Vismodegib in Gorlin-Goltz syndrome: A systematic review

IF 2.2 4区医学 Q2 DERMATOLOGY

Australasian Journal of Dermatology Pub Date : 2024-06-12 DOI:10.1111/ajd.14326

Ana Gusmão Palmeiro MD, Mélissa Carvalho MD, Cristina Gonçalves Castro MD, Bernardo Pimentel MD, Goreti Catorze MD

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引用次数: 0

Abstract

Treatment with Hedgehog Inhibitors in Gorlin-Goltz syndrome (GGS) yields favourable objective clinical responses, yet secondary resistance and class-related toxicity restrict treatment duration. This study aims to review current data on GGS patients undergoing vismodegib therapy, focusing on treatment duration, clinical outcomes and schedule modifications. A systematic search of the PubMed database was conducted for English articles from 1993 to 2023, identifying 31 papers suitable for inclusion. A total of 351 patients, with a mean age of 52 years, were analysed. The average treatment duration was 9.3 months for patients who discontinued treatment, and 25.1 months for those who continued vismodegib at the time this study was published. Vismodegib achieved a complete response rate of 44%. Treatment interruption predominantly occurred due to side effects (69.1%) and secondary resistance (9.1%). The use of alternative regimens, although not compromising efficacy, may enhance treatment compliance. Further investigations are warranted to ascertain the optimal treatment regimen and timeline for GGS patients. Schedule modifications offer promise in ameliorating side effects and facilitating long-term treatment.

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Vismodegib治疗戈林-戈兹综合征：系统综述。

使用刺猬蛋白抑制剂治疗戈林-戈兹综合征（GGS）可产生良好的客观临床反应，但继发性耐药性和同类药物相关毒性限制了治疗持续时间。本研究旨在回顾接受 vismodegib 治疗的 GGS 患者的现有数据，重点关注治疗持续时间、临床疗效和疗程调整。研究人员在PubMed数据库中对1993年至2023年的英文文章进行了系统检索，发现了31篇适合纳入的论文。共分析了 351 名患者，平均年龄为 52 岁。在本研究发表时，停止治疗的患者的平均治疗时间为 9.3 个月，而继续服用 Vismodegib 的患者的平均治疗时间为 25.1 个月。Vismodegib 的完全应答率为 44%。中断治疗的主要原因是副作用（69.1%）和继发性耐药（9.1%）。使用替代方案虽然不会影响疗效，但可提高治疗依从性。有必要开展进一步研究，以确定 GGS 患者的最佳治疗方案和时间表。调整疗程有望减轻副作用并促进长期治疗。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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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’.