Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project

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 co...

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Detalles Bibliográficos
Autores: Giraldo P, Andrade-Campos M, Alfonso P, Irun P, Atutxa K, Acedo A, Barez A, Blanes M, Diaz-Morant V, Fernandez-Galan MA, Franco R, GIL, C., Giner V, Ibanez A, Latre P, Loyola I, Luno E, Hernandez-Martin R, Medrano-Engay B, Puerta J, Roig I, de la Serna J, Salamero O, Villalon L, Pocovi M
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2018
País:España
Institución:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
Repositorio:r-FISABIO. Repositorio Institucional de Producción Científica
OAI Identifier:oai:fisabio.fundanetsuite.com:p2366
Acceso en línea:https://fisabio.portalinvestigacion.com/publicaciones/2366
Access Level:acceso abierto
Palabra clave:Gaucher disease type 1
Miglustat
Efficacy
Safety
Maintenance
Descripción
Sumario: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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