Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey.
The Gaucher Outcome Survey (GOS) is an international disease-specific registry established in 2010 for patients with a confirmed diagnosis of Gaucher disease (GD), regardless of GD type or treatment status. For insight into how GD management varies among countries, we analyzed treatment patterns in GOS. As of October 30, 2015, data on GD-specific treatment (enzyme replacement therapy, substrate reduction therapy, or chemical chaperone therapy) received at any time were available for 647 patients. At analysis, velaglucerase alfa (316/573, 55.1%) and imiglucerase (184/573, 32.1%) were the treatments most widely used. Of the 647 treated patients, 446 (68.9%) had been treated for >5years and 368 (56.9%) had received only one GD-specific drug therapy. There were 377 patients who received velaglucerase alfa. Velaglucerase alfa was most widely used at 60U/kg every other week (134/492 dose entries, 27.2%), but there were differences in dosing between the three highest-enrolling countries (defined as >100 GOS patients enrolled in each), with most patients in Israel receiving <20U/kg, most patients in the United Kingdom receiving 20 to <40U/kg, and most in the United States receiving 60U/kg. This analysis provides a foundation upon which to examine real-life outcomes data from different treatment regimens globally.
Addenbrooke's Hospital, Cambridge, United Kingdom. Electronic address: patrick.deegan@addenbrookes.nhs.uk.
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Deegan
Instituto William Osler, Buenos Aires, Argentina. Electronic address: diegofernandezsasso@criocenter.com.
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Fernandez-Sasso
Translational Research Unit, IIS Aragon, Zaragoza, Spain; CIBER de Enfermedades Raras, IIS Aragon, Zaragoza, Spain. Electronic address: giraldocastellano@gmail.com.
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Giraldo
Division of Neurogenetics, New York University School of Medicine, New York, NY, United States. Electronic address: heather.lau@nyumc.org.
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Lau
Shire, Zug, Switzerland. Electronic address: zpanahloo@shire.com.
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Panahloo
Gaucher Clinic, Shaare Zedek Medical Center, The Hebrew University-Hadassah Medical School, Jerusalem, Israel. Electronic address: azimran@gmail.com.
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Zimran
D004334:Drug Administration Schedule; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000453:epidemiology; D005962:Glucosylceramidase / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D016896:Treatment Outcome
D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human; C090568:imiglucerase
Enzyme replacement therapy; Gaucher disease; Substrate reduction therapy; Velaglucerase alfa