Gaucher disease type I : associated morbidities and long term efficacy of enzyme replacement therapy

Thesis

Titlepage

Contents

Chapter 1. Introduction

Chapter 2. Low frequency maintenance therapy with imiglucerase in adult type I Gaucher disease: a prospective randomized controlled trial

Chapter 3. Superior effects of high dose enzyme replacement therapy in type I Gaucher disease on bone marrow involvement and chitotriosidase levels; a two center retrospective analysis

Chapter 4. Persistent bone disease in adult type I Gaucher disease despite increasing doses of enzyme replacement therapy

Chapter 5. Marked Elevation of the Chemokine CCL18 / PARC in Gaucher disease; A novel surrogate marker for assessing therapeutic intervention

Chapter 6. The plasma level of the macrophage-derived soluble CD163 is increased and positively correlates with severity in Gaucher disease

Chapter 7. Increased plasma macrophage inflammatory protein (MIP)-1α and MIP-1β levels in type I Gaucher disease

Chapter 8. Correction in plasma macrophage inflammatory protein (MIP)-1β is dose dependent during the initial phase of enzyme replacement therapy of type I Gaucher disease

Chapter 9. Low HDL levels in Gaucher disease type I does not lead to an increased risk of cardio vascular disease as assessed by carotid intima-media thickness

Chapter 10. Increased incidence of cancer in adult Gaucher disease in Western Europe

Chapter 11. Immunoglobulin and free light chain abnormalities in Gaucher disease type I; data from an adult cohort of 63 patients and review of the literature

Chapter 12. Summary

Chapter 12. Nederlandse samenvatting

Chapter 13. Discussion

Chapter 14. Dankwoord

Appendix