Endocrine Practice Heidelberg, Molecular Genetic Laboratory, D-69120 Heidelberg, Germany.
Institute of Medical Biometry and Informatics, University of Heidelberg, D-69120 Heidelberg, Germany.
J Clin Endocrinol Metab. 2021 Aug 18;106(9):e3582-e3591. doi: 10.1210/clinem/dgab326.
Long-term data are scarce on large cohorts with sporadic (sMTC) and hereditary medullary thyroid carcinoma (hMTC).
To compare long-term disease-specific survival (DSS) and outcomes between sMTC and hMTC groups.
Retrospective analysis.
German tertiary referral center.
A total of 673 patients with MTC that underwent surgery from January 1974 to July 2019.
None (observational study).
Differences between sMTC and hMTC in long-term, stage-dependent survival and outcomes.
Surgery was performed at median ages of 49 years for sMTC (n = 477, 44% male) and 29 years for hMTC (n = 196, 43% male; P < 0.0001). The mean follow-up times were 9.2 ± 8.0 (sMTC) and 14.6 ± 10.3 years (hMTC). Age and tumor stage at diagnosis were significantly different between the 2 groups (P < 0.0001). The sMTC and hMTC groups had different overall DSS (log rank, P = 0.0183), but similar stage-dependent DSS (log rank, P = 0.1242-0.8981). In a multivariate analysis, sMTC and hMTC did not differ in DSS (hazard ratio [HR] = 1.56; 95% CI, 0.94-2.57), but in both groups, a worse DSS was significantly associated with age at diagnosis (HR = 1.04; 95% CI, 1.02-1.05), male sex (HR = 0.49; 95% CI, 0.32-0.76), and stages III and IV at diagnosis (HR = 20.00; 95% CI, 2.74-145.91 and HR = 97.47; 95% CI, 13.07-726.67, respectively). The groups had significantly different (P < 0.0001) outcomes (i.e., cured, minimal residual disease, structural detectable disease, and death), but similar stage-dependent outcomes (P = 0.9449-0.0511), except for stage III (P = 0.0489).
Patients with sMTC and hMTC had different ages of onset, but similar stage-dependent DSS and outcomes after the MTC diagnosis. This finding suggested that tumor behavior was similar in sMTC and hMTC.
关于散发性（sMTC）和遗传性髓样甲状腺癌（hMTC）的大型队列的长期数据稀缺。
比较 sMTC 和 hMTC 组之间的疾病特异性生存（DSS）和结局的长期差异。
回顾性分析。
德国三级转诊中心。
1974 年 1 月至 2019 年 7 月期间接受手术的共 673 例 MTC 患者。
无（观察性研究）。
sMTC 和 hMTC 在长期、基于分期的生存和结局方面的差异。
sMTC（n = 477，44%男性）和 hMTC（n = 196，43%男性；P < 0.0001）的手术中位年龄分别为 49 岁和 29 岁。平均随访时间分别为 9.2 ± 8.0 年（sMTC）和 14.6 ± 10.3 年（hMTC）。两组间年龄和诊断时的肿瘤分期存在显著差异（P < 0.0001）。sMTC 和 hMTC 组的总 DSS 不同（对数秩检验，P = 0.0183），但基于分期的 DSS 相似（对数秩检验，P = 0.1242-0.8981）。在多变量分析中，sMTC 和 hMTC 的 DSS 无差异（风险比[HR] = 1.56；95%置信区间，0.94-2.57），但在两组中，诊断时年龄较大（HR = 1.04；95%置信区间，1.02-1.05）、男性（HR = 0.49；95%置信区间，0.32-0.76）和诊断时 III 期和 IV 期（HR = 20.00；95%置信区间，2.74-145.91 和 HR = 97.47；95%置信区间，13.07-726.67）与较差的 DSS 显著相关。两组的结局（即治愈、微小残留疾病、结构可检测疾病和死亡）存在显著差异（P < 0.0001），但基于分期的结局相似（P = 0.9449-0.0511），除了 III 期（P = 0.0489）。
sMTC 和 hMTC 患者的发病年龄不同，但 MTC 诊断后基于分期的 DSS 和结局相似。这一发现表明，sMTC 和 hMTC 的肿瘤行为相似。
