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dc.contributor.authorΜαρινάκης, Θ.ell
dc.contributor.authorΡέπα, Κ.ell
dc.contributor.authorΚαρκαλούσος, Πέτροςell
dc.contributor.authorΜιχελάκης, Χ.ell
dc.identifier.citationMarinakis, T., Repa, C., Karkalousos, P. and Michelakakis, H. (2004) Extended interval between enzyme therapy infusions for adult patients with Gaucher’s disease type 1. In "Sixth EWGGD Workshop". Barcelona: The European Working Group on Gaucher Disease.eng
dc.rightsΑναφορά Δημιουργού-Μη Εμπορική Χρήση-Όχι Παράγωγα Έργα 3.0 Ηνωμένες Πολιτείες*
dc.subjectEnzyme replacement therapy (ERT)-
dc.subjectMedicine, Biochemic-
dc.subjectΘεραπεία ενζυμικής υποκατάστασης-
dc.subjectΒιοχημική ιατρική-
dc.titleExtended interval between enzyme therapy infusions for adult patients with Gaucher’s disease type 1eng
dc.typeΔημοσίευση σε συνέδριο-
heal.recordProviderΣχολή Επαγγελμάτων Υγείας και Πρόνοιας. Τμήμα Ιατρικών Εργαστηρίωνell
heal.abstractIndroduction: Enzyme replacement therapy (ERT) with intravenous infusions of the enzyme, alglucerase or imiglucerase, has proved to be efficacious in ameliorating or reversing many disease manifestations in adult patients with Gaucher’s disease (GD) including hematological, visceral and skeletal abnormalities and has led to improved overall health and quality of life. This therapy is well tolerated and safe. However financial aspects and difficulties to persuade some patients to continue the enzyme application for a long term leads in some cases to the discontinuation of therapy. Purpose: The purpose of our study was to investigate whether an extention of the interval between enzyme infusions to once every three weeks or four weeks could be effective in maintaining the clinical responses achieved with the twice monthly regimen. Seven consecutive patients with GD type 1 without bone disease were subjected to enzyme dose/frequency changes. Their characteristics and enzyme dosage at the beginning of therapy are seen at the table below: [table] Patients were assessed every six months for alterations in hematological parameters, liver and spleen size and bone symptoms. Results: A) Pts no 1,2,3,4: After a period of 6-12 months with a satisfactory response we could decrease the dosage and after a period of 19-24 months, when hemoglobin values were normal, platelet values>100.000/mm3 and a mean decrease of splenomegaly and hepatomegaly was observed 50% and 30% respectively, they could continue with 15–30 U/Kg every 4 weeks. The disease was overall stable or ameliorated for the next 2 to 4 years, hence these patients now continue with 15 U/Kg every 4 weeks. B) Pts no 5,6,7: After an optimal response in hemoglobin, platelets count, liver and spleen size, they switched to the same dose every 4 weeks. After a period of 6-10 months we observed a recurrence of the disease (decrease of hemoglobin) and we changed again the dosage and the frequency. Conclusions: Our limited experience suggests in some adult patients the interval between enzyme therapy infusions can be extended. Further studies are needed to define the specific patients in whom an extended infusion interval or a decrease of dosages could be safely applied.en
heal.publisherThe European Working Group on Gaucher Diseaseeng
heal.conferenceNameSixth EWGGD Workshopeng
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