RT Journal Article
T1 Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
A1 Giraldo, Pilar
A1 Andrade-Campos, Marcio
A1 Alfonso, Pilar
A1 Irun, Pilar
A1 Atutxa, Koldo
A1 Acedo, Antonio
A1 Barez, Abelardo
A1 Blanes, Margarita
A1 Diaz-Morant, Vicente
A1 Fernández-Galán, Ma Angeles
A1 Franco, Rafael
A1 Gil-Cortes, Cristina
A1 Giner, Vicente
A1 Ibañez, Angela
A1 Latre, Paz
A1 Loyola, Ines
A1 Luño, Elisa
A1 Hernández-Martin, Roberto
A1 Medrano-Engay, Blanca
A1 Puerta, José
A1 Roig, Inmaculada
A1 de la Serna, Javier
A1 Salamero, Olga
A1 Villalón, Lucia
A1 Pocovi, Miguel
K1 Efficacy
K1 Gaucher disease type 1
K1 Maintenance
K1 Miglustat
K1 Safety
AB 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
YR 2016
FD 2016-10-24
LK http://hdl.handle.net/10668/10599
UL http://hdl.handle.net/10668/10599
LA en
DS RISalud
RD Apr 6, 2025