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 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 cou...

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Detalles Bibliográficos
Autores: 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 Holgado, Inés, Luño, Elisa, Hernández-Martin, Roberto, Medrano-Engay, Blanca, Puerta, José, Roig, Inmaculada, de la Serna, Javier, Salamero, Olga, Villalón, Lucia, Pocovi, Miguel
Tipo de recurso: artículo
Fecha de publicación:2018
País:España
Institución:Servizo Galego de Saúde (SERGAS)
Repositorio:RUNA. Repositorio da Consellería de Sanidade e Sergas
OAI Identifier:oai:runa.sergas.gal:20.500.11940/10041
Acceso en línea:https://apps.webofknowledge.com/full_record.do?product=WOS&search_mode=GeneralSearch&qid=1&SID=D6vfTuPoMgeq6rPKSZy&page=1&doc=1
https://www.ncbi.nlm.nih.gov/pubmed/27836529
http://hdl.handle.net/20.500.11940/10041
Access Level:acceso abierto
Palabra clave:Enzyme Replacement Therapy
1-Deoxynojirimycin
Glucosylceramidase
Gaucher Disease
1-desoxinojirimicina
enfermedad de Gaucher
glucosilceramidasa
tratamiento de sustitución enzimática
Miglustat
Enfermedad de Gaucher
Enfermidade de Gaucher
Descripción
Sumario: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.

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