冯·希佩尔-林道病和散发性病例中颅内血管母细胞瘤立体定向放射外科治疗后的结果：一项国际多中心研究

Outcomes After Stereotactic Radiosurgery for Intracranial Hemangioblastoma in Von Hippel-Lindau Disease and Sporadic Cases: An International Multicenter Study.

作者信息

Shaaban Ahmed, Tos Salem M, Mantziaris Georgios, Pham Duy, Dayawansa Sam, Nabeel Ahmed M, Reda Wael A, Tawadros Sameh R, Karim Khaled Abdel, El-Shehaby Amr M N, Emad Reem M, Wei Zhishuo, McKendrick Lindsay M, Niranjan Ajay, Lunsford L Dade, Peker Selcuk, Samanci Yavuz, Liscak Roman, May Jaromir, Mathieu David, Lee Cheng-Chia, Yang Huai-Che, Dono Antonio, Blanco Angel I, Esquenazi Yoshua, Moreno Nuria Martinez, Álvarez Roberto Martinez, Picozzi Piero, Franzini Andrea, Tripathi Manjul, Sumi Takuma, Uzuka Takeo, Kano Hideyuki, Bailey David, Zacharia Brad E, Cifarelli Christopher P, Cifarelli Daniel T, Hack Joshua D, Speckter Herwin, Lazo Erwin, Warnick Ronald E, Schoenhals Jonathan E, Palmer Joshua D, Asthagiri Ashok R, Xu Zhiyuan, Sheehan Jason P

机构信息

Department of Neurological Surgery, University of Virginia Health, Charlottesville, Virginia, USA.

Gamma Knife Center Cairo, Nasser Institute Hospital, Cairo, Egypt.

出版信息

Neurosurgery. 2025 Jun 19. doi: 10.1227/neu.0000000000003579.

DOI:10.1227/neu.0000000000003579

PMID:40536334

Abstract

BACKGROUND AND OBJECTIVES

Hemangioblastomas (HGBs) are rare, benign, World Health Organization grade 1 vascular tumors, which are most commonly located in the cerebellum, and may occur sporadically or in association with von Hippel-Lindau (VHL) disease, a hereditary VHL-mutated tumor syndrome. Limited data are available regarding factors affecting outcomes after stereotactic radiosurgery (SRS). We aim to provide a contemporary evaluation of SRS for HGBs through an international, multicenter study. In this study, we assess local tumor control and SRS-related complications in patients with intracranial HGBs.

METHODS

A retrospective analysis from 17 centers was performed. Data on patient characteristics, SRS parameters, and outcomes were collected. The study included 104 patients with VHL and 89 sporadic cases, with 433 and 137 tumors, respectively. The median follow-up after the initial SRS was 52 months for patients with VHL and 44 months for sporadic cases.

RESULTS

At the last follow-up, tumor control was achieved in 85% of VHL tumors and 76% of sporadic tumors. Radiation-induced changes were identified in 13 (3.5%) VHL cases and 5 (3.8%) sporadic cases. The overall 3-year and 5-year cumulative incidences of tumor progression were 13% and 22% for all tumors, 14% and 25% for VHL, and 13% and 17% for sporadic cases. Tumor progression was more common in cystic than in solid tumors in the sporadic group. Overall survival probability and progression-free survival were better in VHL cases compared with the sporadic group. Older age at SRS, male sex, and multiple tumors were associated with reduced local tumor control in all tumors and in the VHL group, whereas a margin dose >15 Gy was associated with improved local tumor control in both groups.

CONCLUSION

SRS offers an effective treatment of intracranial HGBs, whether VHL-associated or sporadic, with a favorable risk profile. HGBs without a cystic component were more likely to be controlled after SRS in the sporadic group.

摘要

背景与目的

血管母细胞瘤（HGBs）是罕见的良性肿瘤，世界卫生组织分级为1级血管肿瘤，最常见于小脑，可散发发生或与遗传性VHL基因（VHL）突变肿瘤综合征——冯·希佩尔-林道（VHL）病相关。关于立体定向放射外科治疗（SRS）后影响预后因素的数据有限。我们旨在通过一项国际多中心研究对HGBs的SRS进行当代评估。在本研究中，我们评估颅内HGBs患者的局部肿瘤控制情况和SRS相关并发症。

方法

对17个中心进行回顾性分析。收集患者特征、SRS参数和预后的数据。该研究包括104例VHL病患者和89例散发病例，分别有433个和137个肿瘤。VHL病患者首次SRS后的中位随访时间为52个月，散发病例为44个月。

结果

在最后一次随访时，85%的VHL病相关肿瘤和76%的散发性肿瘤实现了肿瘤控制。在13例（3.5%）VHL病患者和5例（3.8%）散发病例中发现了放射性改变。所有肿瘤的3年和5年累积肿瘤进展发生率分别为13%和22%，VHL病相关肿瘤分别为14%和25%，散发病例分别为13%和17%。在散发性病例组中，囊性肿瘤比实性肿瘤更易发生肿瘤进展。与散发性病例组相比，VHL病患者的总生存概率和无进展生存期更好。SRS时年龄较大、男性以及肿瘤数量较多与所有肿瘤和VHL病组的局部肿瘤控制降低相关，而边缘剂量>15 Gy与两组的局部肿瘤控制改善相关。