在澳大利亚维多利亚州的三个三级转诊中心，刺猬通路抑制剂的八年经验。

IF 1.8 4区医学 Q2 DERMATOLOGY

Australasian Journal of Dermatology Pub Date : 2025-06-10 DOI:10.1111/ajd.14544

Christian Gan, Nicholas Manuelpillai, Peter Foley, Christopher McCormack, Michelle S Goh

{"title":"在澳大利亚维多利亚州的三个三级转诊中心，刺猬通路抑制剂的八年经验。","authors":"Christian Gan, Nicholas Manuelpillai, Peter Foley, Christopher McCormack, Michelle S Goh","doi":"10.1111/ajd.14544","DOIUrl":null,"url":null,"abstract":"This retrospective analysis of Hedgehog inhibitor treatment in 32 patients with Gorlin syndrome, locally advanced and metastatic basal cell carcinoma (BCC) at three tertiary referral centres in Victoria, Australia from April 2017 until 30 June 2024 demonstrated an 84% overall objective response rate (partial and complete response combined). However, 90% of patients experienced adverse effects impacting quality of life. Secondary acquired drug resistance occurred in 77% (10/13) of locally advanced and metastatic BCC patients after a median duration of 13 months. Further work is needed to optimise the neoadjuvant use of Hedgehog inhibitors with radiotherapy or surgery given poor long-term Hedgehog inhibitor tolerability and to develop strategies to counteract the issue of acquired resistance.","PeriodicalId":8638,"journal":{"name":"Australasian Journal of Dermatology","volume":" ","pages":""},"PeriodicalIF":1.8000,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12334801/pdf/","citationCount":"0","resultStr":"{\"title\":\"Eight-Year Experience of Hedgehog Pathway Inhibitors at Three Tertiary Referral Centres in the Australian State of Victoria.\",\"authors\":\"Christian Gan, Nicholas Manuelpillai, Peter Foley, Christopher McCormack, Michelle S Goh\",\"doi\":\"10.1111/ajd.14544\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"This retrospective analysis of Hedgehog inhibitor treatment in 32 patients with Gorlin syndrome, locally advanced and metastatic basal cell carcinoma (BCC) at three tertiary referral centres in Victoria, Australia from April 2017 until 30 June 2024 demonstrated an 84% overall objective response rate (partial and complete response combined). However, 90% of patients experienced adverse effects impacting quality of life. Secondary acquired drug resistance occurred in 77% (10/13) of locally advanced and metastatic BCC patients after a median duration of 13 months. Further work is needed to optimise the neoadjuvant use of Hedgehog inhibitors with radiotherapy or surgery given poor long-term Hedgehog inhibitor tolerability and to develop strategies to counteract the issue of acquired resistance.\",\"PeriodicalId\":8638,\"journal\":{\"name\":\"Australasian Journal of Dermatology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":1.8000,\"publicationDate\":\"2025-06-10\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12334801/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Australasian Journal of Dermatology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1111/ajd.14544\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"DERMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Australasian Journal of Dermatology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/ajd.14544","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"DERMATOLOGY","Score":null,"Total":0}

引用次数: 0

摘要

2017年4月至2024年6月30日，澳大利亚维多利亚州三个三级转诊中心对32例Gorlin综合征、局部晚期和转移性基底细胞癌（BCC）患者进行了回顾性分析，结果显示，总的客观缓解率为84%（部分缓解和完全缓解相结合）。然而，90%的患者经历了影响生活质量的不良反应。77%（10/13）的局部晚期和转移性BCC患者在中位持续时间13个月后出现了继发性获得性耐药。鉴于长期的刺猬抑制剂耐受性差，需要进一步的工作来优化放疗或手术中刺猬抑制剂的新辅助使用，并制定策略来抵消获得性耐药的问题。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Eight-Year Experience of Hedgehog Pathway Inhibitors at Three Tertiary Referral Centres in the Australian State of Victoria.

This retrospective analysis of Hedgehog inhibitor treatment in 32 patients with Gorlin syndrome, locally advanced and metastatic basal cell carcinoma (BCC) at three tertiary referral centres in Victoria, Australia from April 2017 until 30 June 2024 demonstrated an 84% overall objective response rate (partial and complete response combined). However, 90% of patients experienced adverse effects impacting quality of life. Secondary acquired drug resistance occurred in 77% (10/13) of locally advanced and metastatic BCC patients after a median duration of 13 months. Further work is needed to optimise the neoadjuvant use of Hedgehog inhibitors with radiotherapy or surgery given poor long-term Hedgehog inhibitor tolerability and to develop strategies to counteract the issue of acquired resistance.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Australasian Journal of Dermatology 医学-皮肤病学

CiteScore

3.20

自引率

5.00%

发文量

186

审稿时长

6-12 weeks

期刊介绍： Australasian Journal of Dermatology is the official journal of the Australasian College of Dermatologists and the New Zealand Dermatological Society, publishing peer-reviewed, original research articles, reviews and case reports dealing with all aspects of clinical practice and research in dermatology. Clinical presentations, medical and physical therapies and investigations, including dermatopathology and mycology, are covered. Short articles may be published under the headings ‘Signs, Syndromes and Diagnoses’, ‘Dermatopathology Presentation’, ‘Vignettes in Contact Dermatology’, ‘Surgery Corner’ or ‘Letters to the Editor’.