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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Detalhes 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 documento: artigo
Data de publicação:2018
País:España
Recursos:Servizo Galego de Saúde (SERGAS)
Repositório:RUNA. Repositorio da Consellería de Sanidade e Sergas
OAI Identifier:oai:runa.sergas.gal:20.500.11940/10041
Acesso em linha: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 aberto
Palavra-chave: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
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spelling Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL projectGiraldo, PilarAndrade-Campos, MarcioAlfonso, PilarIrun, PilarAtutxa, KoldoAcedo, AntonioBarez, AbelardoBlanes, MargaritaDiaz-Morant, VicenteFernández-Galán, Ma AngelesFranco, RafaelGil-Cortes, CristinaGiner, VicenteIbañez, AngelaLatre, PazLoyola Holgado, InésLuño, ElisaHernández-Martin, RobertoMedrano-Engay, BlancaPuerta, JoséRoig, Inmaculadade la Serna, JavierSalamero, OlgaVillalón, LuciaPocovi, MiguelEnzyme Replacement Therapy1-DeoxynojirimycinGlucosylceramidaseGaucher Disease1-desoxinojirimicinaenfermedad de Gaucherglucosilceramidasatratamiento de sustitución enzimáticaMiglustatEnfermedad de GaucherEnfermidade de GaucherWe 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.This work was partially sponsored by a grant from FEETEG (01-04), FIS: EC07/90737, 07/90938, PS09/02556, and PS12/01219. PG has received research funding and consultancy fees from Actelion Pharmaceuticals Ltd. All authors are members of the Spanish Study Group on Gaucher Disease, which is supported by the Spanish Gaucher Disease Foundation (FEETEG)Fundación Española para el Estudio y Tratamiento de la Enfermedad de Gaucher y otras Lisosomales (FEETEG)Instituto de Salud Carlos IIIActelion Pharmaceuticals Ltd2018info:eu-repo/semantics/articlehttps://apps.webofknowledge.com/full_record.do?product=WOS&search_mode=GeneralSearch&qid=1&SID=D6vfTuPoMgeq6rPKSZy&page=1&doc=1https://www.ncbi.nlm.nih.gov/pubmed/27836529http://hdl.handle.net/20.500.11940/10041reponame:RUNA. Repositorio da Consellería de Sanidade e Sergasinstname:Servizo Galego de Saúde (SERGAS)Inglésinfo:eu-repo/semantics/openAccessoai:runa.sergas.gal:20.500.11940/100412026-06-12T08:40:47Z
dc.title.none.fl_str_mv Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
title Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
spellingShingle Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
Giraldo, Pilar
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
title_short Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
title_full Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
title_fullStr Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
title_full_unstemmed Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
title_sort Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project
dc.creator.none.fl_str_mv 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
author Giraldo, Pilar
author_facet 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
author_role author
author2 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
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv 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
topic 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
description 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.
publishDate 2018
dc.date.none.fl_str_mv 2018
dc.type.none.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv 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
url 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
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.source.none.fl_str_mv reponame:RUNA. Repositorio da Consellería de Sanidade e Sergas
instname:Servizo Galego de Saúde (SERGAS)
instname_str Servizo Galego de Saúde (SERGAS)
reponame_str RUNA. Repositorio da Consellería de Sanidade e Sergas
collection RUNA. Repositorio da Consellería de Sanidade e Sergas
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,300719

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