Correlation of Bone Marrow Response with Hematological, Biochemical, and Visceral Responses to Enzyme Replacement Therapy of Nonneuronopathic (Type 1) Gaucher Disease in 30 Adult Patients

2002 ◽  
Vol 28 (2) ◽  
pp. 209-220 ◽  
Author(s):  
Ludger Wilhelm Poll ◽  
Jens-Albrecht Koch ◽  
Reinhart Willers ◽  
Hans Aerts ◽  
Axel Scherer ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Adult Patients ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease

Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis

Blood ◽  
2006 ◽  
Vol 108 (3) ◽  
pp. 830-835 ◽  
Author(s):  
Maaike de Fost ◽  
Carla E. M. Hollak ◽  
Johanna E. M. Groener ◽  
Johannes M. F. G. Aerts ◽  
Mario Maas ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Bone Marrow Involvement ◽  
Median Dose ◽  
Long Term Outcome ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease

AbstractDosing of enzyme replacement therapy (ERT) for Gaucher disease type 1 is still a subject of debate and varies from 15 to 130 U/kg/mo, making a huge economic difference of US $70 000 to US $380 000 (€55 000-300 000) per patient per year. To investigate whether this difference in dosing ultimately translates into a different response, we retrospectively compared long-term outcome of ERT at 2 large European treatment centers, Academic Medical Center, Amsterdam, The Netherlands (n = 49, median dose, 15-30 U/kg/4 wks) and Heinrich-Heine University, Duesseldorf, Germany (n = 57, median dose, 80 U/kg/4 wks). These adult cohorts had a similar genetic background. All follow-up parameters were matched separately at baseline, to avoid bias with respect to disease severity. Improvement in hemoglobin, platelet count, and hepatosplenomegaly was not significantly different between both cohorts, whereas plasma chitotriosidase and bone marrow involvement by magnetic resonance imaging improved more quickly and was more pronounced in the higher-dosed group. Major bone complications rarely occurred in both groups. In conclusion, different dosing regimens of ERT do not affect outcome of hematologic and visceral parameters, but higher dosing leads to accelerated decrease of chitotriosidase and better objective bone response in adult type 1 Gaucher disease.

scholarly journals Persistent bone disease in adult type 1 Gaucher disease despite increasing doses of enzyme replacement therapy

Haematologica ◽  
2008 ◽  
Vol 93 (7) ◽  
pp. 1119-1120 ◽  
Author(s):  
M. de Fost ◽  
C. J.M. van Noesel ◽  
J. M.F.G. Aerts ◽  
M. Maas ◽  
R. G. Poll ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Bone Disease ◽  
Gaucher Disease ◽  
Adult Type ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease

scholarly journals Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease

2014 ◽  
Vol 9 (1) ◽  
pp. 51 ◽  
Author(s):  
Laura van Dussen ◽  
Marieke Biegstraaten ◽  
Carla EM Hollak ◽  
Marcel GW Dijkgraaf
Keyword(s):  
Cost Effectiveness ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease

Improvement of Splenomegaly and Pancytopenia by Enzyme Replacement Therapy Against Type 1 Gaucher Disease: A Report of Sibling Cases

2001 ◽  
Vol 73 (3) ◽  
pp. 356-362
Author(s):  
Kazuya Tsuboi ◽  
Shinsuke Iida ◽  
Miyuki Kato ◽  
Yoshihito Hayami ◽  
Ichiro Hanamura ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Enzyme Replacement ◽  
Type 1 Gaucher Disease ◽  
Sibling Cases

Regression of skeletal changes in type 1 Gaucher disease with enzyme replacement therapy

1996 ◽  
Vol 25 (5) ◽  
pp. 485-488 ◽  
Author(s):  
Gregory M. Pastores ◽  
G. Hermann ◽  
Karen I. Norton ◽  
Mordechai Lorberboym ◽  
Robert J. Desnick
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Enzyme Replacement ◽  
Skeletal Changes ◽  
Type 1 Gaucher Disease
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