%0 Journal Article
%A Giraldo, Pilar
%A Andrade-Campos, Marcio
%A Alfonso, Pilar
%A Irun, Pilar
%A Atutxa, Koldo
%A Acedo, Antonio
%A Barez, Abelardo
%A Blanes, Margarita
%A Diaz-Morant, Vicente
%A Fernández-Galán, Ma Angeles
%A Franco, Rafael
%A Gil-Cortes, Cristina
%A Giner, Vicente
%A Ibañez, Angela
%A Latre, Paz
%A Loyola, Ines
%A Luño, Elisa
%A Hernández-Martin, Roberto
%A Medrano-Engay, Blanca
%A Puerta, José
%A Roig, Inmaculada
%A de la Serna, Javier
%A Salamero, Olga
%A Villalón, Lucia
%A Pocovi, Miguel
%T Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
%D 2016
%U http://hdl.handle.net/10668/10599
%X 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
%K Efficacy
%K Gaucher disease type 1
%K Maintenance
%K Miglustat
%K Safety
%~