Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications
Open Access
- 24 July 2014
- journal article
- Published by Springer Science and Business Media LLC in Orphanet Journal of Rare Diseases
- Vol. 9 (1), 112
- https://doi.org/10.1186/s13023-014-0112-x
Abstract
Long-term complications and associated conditions of type 1 Gaucher Disease (GD) can include splenectomy, bone complications, pulmonary hypertension, Parkinson disease and malignancies. Enzyme replacement therapy (ERT) reverses cytopenia and reduces organomegaly. To study the effects of ERT on long-term complications and associated conditions, the course of Gaucher disease was modelled.Keywords
This publication has 64 references indexed in Scilit:
- Malignancies and monoclonal gammopathy in Gaucher disease; a systematic review of the literatureBritish Journal of Haematology, 2013
- Liver Fibrosis in Type I Gaucher Disease: Magnetic Resonance Imaging, Transient Elastography and Parameters of Iron StoragePLOS ONE, 2013
- Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 studyBlood, 2010
- Expanding spectrum of the association between Type 1 Gaucher disease and cancers: A series of patients with up to 3 sequential cancers of multiple types—Correlation with genotype and phenotypeAmerican Journal of Hematology, 2010
- Bone events and evolution of biologic markers in Gaucher disease before and during treatmentArthritis Research & Therapy, 2010
- Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosisBritish Journal of Haematology, 2009
- The underrecognized progressive nature of N370S Gaucher disease and assessment of cancer risk in 403 patientsAmerican Journal of Hematology, 2009
- Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48‐month longitudinal cohort studyClinical Genetics, 2008
- Occurrence of Parkinson's syndrome in type 1 Gaucher diseaseQJM: An International Journal of Medicine, 1996
- Gaucher's Disease of the Lung Causing Severe Pulmonary Hypertension with Associated Acute Recurrent PericarditisCirculation, 1967