Response to Gurevich and colleagues: The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: a systematic literature review by a European panel of experts

Dear Editor,

We are grateful to Gurevich and colleagues for their correspondence [1], highlighting our review of clinical outcomes in male patients with Fabry disease after enzyme replacement therapy (ERT) [2]. This is one of a series of papers published in Molecular Genetics and Metabolism (MGM) or MGM Reports that describe the results of a unique systematic review of the published literature on therapeutic outcomes in patients with Fabry disease, initiated and guided by established experts in this field with extensive experience in patients' clinical care [[2], [3], [4], [5], [6]].

We commend the authors for highlighting the importance of standardisation in the design, conduct and reporting of systematic reviews and concur with their comments on the necessity to report on bias in the published literature. The initial series, as submitted to MGM, included a manuscript with the detailed description of the methodology and bias evaluation for all papers. However, MGM's peer review as stand-alone publication resulted in conflicting feedback from reviewers, and this paper has now been published by the European Journal of Medical Genetics [7].

We confirm that the data search and extraction for this series was conducted in line with recommended practice and that our analysis included a grading of evidence using the levels of evidence classification published by the Oxford Centre for Evidence-Based Medicine [8]. All Grade 1–3 publications were assessed for risk of bias based on the Cochrane tool for assessing bias originally developed for randomised trials [9]. Importantly, pharmaceutical sponsorship of a study was also noted and when insufficient data were available to make a judgement on its impact, the item was noted as a potential risk.

Funding

The systematic literature review analyses, the development of the publications and the meetings of the international expert panel were sponsored by Sanofi Genzyme. The authors received editorial/writing support in the preparation of manuscripts from Excerpta Medica, which was funded by Sanofi Genzyme. The authors were responsible for all content and editorial decisions and received no honoraria related to the development of the publications. The views expressed in this letter are those of the signatories.

Declaration of Competing Interest

D.P. Germain is a consultant for Amicus Therapeutics, Sanofi Genzyme and Takeda, and has received speaker honoraria from Amicus Therapeutics, Sanofi Genzyme and Takeda.

B. Falissard has been a consultant for Actelion, Allergan, Almirall, Astellas, AstraZeneca, Bayer, Biotronik, Boehringer Ingelheim, Bristol-Myers Squibb, Daiichi-Sankyo, Eli Lilly and Company, Gilead Sciences, GlaxoSmithKline, Grünenthal, HRA Pharma, Janssen, Lundbeck, MSD, Novartis, Otsuka, Pierre Fabre, Roche, Sanofi, Sanofi Genzyme, Servier, Stallergene, UCB Pharmaceuticals and ViiV Healthcare.

M.J. Hilz has received research/grant support from Bayer Health Care and Novartis Pharma, and has received speaker honoraria and travel support from Sanofi Genzyme.

M. Spada has received speaker and advisory board honoraria and travel support from Sanofi Genzyme and Takeda.

C. Wanner has received research support from Sanofi Genzyme, is a consultant for Actelion Pharmaceuticals, Protalix, Boehringer Ingelheim and Sanofi Genzyme, and is a member of the European Advisory Board of the Fabry Registry.

P.M. Elliott has received speaker honoraria from Takeda, and has received consultancy and speaker honoraria from Gilead Sciences, MyoKardia, Pfizer and Sanofi Genzyme.