Department of Pathology, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Department of Pathology, Beth Israel Deaconess Medical Center, Boston, MA, USA.
Fam Cancer. 2021 Jul;20(3):201-213. doi: 10.1007/s10689-020-00210-4. Epub 2020 Oct 9.
The widespread use of tumor DNA mismatch repair (MMR) protein immunohistochemistry in gastrointestinal tract (GIT) carcinomas has unveiled cases where the MMR protein status differs between synchronous/metachronous tumors from the same patients. This study aims at examining the frequency, patterns and molecular etiologies of such inter-tumoral MMR discordances. We analyzed a cohort of 2159 colorectal cancer (CRC) patients collected over a 5-year period and found that 1.3% of the patients (27/2159) had ≥ 2 primary CRCs, and 25.9% of the patients with ≥ 2 primary CRCs (7/27) exhibited inter-tumoral MMR discordance. We then combined the seven MMR-discordant CRC patients with three additional MMR-discordant GIT carcinoma patients and evaluated their discordant patterns and associated molecular abnormalities. The 10 patients consisted of 3 patients with Lynch syndrome (LS), 1 with polymerase proofreading-associated polyposis (PAPP), 1 with familial adenomatous polyposis (FAP), and 5 deemed to have no cancer disposing hereditary syndromes. Their MMR discordances were associated with the following etiologies: (1) PMS2-LS manifesting PMS2-deficient cancer at an old age when a co-incidental sporadic MMR-proficient cancer also occurred; (2) microsatellite instability-driven secondary somatic MSH6-inactivation occurring in only one-and not all-PMS2-LS associated MMR-deficient carcinomas; (3) "compound LS" with germline mutations in two MMR genes manifesting different tumors with deficiencies in different MMR proteins; (4) PAPP or FAP syndrome-associated MMR-proficient cancer co-occurring metachronously with a somatic MMR-deficient cancer; and (5) non-syndromic patients with sporadic MMR-proficient cancers co-occurring synchronously/metachronously with sporadic MMR-deficient cancers. Our study thus suggests that inter-tumoral MMR discordance is not uncommon among patients with multiple primary GIT carcinomas (25.9% in patients with ≥ 2 CRCs), and may be associated with widely varied molecular etiologies. Awareness of these patterns is essential in ensuring the most effective strategies in both LS detection and treatment decision-making. When selecting patients for immunotherapy, MMR testing should be performed on the tumor or tumors that are being treated.
在胃肠道(GIT)癌中广泛使用肿瘤 DNA 错配修复(MMR)蛋白免疫组织化学检测揭示了这样的病例,即同一患者的同步/异时肿瘤之间的 MMR 蛋白状态存在差异。本研究旨在研究这种肿瘤间 MMR 不和谐的频率、模式和分子病因。我们分析了一组在 5 年内收集的 2159 例结直肠癌(CRC)患者,发现 1.3%的患者(27/2159)有≥2 个原发性 CRC,并且≥2 个原发性 CRC 患者中有 25.9%(7/27)存在肿瘤间 MMR 不和谐。然后,我们将这 7 例 MMR 不和谐的 CRC 患者与另外 3 例 MMR 不和谐的 GIT 癌患者相结合,并评估了他们的不和谐模式和相关的分子异常。这 10 名患者包括 3 名林奇综合征(LS)患者、1 名聚合酶校对相关息肉病(PAPP)患者、1 名家族性腺瘤性息肉病(FAP)患者和 5 名认为没有癌症遗传综合征的患者。他们的 MMR 不和谐与以下病因相关:(1)PMS2-LS 表现为 PMS2 缺陷型癌症,年龄较大时同时发生偶然的散发性 MMR 功能正常型癌症;(2)微卫星不稳定性驱动的二次体细胞 MSH6 失活,仅发生在一个而非所有 PMS2-LS 相关的 MMR 缺陷型癌中;(3)具有两种 MMR 基因种系突变的“复合 LS”表现为不同的肿瘤,具有不同的 MMR 蛋白缺陷;(4)PAPP 或 FAP 综合征相关的 MMR 功能正常型癌症与体细胞 MMR 缺陷型癌症同时或异时发生;(5)非综合征患者的散发性 MMR 功能正常型癌症与散发性 MMR 缺陷型癌症同时或异时发生。因此,我们的研究表明,多个胃肠道主要癌(在有≥2 个 CRC 的患者中占 25.9%)患者之间的肿瘤间 MMR 不和谐并不少见,并且可能与广泛的分子病因相关。了解这些模式对于确保 LS 检测和治疗决策制定的最有效策略至关重要。在选择患者进行免疫治疗时,应在正在治疗的肿瘤或肿瘤上进行 MMR 检测。