Tripepi Marta, da Costa Ana G, Chi Dennis S, Lima Jorge, Casanova João
Department of Women and Children's Health, Clinic of Gynecology and Obstetrics, University of Padua, Padua, Italy.
Gynecologic Oncology Unit, Obstetrics and Gynecology Service, Department of Surgery, Hospital da Luz Lisboa, Lisbon, Portugal.
Front Oncol. 2025 Aug 15;15:1645361. doi: 10.3389/fonc.2025.1645361. eCollection 2025.
Small cell carcinoma of the ovary, hypercalcemic type (SCCOHT) is a rare and highly aggressive ovarian neoplasm, predominantly affecting young women, often in their second or third decade of life. Despite its distinctive clinical and pathological features, diagnosis is frequently delayed due to overlapping characteristics with other small round blue cell tumors. A hallmark of SCCOHT is the biallelic inactivation of the SMARCA4 gene, which leads to loss of BRG1 protein expression and disrupts epigenetic regulation via the SWI/SNF chromatin-remodeling complex. Unlike many other malignancies, SCCOHT exhibits low mutational burden and diploid karyotype, suggesting that epigenetic dysregulation, rather than genomic instability, is the underlying oncogenic mechanism. Clinically, SCCOHT often presents with nonspecific abdominal or pelvic symptoms and is uniquely associated with paraneoplastic hypercalcemia in up to two-thirds of cases. Diagnosis requires a combination of imaging, laboratory evaluation, histopathology, and immunohistochemistry. Treatment is not standardized but typically involves a multimodal approach, including radical surgery and platinum-based chemotherapy, often with multi-agent regimens. The role of radiotherapy is less well defined but may be considered for local control or palliation. Prognosis remains poor, with high recurrence rates and limited response to salvage therapy. Emerging molecular insights have prompted investigations into targeted therapies and immunotherapy, though clinical data are limited. Given the frequent presence of germline SMARCA4 mutations, genetic counseling is strongly recommended, and ongoing research is essential to improve diagnostic accuracy, personalize treatment, and enhance outcomes for this devastating malignancy.
卵巢高钙血症型小细胞癌(SCCOHT)是一种罕见且侵袭性很强的卵巢肿瘤,主要影响年轻女性,发病年龄通常在二十多岁或三十多岁。尽管其具有独特的临床和病理特征,但由于与其他小圆蓝细胞肿瘤有重叠特征,诊断常常延迟。SCCOHT的一个标志是SMARCA4基因的双等位基因失活,这导致BRG1蛋白表达缺失,并通过SWI/SNF染色质重塑复合物破坏表观遗传调控。与许多其他恶性肿瘤不同,SCCOHT表现出低突变负担和二倍体核型,这表明表观遗传失调而非基因组不稳定是潜在的致癌机制。临床上,SCCOHT常表现为非特异性的腹部或盆腔症状,在多达三分之二的病例中与副肿瘤性高钙血症独特相关。诊断需要结合影像学、实验室评估、组织病理学和免疫组织化学。治疗尚无标准化方案,但通常采用多模式方法,包括根治性手术和铂类化疗,常采用多药联合方案。放疗的作用尚不明确,但可考虑用于局部控制或缓解症状。预后仍然很差,复发率高,对挽救治疗的反应有限。新出现的分子见解促使人们对靶向治疗和免疫治疗进行研究,尽管临床数据有限。鉴于种系SMARCA4突变经常存在,强烈建议进行遗传咨询,持续的研究对于提高诊断准确性、个性化治疗以及改善这种毁灭性恶性肿瘤的治疗效果至关重要。