Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, No.119, the South Fourth Ring West Road, Fengtai District, Beijing, 100070, China.
Orphanet J Rare Dis. 2022 Mar 18;17(1):126. doi: 10.1186/s13023-022-02281-1.
Craniopharyngioma (CP) and cranial fibrous dysplasia (CFD) are rare embryonic benign cranial diseases that most commonly present during childhood or adolescence. The coexistence of CP and CFD is extremely rare and has not yet been reported.
We retrospectively reviewed the data of five patients with concomitant CP and CFD treated at Beijing Tiantan Hospital from January 2003 to January 2021 and summarized their clinicopathological features, treatment modalities, and outcomes. We also performed a comprehensive literature review, tested the patients for characteristic GNAS gene mutations related to CFD, and tested the CP specimens for corresponding Gsα protein to explore the potential connection leading to the coexistence of CP and CFD.
The cohort comprised four men and one woman (median age, 39 years). The symptoms mainly included headache, dizziness, fatigue, polyuria/polydipsia, hypogonadism, and blurred vision. CFD most commonly involved the sphenoid bone (n = 4). Four patients underwent surgery to remove the CP (one trans-sphenoidal and three transcranial resections); complete and subtotal resection were achieved in two patients, respectively. The tumor subtype was adamantinomatous in three patients and unknown in one. The common postoperative complications were panhypopituitarism, diabetes insipidus, and hypothyroidism. The mean follow-up duration was 57.2 months. Two patients required postoperative hormone replacement therapy. Three patients underwent genetic study of the tumor specimens; GNAS mutations were not detected, but these patients were positive for Gsα protein.
Although a definite causative relationship has not been proved, the coexistence of CP and CFD means that potential interplay or an atypical fibrous dysplasia course as uncommon manifestations of CP cannot be excluded. It is more challenging to initiate prompt diagnosis and appropriate treatment for concomitant CP and CFD than for solitary CP because of skull base deformations. Current management strategies are aimed at surgical treating the CP and regularly monitoring the CFD.
颅咽管瘤(CP)和颅纤维结构不良(CFD)是罕见的胚胎良性颅部疾病,最常见于儿童或青少年期发病。CP 和 CFD 同时存在极为罕见,尚未见报道。
我们回顾性分析了 2003 年 1 月至 2021 年 1 月期间在北京天坛医院接受治疗的 5 例 CP 和 CFD 共存患者的临床病理资料,总结了其临床病理特征、治疗方式及转归。我们还进行了全面的文献回顾,对患者进行了与 CFD 相关的特征性 GNAS 基因突变检测,并对 CP 标本进行了相应的 Gsα 蛋白检测,以探讨导致 CP 和 CFD 共存的潜在联系。
该队列包括 4 例男性和 1 例女性(中位年龄,39 岁)。症状主要包括头痛、头晕、乏力、多尿/多饮、性腺功能减退和视力模糊。CFD 最常累及蝶骨(n = 4)。4 例患者接受手术切除 CP(1 例经蝶窦入路,3 例开颅手术);2 例患者分别达到全切和次全切除。肿瘤亚型 3 例为造釉细胞瘤型,1 例为未分类。常见的术后并发症包括全垂体功能减退、尿崩症和甲状腺功能减退。中位随访时间为 57.2 个月。2 例患者术后需要激素替代治疗。3 例患者对肿瘤标本进行了遗传研究;未发现 GNAS 突变,但这些患者的 Gsα 蛋白阳性。
虽然尚未证明存在明确的因果关系,但 CP 和 CFD 同时存在意味着不能排除潜在的相互作用或作为 CP 不常见表现的非典型纤维结构不良过程。由于颅底畸形,与单纯 CP 相比,CP 和 CFD 同时存在更难以及时诊断和进行适当治疗。目前的治疗策略是手术治疗 CP,并定期监测 CFD。