Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease

American Journal of Hematology
Deborah ElsteinAri Zimran

Abstract

Gaucher disease (GD) is a lysosomal storage disorder; symptomatic patients with type 1 GD need long-term disease-specific therapy of which the standard of care has been enzyme replacement therapy (ERT). Thirty-eight of 40 patients (aged 9-71 years) clinically stable on ERT with imiglucerase, safely switched to a comparable dose of velaglucerase alfa (units/kg) during TKT034, a 12-month, open-label clinical study, and for 10-50 months in an extension study. The most common adverse events (AEs) judged to be drug-related in the extension were fatigue and bone pain. No drug-related serious AEs were reported. No AEs led to study withdrawal. At 24 months from baseline (baseline being TKT034 week 0), patients had generally stable hemoglobin, platelet, spleen, liver, and bone density parameters. Nevertheless, dose adjustment based on the achievement of therapeutic goals was permitted, and 10 patients, including seven patients who had platelet counts <100 × 10(9) /L at baseline, were given at least one 15 U/kg-dose increase during the extension. Trends indicative of improvement in platelet count and spleen volume, and decreasing levels of GD biomarkers, chitotriosidase and CCL18, were observed. Immunogenicity was seen in one patient pos...Continue Reading

References

Oct 1, 1994·Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research·H K GenantC Van Kuijk
Mar 1, 1994·The Journal of Clinical Investigation·C E HollakJ M Aerts
Aug 1, 1997·Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research·J Hanson
Sep 25, 1998·The Journal of Biological Chemistry·R G BootJ M Aerts
Oct 7, 2004·Seminars in Hematology·Gregory M PastoresAnna Tylki-Szymańska
Dec 1, 2006·Osteoporosis International : a Journal Established As Result of Cooperation Between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA·T N Hangartner
Apr 6, 2007·Clinica Chimica Acta; International Journal of Clinical Chemistry·Aricha SchoonhovenJohannes M F G Aerts
Feb 13, 2013·American Journal of Hematology·Hadhami Ben TurkiaAtul Mehta
Nov 5, 2013·Journal of Clinical Densitometry : the Official Journal of the International Society for Clinical Densitometry·John T SchousboeSanford Baim
Nov 23, 2013·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Gregory M PastoresDavid Zahrieh
Mar 13, 2014·The Journal of Obstetrics and Gynaecology Research·Deborah ElsteinAri Zimran

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