Elliott, PM

Falissard, B

Germain, DP

Hilz, MJ

Spada, M

Wanner, C

UCL Discovery

Contents lists available at ScienceDirectMolecular Genetics and Metabolism Reportsjournal homepage: www.elsevier.com/locate/ymgmrCorrespondenceResponse to Gurevich and colleagues: The effect of enzyme replacement therapy on clinicaloutcomes in male patients with Fabry disease: a systematic literature review by a European panel ofexpertsA R T I C L E I N F OKeywords:Fabry diseaseEnzyme replacement therapyAgalsidase alfaAgalsidase betaDear Editor,We are grateful to Gurevich and colleagues for their correspondence[1], highlighting our review of clinical outcomes in male patients withFabry disease after enzyme replacement therapy (ERT) [2]. This is oneof a series of papers published in Molecular Genetics and Metabolism(MGM) or MGM Reports that describe the results of a unique systematicreview of the published literature on therapeutic outcomes in patientswith Fabry disease, initiated and guided by established experts in thisfield with extensive experience in patients' clinical care [2–6].We commend the authors for highlighting the importance of stan-dardisation in the design, conduct and reporting of systematic reviewsand concur with their comments on the necessity to report on bias inthe published literature. The initial series, as submitted to MGM, in-cluded a manuscript with the detailed description of the methodologyand bias evaluation for all papers. However, MGM's peer review asstand-alone publication resulted in conflicting feedback from reviewers,and this paper has now been published by the European Journal ofMedical Genetics [7].We confirm that the data search and extraction for this series wasconducted in line with recommended practice and that our analysisincluded a grading of evidence using the levels of evidence classifica-tion published by the Oxford Centre for Evidence-Based Medicine [8].All Grade 1–3 publications were assessed for risk of bias based on theCochrane tool for assessing bias originally developed for randomisedtrials [9]. Importantly, pharmaceutical sponsorship of a study was alsonoted and when insufficient data were available to make a judgementon its impact, the item was noted as a potential risk.FundingThe systematic literature review analyses, the development of thepublications and the meetings of the international expert panel weresponsored by Sanofi Genzyme. The authors received editorial/writingsupport in the preparation of manuscripts from Excerpta Medica, whichwas funded by Sanofi Genzyme. The authors were responsible for allcontent and editorial decisions and received no honoraria related to thedevelopment of the publications. The views expressed in this letter arethose of the signatories.Declaration of Competing InterestD.P. Germain is a consultant for Amicus Therapeutics, SanofiGenzyme and Takeda, and has received speaker honoraria from AmicusTherapeutics, 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 HealthCare and Novartis Pharma, and has received speaker honoraria andtravel support from Sanofi Genzyme.M. Spada has received speaker and advisory board honoraria andtravel support from Sanofi Genzyme and Takeda.C. Wanner has received research support from Sanofi Genzyme, is aconsultant for Actelion Pharmaceuticals, Protalix, BoehringerIngelheim and Sanofi Genzyme, and is a member of the EuropeanAdvisory Board of the Fabry Registry.P.M. Elliott has received speaker honoraria from Takeda, and hasreceived consultancy and speaker honoraria from Gilead Sciences,MyoKardia, Pfizer and Sanofi Genzyme.References[1] A. Gurevich, J. Schenk, H. Wellhoefer, V. Kalampoki, A response to Germain, et al.,The effect of enzyme replacement therapy on clinical outcomes in male patients withFabry disease: a systematic literature review by a European panel of experts, Mol.Genet. Metab. Rep. 19 (2019) 100471, , https://doi.org/10.1016/j.ymgmr.2019.100471 (Feb 6 2019).