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Title: Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
Authors: 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
Keywords: Efficacy;Gaucher disease type 1;Maintenance;Miglustat;Safety
metadata.dc.subject.mesh: 1-Deoxynojirimycin
Follow-Up Studies
Gaucher Disease
Glycoside Hydrolase Inhibitors
Middle Aged
Organ Size
Prospective Studies
Young Adult
Issue Date: 24-Oct-2016
Abstract: 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
metadata.dc.identifier.doi: 10.1016/j.bcmd.2016.10.017
Appears in Collections:Producción 2020

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