BACKGROUND Oral migalastat has recently been approved for the treatment of Anderson-Fabry disease (FD) in patients aged ≥16 years with amenable mutations on the basis of two phase III trials, FACETS and ATTRACT. However, with the introduction of migalastat into clinical practice, it is important to correctly identify the patients who may gain the most benefits from this therapy. Due to the relatively recent availability of migalastat, its role in clinical practice still has to be included in guidelines or recommendations. On these bases, a multidisciplinary group of Italian Experts in the treatment of FD has run the GALA project, with the aim to collect the opinions of expert physicians and to propose some starting points for an experience-based use of migalastat. RESULTS Overall, although studies and data from longer-term follow-up with migalastat are still emerging, available evidence is consistent in showing that this molecule does represent a suitable therapy for the treatment of FD, in patients aged ≥16 years and with amenable mutations. The use of migalastat as an oral option appears to be overall safe, and experience thus far indicates potential for improving quality of life, controlling GI symptoms, stabilizing renal function and reducing cardiac hypertrophy. CONCLUSION Migalastat can be considered either as a first-line therapy - given its efficacy, extensive tissue penetration, convenient oral regimen, and the current limited therapeutic options available - or in patients on enzyme-replacement therapy (ERT) who experience side effects, with poor compliance to chronic i.v. therapy, or with clinical evidence of progression of the disease.

中文翻译：

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GALA项目：根据意大利专家进行的结构性调查，在临床实践中使用米加司他的实用建议。

背景技术基于两项FACETS和ATTRACT III期III期临床试验，口服migalastat最近已被批准用于治疗年龄≥16岁且有可适应突变的Anderson-Fabry病（FD）。但是，随着将米加司他引入临床实践，正确识别可能从该疗法中获得最大益处的患者非常重要。由于migalastat的使用相对较新，因此其在临床实践中的作用仍必须包含在指南或建议中。在这些基础上，由FD治疗的意大利专家组成的多学科小组开展了GALA项目，旨在收集专家医生的意见，并为基于经验的migalastat的使用提供一些起点。结果总体而言，尽管仍存在关于Migalastat长期随访的研究和数据，但现有证据一致表明，该分子确实代表≥16岁且具有适当突变的患者的FD治疗的合适疗法。使用migalastat口服剂似乎是整体安全的，迄今为止的经验表明，改善生活质量，控制GI症状，稳定肾功能和减少心脏肥大的潜力。结论鉴于米加司他的疗效，广泛的组织渗透性，便捷的口服方案以及当前可用的有限治疗选择，可以将其视为一线疗法，也可以考虑接受酶替代疗法（ERT）且出现副作用的患者对慢性静脉治疗的依从性差，