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Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
| Content Provider | Scilit |
|---|---|
| Author | Giraldo, Pilar Andrade-Campos, Marcio Alfonso, Pilar Irun, Pilar Atutxa, Koldo Acedo, Antonio Barez, Abelardo Blanes, Margarita Diaz-Morant, Vicente Fernández-Galán, Ma Angeles Franco, Rafael Gil-Cortes, Cristina Giner, Vicente Ibañez, Angela Latre, Paz Loyola, Ines Luño, Elisa Hernández-Martin, Roberto Medrano-Engay, Blanca Puerta, José Roig, Inmaculada de la Serna, Javier Salamero, Olga Villalón, Lucia Pocovi, Miguel |
| Copyright Year | 2018 |
| Description | Journal: Blood Cells, Molecules, and Diseases We report data from a prospective, observational study (ZAGAL) evaluating miglustat 100mg three times daily orally. in treatment-naïve 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-naïve; 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 long-term therapy in mild to moderate naïve and ERT stabilized patients. No unexpected safety signals were identified during 12-years follow-up. |
| Related Links | http://zaguan.unizar.es/record/78249/files/texto_completo.pdf |
| Ending Page | 179 |
| Page Count | 7 |
| Starting Page | 173 |
| ISSN | 10799796 |
| e-ISSN | 10960961 |
| DOI | 10.1016/j.bcmd.2016.10.017 |
| Journal | Blood Cells, Molecules, and Diseases |
| Volume Number | 68 |
| Language | English |
| Publisher | Elsevier BV |
| Publisher Date | 2018-02-01 |
| Access Restriction | Open |
| Subject Keyword | Journal: Blood Cells, Molecules, and Diseases Gaucher Disease Type 1 |
| Content Type | Text |
| Resource Type | Article |
| Subject | Cell Biology Hematology Molecular Biology Molecular Medicine |
