Kyriakidis Filippos, Kogias Dionysios, Venou Theodora Maria, Karlafti Eleni, Paramythiotis Daniel
Second Chemotherapy Department, Theagenio Cancer Hospital of Thessaloniki, Thessaloniki, Greece.
First Department of Internal Medicine, University General Hospital of Alexandroupolis, Alexandroupolis, Greece.
Appl Clin Genet. 2023 Aug 14;16:139-153. doi: 10.2147/TACG.S372241. eCollection 2023.
Familial adenomatous polyposis (FAP) is an autosomal dominant cancer predisposition syndrome marked by extensive colorectal polyposis and a high risk of colorectal cancer (CRC). Having access to screening and enrollment programs can improve survival for patients with FAP by enabling them to undergo surgery before the development of colorectal cancer. Provided that there are a variety of surgical options available to treat colorectal polyps in patients with adenomatous polyposis, the appropriate surgical option for each patient should be considered. The gold-standard treatment to reduce this risk is prophylactic colectomy, typically by the age of 40. However, colectomy is linked to morbidity and constitutes an ineffective way at preventing extra-colonic disease manifestations, such as desmoid disease, thyroid malignancy, duodenal polyposis, and cancer. Moreover, extensive studies have been conducted into the use of chemopreventive agents to prevent disease progression and delay the necessity for a colectomy as well as the onset of extracolonic disease. The ideal chemoprevention agent should demonstrate a biologically plausible mechanism of action and provide safety, easy tolerance over an extended period of time and a lasting and clinically meaningful effect. Although many pharmaceutical and non-pharmaceutical products have been tested through the years, there has not yet been a chemoprevention agent that meets these criteria. Thus, it is necessary to develop new FAP agents that target novel pathways, such as the mTOR pathway. The aim of this article is to review the prior literature on FAP in order to concentrate the current and future perspectives of diagnosis and treatment. In conclusion, we will provide an update on the diagnostic and therapeutic options, surgical or pharmaceutical, while focusing on the potential treatment strategies that could further reduce the risk of CRC.
家族性腺瘤性息肉病(FAP)是一种常染色体显性遗传的癌症易感综合征,其特征为广泛的结肠直肠息肉病以及患结直肠癌(CRC)的高风险。能够参加筛查和登记项目可以使FAP患者在结直肠癌发生之前接受手术,从而提高其生存率。鉴于有多种手术方案可用于治疗腺瘤性息肉病患者的结肠直肠息肉,应考虑为每位患者选择合适的手术方案。降低这种风险的金标准治疗方法是预防性结肠切除术,通常在40岁时进行。然而,结肠切除术与发病率相关,并且在预防结外疾病表现(如硬纤维瘤病、甲状腺恶性肿瘤、十二指肠息肉病和癌症)方面是一种无效的方法。此外,已经对使用化学预防剂来预防疾病进展、推迟结肠切除术的必要性以及结外疾病的发生进行了广泛研究。理想的化学预防剂应具有生物学上合理的作用机制,并提供安全性、长时间易于耐受以及持久且具有临床意义的效果。尽管多年来已经测试了许多药物和非药物产品,但尚未有一种化学预防剂符合这些标准。因此,有必要开发针对新途径(如mTOR途径)的新型FAP药物。本文的目的是回顾关于FAP的先前文献,以便集中阐述当前和未来的诊断与治疗观点。总之,我们将提供诊断和治疗选择(手术或药物)的最新情况,同时重点关注可能进一步降低CRC风险的潜在治疗策略。