Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
Por:
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 and Pocovi M
Publicada:
1 feb 2018
Ahead of Print:
24 oct 2016
Resumen:
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.
Filiaciones:
Giraldo P:
Haematology, Miguel Servet University Hospital, Zaragoza, Spain
CIBER de Enfermedades Raras (CIBERER), Spain
Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG), Spain
Institute of Health Research Aragon (IIS Aragon), Zaragoza, Spain
Andrade-Campos M:
Haematology, Miguel Servet University Hospital, Zaragoza, Spain
CIBER de Enfermedades Raras (CIBERER), Spain
Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG), Spain
Institute of Health Research Aragon (IIS Aragon), Zaragoza, Spain
Alfonso P:
CIBER de Enfermedades Raras (CIBERER), Spain
Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG), Spain
Irun P:
CIBER de Enfermedades Raras (CIBERER), Spain
Biochemistry and Molecular and Cellular Biology, Zaragoza University, Spain
Atutxa K:
Haematology, Galdakao Hospital, Usansolo, Spain
:
Haematology, Vega Baja Hospital, Alicante, Spain
Barez A:
Haematology, Ntra Sra de Sonsoles Hospital, Avila, Spain
Blanes M:
Haematology, Virgen de la Salud Hospital, Elda, Spain
Diaz-Morant V:
Internal Medicine, Ronda Hospital, Ronda, Spain
Fernandez-Galan MA:
Virgen del Puerto Hospital, Plasencia, Spain
Franco R:
Haematology, Punta Europa University Hospital, Cadiz, Spain
Gil C:
Haematology, San Vicente Hospital, Alicante, Spain
:
Internal Medicine, Virgen de Los Lirios Hospital, Alcoy, Spain
Ibanez A:
Haematology, Complejo Hospitalario de Albacete, Spain
Latre P:
Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG), Spain
Loyola I:
Haematology, Complexo Hospitalario de Pontevedra, Spain
Luno E:
Haematology, Central de Asturias University Hospital, Oviedo, Spain
Hernandez-Martin R:
Haematology, Virgen de la Concha Hospital, Zamora, Spain
Medrano-Engay B:
Institute of Health Research Aragon (IIS Aragon), Zaragoza, Spain
Puerta J:
Haematology, Virgen de las Nieves Hospital. Granada, Spain
Roig I:
Haematology, Parc Tauli Hospital, Sabadell, Spain
de la Serna J:
Haematology, Doce de Octubre University Hospital, Madrid, Spain
Salamero O:
Haematology, Vall d'Hebron University Hospital, Barcelona, Spain
Villalon L:
Haematology, Alcorcon Hospital, Madrid, Spain
Pocovi M:
Institute of Health Research Aragon (IIS Aragon), Zaragoza, Spain
Biochemistry and Molecular and Cellular Biology, Zaragoza University, Spain
Green Accepted
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