Department of Medical Oncology, Kindai University Faculty of Medicine, Osaka-Sayama, Japan.
Invest New Drugs. 2022 Feb;40(1):194-197. doi: 10.1007/s10637-021-01168-5. Epub 2021 Aug 31.
Extramammary Paget disease (EMPD) is a rare cutaneous adenocarcinoma that usually is of epidermal origin and shows glandular differentiation and that is treated by wide local excision depending on the disease extent. For widely metastatic disease, however, a standard treatment remains to be established. Similar to breast cancer, EMPD has been found to overexpress human epidermal growth factor receptor 2 (HER2) or hormone receptors (HRs). Whereas HER2-directed therapy was recently shown to be effective for HER2-positive EMPD, the potential role of endocrine therapy for HR-positive EMPD has remained unknown. We here report a case of metastatic EMPD with HR positivity that was successfully treated with the selective estrogen receptor modulator tamoxifen. This first-line treatment of systemic metastasis resulted in durable tumor regression for > 20 months without any treatment-related toxicities. This is the first report to reveal the promise of tamoxifen as a safe and effective treatment for HR-positive metastatic EMPD.
乳房外帕哲病（EMPD）是一种罕见的皮肤腺癌，通常来源于表皮，具有腺体分化特征，治疗方法是根据疾病范围进行广泛的局部切除。然而，对于广泛转移的疾病，仍需要建立标准的治疗方法。与乳腺癌类似，EMPD 被发现过度表达人表皮生长因子受体 2（HER2）或激素受体（HRs）。最近已经证明，针对 HER2 的治疗对 HER2 阳性 EMPD 有效，而针对 HR 阳性 EMPD 的内分泌治疗的潜在作用仍不清楚。我们在此报告一例 HR 阳性转移性 EMPD 病例，该病例用选择性雌激素受体调节剂他莫昔芬成功治疗。这种一线全身转移治疗方法导致肿瘤持续消退超过 20 个月，且无任何治疗相关的毒性。这是第一个报道他莫昔芬作为 HR 阳性转移性 EMPD 的安全有效治疗方法的报告。
