title: Booster-effect with velaglucerase alfa in patients with Gaucher disease switched from long-term imiglucerase therapy: early Access Program results from Jerusalem.
abstract: BACKGROUND
  Decreased spleen and liver volumes and increased hemoglobin levels and platelet counts usually occur with enzyme replacement therapy (ERT) in symptomatic patients with Gaucher disease. Because of decreased supply of imiglucerase, an FDA-approved Early Access Program (EAP) allowed use of a new, pre-licensed ERT, velaglucerase alfa. This report provides safety and efficacy findings in patients on EAP velaglucerase alfa who completed 6, 9, or 12 months as intravenous every-other-week ERT.
  
  
  METHODS
  EAP was approved by the Israeli Ministry of Health. All patients enrolled in the EAP were included for safety measures; only those with >6 month evaluations of hemoglobin, platelet counts, and liver and spleen volumes were included for efficacy. Descriptive statistics were employed.
  
  
  RESULTS
  Among 71 EAP patients, there were no drug-related serious adverse events or withdrawals; one patient (1.4%) with previous hypersensitivity to a different ERT had a drug-related allergic reaction. Of 44 patients with appropriate time-period evaluations, 8 patients were treatment-naïve and responded well to velaglucerase alfa. The 36 switch-over patients remained at imiglucerase low-doses; a majority of patients showed improvements in each efficacy parameter.
  
  
  CONCLUSIONS
  Switch-over from imiglucerase (10-224 months) was safe and in several patients velaglucerase alfa induced a booster-effect.
authors: Gaucher Clinic, Shaare Zedek Medical Center, the Hebrew University and Hadassah Medical School, Jerusalem, Israel. elstein@szmc.org.il
authors: Deborah
authors: D
authors: Elstein
authors: Gheona
authors: G
authors: Altarescu
authors: Hannah
authors: H
authors: Maayan
authors: Mici
authors: M
authors: Phillips
authors: Aya
authors: A
authors: Abrahamov
authors: Irith
authors: I
authors: Hadas-Halpern
authors: Maayan
authors: M
authors: Tiomkin
authors: Ari
authors: A
authors: Zimran
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001792:Blood Platelets / Q000378:metabolism; D002648:Child; D002675:Child, Preschool; D057915:Drug Substitution; D064420:Drug-Related Side Effects and Adverse Reactions; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000201:enzymology / Q000473:pathology; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000627:therapeutic use; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007557:Israel; D008099:Liver / Q000187:drug effects / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009929:Organ Size / Q000187:drug effects; D013154:Spleen / Q000187:drug effects / Q000473:pathology
chemical_list: D006454:Hemoglobins; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human; C090568:imiglucerase