Singhi Aatur D, Krasinskas Alyssa M, Choudry Haroon A, Bartlett David L, Pingpank James F, Zeh Herbert J, Luvison Alyssa, Fuhrer Kimberly, Bahary Nathan, Seethala Raja R, Dacic Sanja
Department of Pathology, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, GA, USA.
Mod Pathol. 2016 Jan;29(1):14-24. doi: 10.1038/modpathol.2015.121. Epub 2015 Oct 23.
Cytoreductive surgery and hyperthermic intraperitoneal chemoperfusion for patients with malignant peritoneal mesothelioma has resulted in improved disease control and increased survival. Despite these results, there are significant perioperative risks associated with this aggressive procedure that necessitate consideration of prognostic markers during patient selection. The molecular pathogenesis of peritoneal mesothelioma remains relatively unknown, but extrapolation of findings from their pleural counterpart would suggest frequent alterations in CDKN2A, NF2, and BAP1. Homozygous deletions in CDKN2A portend a worse overall survival in peritoneal mesothelioma. However, the prevalence and prognostic significance of NF2 and BAP1 abnormalities has not been studied. Dual-color fluorescence in situ hybridization using CDKN2A and NF2 locus-specific probes and BAP1 immunohistochemistry identified homozygous CDKN2A deletions (n=25, 29%), hemizygous NF2 loss (n=30, 35%), and/or loss of BAP1 protein expression (n=49, 57%) in 68 of 86 (79%) peritoneal mesotheliomas. Homozygous CDKN2A deletions or hemizygous NF2 loss correlated with shorter progression-free survival (P<0.02) and poor overall survival (P<0.03). Moreover, the significance of these findings was cumulative. Patients harboring both homozygous CDKN2A deletions and hemizygous NF2 loss had a 2-year progression-free survival rate of 9% with a median of 6 months (P<0.01) and overall survival rate of 18% with a median of 8 months (P<0.01). By multivariate analysis, combined homozygous CDKN2A deletions and hemizygous NF2 loss was a negative prognostic factor for both progression-free survival and overall survival, independent of patient age, peritoneal cancer index, completeness of cytoreduction, and extent of invasion. In contrast, loss of BAP1 was not associated with clinical outcome. In summary, homozygous deletions in CDKN2A and hemizygous loss of NF2 as detected by fluorescence in situ hybridization would confer a poor clinical outcome and may guide future treatment decisions for patients with peritoneal mesothelioma.
对于恶性腹膜间皮瘤患者,细胞减灭术及热灌注腹腔内化疗已改善了疾病控制并延长了生存期。尽管有这些结果,但这种积极的手术仍存在显著的围手术期风险,这使得在患者选择过程中需要考虑预后标志物。腹膜间皮瘤的分子发病机制仍相对不明,但从其胸膜对应物的研究结果推断,提示CDKN2A、NF2和BAP1常发生改变。CDKN2A的纯合缺失预示着腹膜间皮瘤患者的总生存期更差。然而,NF2和BAP1异常的发生率及预后意义尚未得到研究。使用CDKN2A和NF2基因座特异性探针的双色荧光原位杂交以及BAP1免疫组化在86例腹膜间皮瘤中的68例(79%)中检测到CDKN2A纯合缺失(n = 25,29%)、NF2半合子缺失(n = 30,35%)和/或BAP1蛋白表达缺失(n = 49,57%)。CDKN2A纯合缺失或NF2半合子缺失与无进展生存期缩短(P < 0.02)和总生存期较差(P < 0.03)相关。此外,这些发现的意义是累积性的。同时存在CDKN2A纯合缺失和NF2半合子缺失的患者2年无进展生存率为9%,中位生存期为6个月(P < 0.01),总生存率为18%,中位生存期为8个月(P < 0.01)。通过多变量分析,CDKN2A纯合缺失和NF2半合子缺失的联合是无进展生存期和总生存期的负性预后因素,独立于患者年龄、腹膜癌指数、细胞减灭的完整性及侵袭范围。相比之下,BAP1缺失与临床结局无关。总之,通过荧光原位杂交检测到的CDKN2A纯合缺失和NF2半合子缺失将导致不良临床结局,并可能指导腹膜间皮瘤患者未来的治疗决策。