Sugitani Iwao, Onoda Naoyoshi, Ito Ken-Ichi, Suzuki Shinichi
Department of Endocrine Surgery, Nippon Medical School Graduate School of Medicine.
Department of Surgical Oncology, Osaka City University Graduate School of Medicine.
J Nippon Med Sch. 2018;85(1):18-27. doi: 10.1272/jnms.2018_85-3.
Anaplastic thyroid carcinoma (ATC) accounts for only 1 to 2% of all thyroid carcinomas, but it is one of the most lethal neoplasms in humans. To obtain further insights into this "orphan disease," we have established the ATC Research Consortium of Japan (ATCCJ) in 2009. It represents a multicenter registry for ATC that have been treated in Japan. To date, 67 institutions have taken part in the collaborative research system and over 1,200 cases have been accumulated in its database. Using this big data, several retrospective studies were carried out to evaluate 1) prognostic factors to determine initial treatment policy, 2) significance of extended radical surgery for Stage IVB cases, 3) characteristics of ATC incidentally found on pathological examination and 4) pathological features of ATC with long-term survival. Moreover, the ATCCJ has conducted an investigator-initiated, nationwide, prospective clinical trial since 2012; namely, the feasibility, safety and efficacy study of weekly paclitaxel administration for patients with ATC (UMIN: 000008574). Revised Japanese guidelines for treatment of thyroid tumors are going to adopt the recommendations from the results of this research. Since 2016, the ATCCJ has started the phase II study assessing the efficacy and safety of lenvatinib, a newly developed tyrosine kinase inhibitor for ATC (UMIN: 000020773). Our nationwide clinical trial network will strengthen the activity to recruit orphan disease patients and may discover new strategies to conquer this dismal malignancy in the near future.
间变性甲状腺癌（ATC）仅占所有甲状腺癌的1%至2%，但却是人类最致命的肿瘤之一。为了进一步深入了解这种“罕见病”，我们于2009年成立了日本ATC研究联盟（ATCCJ）。它是一个针对在日本接受治疗的ATC的多中心登记处。迄今为止，已有67家机构参与了这一合作研究系统，其数据库中已积累了1200多例病例。利用这些大数据，开展了多项回顾性研究，以评估：1）用于确定初始治疗策略的预后因素；2）IVB期病例扩大根治性手术的意义；3）病理检查时偶然发现的ATC的特征；4）长期存活的ATC的病理特征。此外，自2012年以来，ATCCJ开展了一项由研究者发起的全国性前瞻性临床试验；即每周给予紫杉醇治疗ATC患者的可行性、安全性和疗效研究（UMIN：000008574）。修订后的日本甲状腺肿瘤治疗指南将采纳这项研究结果中的建议。自2016年以来，ATCCJ已启动II期研究，评估一种新开发的用于治疗ATC的酪氨酸激酶抑制剂乐伐替尼的疗效和安全性（UMIN：000020773）。我们的全国性临床试验网络将加强招募罕见病患者的活动，并可能在不久的将来发现攻克这种恶性疾病的新策略。
