Därr Roland, Kater Jonas, Sekula Peggy, Bausch Birke, Krauss Tobias, Bode Christoph, Walz Gerd, Neumann Hartmut P, Zschiedrich Stefan
Department of Cardiology and Angiology I, Heart Center Freiburg University, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Department of Medicine IV, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Endocr Connect. 2020 Aug;9(8):834-844. doi: 10.1530/EC-20-0208.
The optimal treatment strategy for patients with small non-functioning VHL-related incidentalomas is unclear. We searched the Freiburg VHL registry for patients with radiologic evidence of pheochromocytoma/paraganglioma (PHEO/PGL). In total, 176 patients with single, multiple, and recurrent tumours were identified (1.84 tumours/patient, range 1-8). Mean age at diagnosis was 32 ± 16 years. Seventy-four percent of tumours were localised to the adrenals. Mean tumour diameter was 2.42 ± 2.27 cm, 46% were <1.5 cm. 24% of tumours were biochemically inactive. Inactive tumours were significantly smaller than active PHEO/PGL at diagnosis (4.16 ± 2.80 cm vs 1.43 ± 0.45 cm; P < 0.025) and before surgery (4.89 ± 3.47 cm vs 1.36 ± 0.43 cm; P < 0.02). Disease was stable in 67% of 21 patients with evaluable tumours ≤1.5 cm according to RECIST and progressed in 7. Time till surgery in these patients was 29.5 ± 20.0 months. A total of 155 patients underwent surgery. PHEO/PGL was histologically excluded in 4 and proven in 151. Of these, one had additional metastatic disease, one harboured another tumour of a different type, and in 2 a second surgery for suspected disease recurrence did not confirm PHEO/PGL. Logistic regression analysis revealed 50% probability for a positive/negative biochemical test result at 1.8 cm tumour diameter. Values of a novel symptom score were positively correlated with tumour size (Rs = 0.46, P < 0.0001) and together with a positive biochemistry a linear size predictor (P < 0.01). Results support standardised clinical assessment and measurement of tumour size and metanephrines in VHL patients with non-functioning incidentalomas <1.5 cm at one year following diagnosis and at individualised intervals thereafter depending on evolving growth dynamics, secretory activity and symptomatology.
对于患有小型无功能的VHL相关偶发瘤患者的最佳治疗策略尚不清楚。我们在弗莱堡VHL登记处搜索了有嗜铬细胞瘤/副神经节瘤（PHEO/PGL）放射学证据的患者。总共确定了176名单发、多发和复发性肿瘤患者（平均每名患者1.84个肿瘤，范围为1 - 8个）。诊断时的平均年龄为32±16岁。74%的肿瘤位于肾上腺。肿瘤平均直径为2.42±2.27厘米，46%的肿瘤<1.5厘米。24%的肿瘤生化无活性。诊断时，无活性肿瘤明显小于活性PHEO/PGL（4.16±2.80厘米对1.43±0.45厘米；P<0.025），手术前也是如此（4.89±3.47厘米对1.36±0.43厘米；P<0.02）。根据RECIST标准，在21名可评估肿瘤≤1.5厘米的患者中，67%的疾病稳定，7名患者病情进展。这些患者直到手术的时间为29.5±20.0个月。共有155名患者接受了手术。组织学检查排除了4例PHEO/PGL，151例得到证实。其中，1例有额外的转移性疾病，1例有另一种不同类型的肿瘤，2例因疑似疾病复发进行的二次手术未证实为PHEO/PGL。逻辑回归分析显示，肿瘤直径为1.8厘米时，生化检测结果呈阳性/阴性的概率为50%。一种新的症状评分值与肿瘤大小呈正相关（Rs = 0.46，P<0.0001），并且与生化检测呈阳性一起构成线性大小预测指标（P<0.01）。结果支持对诊断后一年时肿瘤<1.5厘米的VHL无功能偶发瘤患者进行标准化临床评估以及肿瘤大小和甲氧基肾上腺素的测量，并在此后根据不断变化的生长动态、分泌活性和症状表现按个体化间隔进行评估。