[2] D.P. Germain, P.M. Elliott, B. Falissard, V.V. Fomin, M.J. Hilz, A. Jovanovic,I. Kantola, A. Linhart, R. Mignani, M. Namdar, A. Nowak, J.P. Oliveira, M. Pieroni,M. Viana-Baptista, C. Wanner, M. Spada, The effect of enzyme replacement therapyon clinical outcomes in male patients with Fabry disease: a systematic literaturereview by a European panel of experts, Mol. Genet. Metab. Rep. 19 (2019) 100454, ,https://doi.org/10.1016/j.ymgmr.2019.100454.[3] M. Spada, R. Baron, P.M. Elliott, B. Falissard, M.J. Hilz, L. Monserrat, C. Tøndel,A. Tylki-Szymańska, C. Wanner, D.P. Germain, The effect of enzyme replacementtherapy on clinical outcomes in paediatric patients with Fabry disease - a systematicliterature review by a European panel of experts, Mol. Genet. Metab. 126 (2019)212–223, https://doi.org/10.1016/j.ymgme.2018.04.007.https://doi.org/10.1016/j.ymgmr.2019.100493Received 2 July 2019; Received in revised form 5 July 2019; Accepted 5 July 2019Molecular Genetics and Metabolism Reports 20 (2019) 100493Available online 22 July 20192214-4269/ © 2019 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/BY-NC-ND/4.0/).T[4] D.P. Germain, M. Arad, A. Burlina, P.M. Elliott, B. Falissard, U. Feldt-Rasmussen,M.J. Hilz, D.A. Hughes, A. Ortiz, C. Wanner, F. Weidemann, M. Spada, The effect ofenzyme replacement therapy on clinical outcomes in female patients with Fabrydisease - a systematic literature review by a European panel of experts, Mol. Genet.Metab. 126 (2019) 224–235, https://doi.org/10.1016/j.ymgme.2018.09.007.[5] C. Wanner, M. Arad, R. Baron, A. Burlina, P.M. Elliott, U. Feldt-Rasmussen,V.V. Fomin, D.P. Germain, D.A. Hughes, A. Jovanovic, I. Kantola, A. Linhart,R. Mignani, L. Monserrat, M. Namdar, A. Nowak, J.P. Oliveira, A. Ortiz, M. Pieroni,M. Spada, A. Tylki-Szymańska, C. Tøndel, M. Viana-Baptista, F. Weidemann,M.J. Hilz, European expert consensus statement on therapeutic goals in Fabry dis-ease, Mol. Genet. Metab. 124 (2018) 189–203, https://doi.org/10.1016/j.ymgme.2018.06.004.[6] C. Wanner, D.P. Germain, M.J. Hilz, M. Spada, B. Falissard, P.M. Elliott, Therapeuticgoals in Fabry disease: recommendations of a European expert panel, based oncurrent clinical evidence with enzyme replacement therapy, Mol. Genet. Metab. 126(2019) 210–211, https://doi.org/10.1016/j.ymgme.2018.04.004.[7] P.M. Elliott, D.P. Germain, M.J. Hilz, M. Spada, C. Wanner, B. Falissard, Why sys-tematic literature reviews in Fabry disease should include all published evidence,Eur. J. Med. Genet. (2019) 103702Epub ahead of print https://doi.org/10.1016/j.ejmg.2019.103702.[8] CEBM, Oxford Centre for Evidence-based Medicine – Levels of Evidence, https://www.cebm.net/oxford-centre-evidence-based-medicine-levels-evidence-march-/2009, (March 2009) (accessed 22.06.2019).[9] J.P. Higgins, D.G. Altman, P.C. Gøtzsche, P. Jüni, D. Moher, A.D. Oxman, J. Savovic,K.F. Schulz, L. Weeks, J.A. Sterne, Cochrane Bias Methods Group, CochraneStatistical Methods Group, The Cochrane Collaboration’s tool for assessing risk ofbias in randomised trials, BMJ 343 (2011) d5928, https://doi.org/10.1136/bmj.d5928.Dominique P. Germaina,⁎, Bruno Falissardb, Max J. Hilzc, Marco Spadad,Christoph Wannere, Perry M. Elliottfa French Referral Center for Fabry disease, Division of Medical Genetics andINSERM U1179, University of Versailles, Paris-Saclay University,Montigny, Franceb INSERM U1018, University of Paris-Sud, Paris Descartes University, Paris,FrancecDepartment of Neurology, University of Erlangen-Nuremberg, Erlangen,GermanydDepartment of Paediatrics, University of Torino, Torino, Italye Division of Nephrology, University Clinic, University of Würzburg,Würzburg, GermanyfUniversity College London and Barts Heart Centre, London, UnitedKingdomE-mail address: dominique.germain@uvsq.fr (D.P. Germain).⁎ Corresponding author.Correspondence Molecular Genetics and Metabolism Reports 20 (2019) 1004932

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

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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

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