Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe

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• 作者：Nadia Belmatoug^a ; ^{nadia.belmatoug@aphp.fr} ; Maja Di Rocco^b ; ^{majadirocco@ospedale-gaslini.ge.it} ; Cristina Fraga^c ; ^{maria.cf.barros@azores.gov.pt} ; Pilar Giraldo^d ; ^{giraldocastellano@gmail.com} ; Derralynn Hughes^e ; Elena Lukina^f ; ^{elenalukina02@gmail.com} ; Pierre Maison-Blanche^g ; ^{ipmb@wanadoo.fr} ; Martin Merkel^h ; ^{m.merkel@asklepios.com} ; Claus Niederauⁱ ; ^{c.niederau@kk-ob.de} ; Ursula Plӧckinger^j ; ^{ursula.ploeckinger@charite.de} ; Johan Richter^k ; ^{johan.richter@med.lu.se} ; Thomas M. Stulnig^l ; ^{thomas.stulnig@meduniwien.ac.at} ; Stephan vom Dahl^m ; ^{Stephan.vomdahl@med.uni-duesseldorf.de} ; Timothy M. Coxⁿ ; ^{tmc12@medschl.cam.ac.uk}
• 关键词：BMD ; bone mineral density ; ECG ; electrocardiogram ; ERT ; enzyme replacement therapy ; ICGG ; International Collaborative Gaucher Group
• 刊名：European Journal of Internal Medicine
• 出版年：2017
• 出版时间：January 2017
• 年：2017
• 卷：37
• 期：Complete
• 页码：25-32
• 全文大小：526 K
• 卷排序：37

文摘

Eliglustat is approved for adults with Gaucher disease and compatible CYP2D6 metaboliser genotypes. Distinctive characteristics of eliglustat require additional investigation and monitoring. Recommendations for all other aspects of Gaucher disease remain unchanged. Eliglustat dose is determined by predicted CYP2D6 metaboliser phenotype. Concomitant use of drugs metabolised by CYP2D6 and CYP3A requires eliglustat dose adjustment.