title: Evaluation of treatment response to enzyme replacement therapy with Velaglucerase alfa in patients with Gaucher disease using whole-body magnetic resonance imaging.
abstract: OBJECTIVE
  This was a retrospective data analysis to evaluate the treatment response to enzyme replacement therapy (ERT) with Velaglucerase alfa using whole-body magnetic resonance imaging (MRI).
  
  
  METHODS
  A baseline and follow-up MRI were performed on 18 Gaucher Type 1 patients at an interval of 11.6 months. The MRI score systems determined the Bone-Marrow-Burden (BMB) score, the Düsseldorf-Gaucher score (DGS), and the Vertebra-Disc-Ratio (VDR). The Severity Score Index Type 1 (GD-DS3) was also assessed.
  
  
  RESULTS
  The baseline MRI medians were: BMB, 7.00; DGS, 3.00; and VDR: 1.70; while, the follow-up MRI medians were: BMB, 7.00; DGS, 3.00; and VDR: 1.73. The baseline GD-DS3 median was 2.40 (BMB excl.: 0.50) and the follow-up median was 2.00 (BMB excl.: 0.50). There was weak statistical significance with the Wilcoxon signed-rank test for the DGS (p=0.034) and GD-DS3 (p=0.047) between both MRIs.
  
  
  CONCLUSIONS
  Velaglucerase alfa therapy is a effective long-term treatment for Gaucher Type 1 patients who are newly diagnosed or switching therapies. Measurements with whole-body MRI and an objective scoring system were reliable tools for detecting early stage bone marrow activity. Further research is needed to evaluate the "Booster-Effect" of Velaglucerase alfa therapy in Gaucher skeletal disease.
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany. Electronic address: katharina.laudemann@unimedizin-mainz.de.
authors: K
authors: K
authors: Laudemann
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: L
authors: L
authors: Moos
authors: Villa Metabolica, Center of Pediatric and Adolescent Medicine, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: E
authors: E
authors: Mengel
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: A
authors: A
authors: Lollert
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: C
authors: C
authors: Hoffmann
authors: Villa Metabolica, Center of Pediatric and Adolescent Medicine, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: M
authors: M
authors: Brixius-Huth
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: D
authors: D
authors: Wagner
authors: Department of Diagnostic and Interventional Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: C
authors: C
authors: Düber
authors: Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg University, Mainz, Germany.
authors: G
authors: G
authors: Staatz
mesh_terms: D000328:Adult; D000368:Aged; D001853:Bone Marrow / Q000187:drug effects / Q000473:pathology; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000175:diagnosis* / Q000188:drug therapy* / Q000473:pathology; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods*; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D011994:Recombinant Proteins / Q000627:therapeutic use; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome; D051598:Whole Body Imaging / Q000379:methods
chemical_list: D011994:Recombinant Proteins; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human
keywords: Gaucher disease; Score systems; Velaglucerase alfa; Whole-body MRI