title: Effects of switching from a reduced dose imiglucerase to velaglucerase in type 1 Gaucher disease: clinical and biochemical outcomes.
abstract: This paper describes the effects of a switch to velaglucerase alfa in a group of adult patients with type 1 Gaucher disease, all of whom had previously had their dose reduced as a consequence of the worldwide imiglucerase shortage. Thirty-two patients from two large European Gaucher centers switched to treatment with velaglucerase alfa after 1-8.5 months of dose reduction. The course of important Gaucher disease parameters was studied at four time points: one year before the shortage, just before the shortage, before a switch to velaglucerase and after up to one year of treatment with velaglucerase. These parameters included hemoglobin concentration, platelet count, plasma chitotriosidase activity in all patients, and spleen and liver volumes (as well as bone marrow fat fraction images) in 10 patients. Decreases in platelet counts as a result of reduced treatment with imiglucerase were quickly restored on treatment with velaglucerase alfa. Chitotriosidase activity declined overall after switching. Five out of 10 patients had an increase in liver volume of at least 10% after six months of velaglucerase treatment, which was reversible in 3. Most patients received infusions at home and no important side effects were observed. Velaglucerase alfa appears to be a safe and effective alternative for imiglucerase.
authors: Department of Endocrinology and Metabolism, Academic Medical Centre, Amsterdam, The Netherlands.
authors: Laura
authors: L
authors: van Dussen
authors: Timothy M
authors: TM
authors: Cox
authors: Erik J
authors: EJ
authors: Hendriks
authors: Elizabeth
authors: E
authors: Morris
authors: Erik M
authors: EM
authors: Akkerman
authors: Mario
authors: M
authors: Maas
authors: Johanna E M
authors: JE
authors: Groener
authors: Johannes M F G
authors: JM
authors: Aerts
authors: Patrick B
authors: PB
authors: Deegan
authors: Carla E M
authors: CE
authors: Hollak
mesh_terms: D000328:Adult; D000368:Aged; D056947:Enzyme Replacement Therapy; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000201:enzymology* / Q000473:pathology / Q000628:therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006454:Hemoglobins / Q000378:metabolism; D006596:Hexosaminidases / Q000378:metabolism; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012189:Retrospective Studies; D013156:Splenectomy
chemical_list: D006454:Hemoglobins; D006596:Hexosaminidases; C086400:chitotriosidase; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human; C090568:imiglucerase