maria, Butenschoen Vicki M, Meyer Bernhard, Siller Sebastian, Schmidt Nils Ole, Onken Julia, Vajkoczy Peter, Santos Alejandro N, Rauschenbach Laurèl, Dammann Philipp, Sure Ulrich, Klingler Jan-Helge, Doria-Medina Roberto, Beck Jürgen, Blaß Bianca-Ioana, Gizaw Christine Julia, Hohenhaus Romina, Krieg Sandro, Alhalabi Obada T, Klein Lukas, Thomé Claudius, Kögl Nikolaus, Kunert Przemyslaw, Czernicki Tomasz, Pantel Tobias, Middelkamp Maximilian, Eicker Sven Oliver, Kattaa Ahed H, Park David J, Chang Steven D, Kilinc Fatma, Czabanka Marcus, Güresir Erdem
机构信息
Comprehensive Cancer Center Central Germany, Partner Site Leipzig, Leipzig, Germany.
Department of Neurosurgery, University Hospital Leipzig, Leipzig, Germany.
出版信息
Neuro Oncol. 2025 Jul 30;27(6):1567-1578. doi: 10.1093/neuonc/noaf041.
BACKGROUND
Spinal hemangioblastomas (sHBs) are rare vascular tumors with significant neurological implications. Their management, particularly in von Hippel-Lindau (VHL) disease, remains challenging due to recurrence and functional decline. Timely identification and intervention are critical for optimal outcomes.
METHODS
This international, multicenter retrospective cohort study included 357 patients (199 VHL-associated, 158 sporadic) from 13 neuro-oncological centers. Clinical and imaging data were analyzed to assess progression-free survival (PFS) and functional outcomes using the modified McCormick Scale (mMCS) at 12 months. Secondary analyses identified factors associated with VHL disease in sHBs.
RESULTS
Complete resection was achieved in 87.7% of cases, leading to significantly improved PFS at 72 months (sporadic: 95.1%, VHL-associated: 91.1%; hazard ratio: 0.18, 95% CI: 0.08-0.4). Multivariable analysis identified predictors of unfavorable outcomes at 12 months: preoperative mMCS ≥2 (odds ratio [OR]: 5.17, P = .008), intramedullary tumor location (OR: 9.48, P = .01), and preoperative bleeding (OR: 31.12, P = .02). Factors independently associated with VHL disease in sHBs included non-cervical tumor location (OR: 2.08, P = .004), intramedullary growth (OR: 2.39, P < .001), and age <43 years (OR: 3.24, P < .001). Functional improvements were observed in most patients, particularly those with sporadic sHBs.
CONCLUSIONS
Complete surgical resection is essential for long-term tumor control and favorable functional outcomes in both sporadic and VHL-associated sHBs. Early intervention, particularly in mild symptomatic and progressive cases, before neurological deterioration or hemorrhage, optimizes recovery. This study, the largest of its kind in a multicentric international setting, provides robust evidence to guide the management of both sporadic and VHL-associated sHBs.
背景
脊髓血管母细胞瘤（sHBs）是罕见的血管性肿瘤，具有重大的神经学意义。由于复发和功能衰退，其治疗，尤其是在冯·希佩尔-林道（VHL）病中的治疗，仍然具有挑战性。及时识别和干预对于获得最佳结果至关重要。
方法
这项国际多中心回顾性队列研究纳入了来自13个神经肿瘤中心的357例患者（199例与VHL相关，158例散发性）。分析临床和影像数据，使用改良的麦考密克量表（mMCS）评估12个月时的无进展生存期（PFS）和功能结果。二次分析确定了与sHBs中VHL病相关的因素。
结果
87.7%的病例实现了完全切除，使72个月时的PFS显著改善（散发性：95.1%，VHL相关：91.1%；风险比：0.18，95%CI：0.08 - 0.4）。多变量分析确定了12个月时不良结果的预测因素：术前mMCS≥2（比值比[OR]：5.17，P = 0.008）、髓内肿瘤位置（OR：9.48，P = 0.01）和术前出血（OR：31.12，P = 0.02）。sHBs中与VHL病独立相关的因素包括非颈椎肿瘤位置（OR：2.08，P = 0.004）、髓内生长（OR：2.39，P < 0.001）和年龄<43岁（OR：3.24，P < 0.001）。大多数患者，尤其是散发性sHBs患者，观察到功能改善。
结论
对于散发性和VHL相关的sHBs，完整的手术切除对于长期肿瘤控制和良好的功能结果至关重要。早期干预，尤其是在轻度症状性和进展性病例中，在神经功能恶化或出血之前进行，可优化恢复。这项在多中心国际环境中同类研究中规模最大的研究，为指导散发性和VHL相关sHBs的治疗提供了有力证据。
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