BMSEHA15
Published online 16 July 2009
Haematologica, Vol 94, Issue 12, 1771-1775 doi:10.3324/haematol.2009.008078
Copyright © 2009 by Ferrata Storti Foundation
This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow Giraldo et al. Supplementary Appendix
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Citing Articles
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by Giraldo, P.
Right arrow Articles by Pocovi, M.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Giraldo, P.
Right arrow Articles by Pocovi, M.

Gaucher Disease

Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project

Pilar Giraldo1,2,9,11, Pilar Alfonso2,11, Koldo Atutxa3, María A. Fernández-Galán4, Abelardo Barez5, Rafael Franco6, Dora Alonso7, Alejandro Martin8, Paz Latre9, Miguel Pocovi1,2,9,11

1 Haematology Department, Miguel Servet University Hospital, Zaragoza
2 CIBER de Enfermedades Raras (CIBERER)
3 Haematology Department, Basurto, Bilbao
4 Haematology Department, Virgen del Puerto Plasencia, Plasencia
5 Haematology Department, Ntra Sra de Sonsoles Hospital, Avila
6 Haematology Department, Punta Europa University Hospital, Cádiz
7 Haematology Department, Virgen del Rocío University Hospital, Sevilla
8 Haematology Department, Virgen de la Concha Hospital, Zamora
9 Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG)
10 Biochemistry and Molecular and Cellular Biology Department, Zaragoza University, Zaragoza
11 Aragon Institute of Health Sciences (I+CS), Zaragoza, Spain

Correspondence: Pilar Giraldo, Sº Hematología, Hospital Universitario Miguel Servet, Pº Isabel La Católica 1–3, 50006 Zaragoza, Spain., E-mail: pgiraldo{at}salud.aragon.es

There are few published data from real-world clinical experience with miglustat (Zavesca®), an oral inhibitor of glucosylceramide synthase, in type 1 Gaucher disease. We report data from a prospective, open-label investigational study that evaluated substrate reduction therapy with miglustat 100 mg t.i.d. as a maintenance therapy in patients with Type 1 Gaucher disease who had been switched from previous enzyme replacement therapy. Long-term data on changes in organ size, blood counts, disease severity bio-markers, bone marrow infiltration, overall clinical status and safety/tolerability were analyzed from 28 patients with Type 1 Gaucher disease who were attending routine clinic visits. Assessments were performed at six, 12, 24, 36 and 48 months of therapy. Disease severity biomarkers improved up to 48 months after initiation of miglustat, while other disease parameters remained stable. Miglustat showed an acceptable profile of safety and tolerability throughout treatment. In conclusion, miglustat is an effective therapy for the long-term maintenance of patients with Type 1 Gaucher disease previously stabilized with enzyme replacement therapy.

Key words: type 1 Gaucher disease, substrate reduction therapy, efficacy and safety, maintenance, real clinical setting.