期刊
BLOOD CELLS MOLECULES AND DISEASES
卷 68, 期 -, 页码 173-179出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.bcmd.2016.10.017
关键词
Gaucher disease type 1; Miglustat; Efficacy; Safety; Maintenance
类别
资金
- FEETEG [01-04]
- FIS [EC07/90737, 07/90938, PS09/02556, PS12/01219]
- Actelion Pharmaceuticals Ltd.
- Spanish Gaucher Disease Foundation (FEETEG)
We report data from a prospective, observational study (ZAGAL) evaluating miglustat 100 mg three times daily orally. in treatment-naive patients and patients with type 1 Gaucher Disease (GD1) switched from previous enzyme replacement therapy (ERT). Clinical evolution, changes in organ size, blood counts, disease biomarkers, bone marrow infiltration (S-MRI), bone mineral density by broadband ultrasound densitometry (BMD), safety and tolerability annual reports were analysed. Between May 2004 and April 2016, 63 patients received miglustat therapy; 20 (32%) untreated and 43 (68%) switched. At the time of this report 39 patients (14 [36%] treatment-naive; 25 [64%] switch) remain on miglustat. With over 12-year follow-up, hematologic counts, liver and spleen volumes remained stable. In total, 80% of patients achieved current GD1 therapeutic goals. Plasma chitotriosidase activity and CCL-18/PARC concentration showed a trend towards a slight increase. Reductions on S-MRI (p=0.042) with an increase in BMD (p < 0.01) were registered. Gastrointestinal disturbances were reported in 25/63 (40%), causing miglustat suspension in 11/63 (17.5%) cases. Thirty-eight patients (60%) experienced a fine hand tremor and two a reversible peripheral neuropathy. Overall, miglustat was effective as a longtermtherapy in mild to moderate naive and ERT stabilized patients. No unexpected safety signals were identified during 12-years follow-up. (C) 2016 Elsevier Inc. All rights reserved.
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