2004
DOI: 10.1053/j.seminhematol.2004.07.010
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Gaucher disease type 1: Revised recommendations on evaluations and monitoring for adult patients

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Cited by 152 publications
(126 citation statements)
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“…In children and adults with diverse presentations of disease, treatment with intravenous macrophage-targeted enzyme replacement therapy (imiglucerase, Cerezyme 1 , Genzyme Corporation) has been shown to abate anemia, thrombocytopenia, and organomegaly and to impede the progression of bone disease [10]. Imiglucerase has been the standard of care for the treatment of patients with GD1 since its introduction in 1994 [11]. In some countries, an oral therapy based on the principle of reducing substrate load (miglustat, Zavesca 1 , Actelion Pharmaceuticals, Ltd.) is licensed for adult patients with mild to moderate GD1 for whom enzyme replacement therapy is not a therapeutic option due to allergy, hypersensitivity, or poor venous access [12].…”
Section: Introductionmentioning
confidence: 99%
See 1 more Smart Citation
“…In children and adults with diverse presentations of disease, treatment with intravenous macrophage-targeted enzyme replacement therapy (imiglucerase, Cerezyme 1 , Genzyme Corporation) has been shown to abate anemia, thrombocytopenia, and organomegaly and to impede the progression of bone disease [10]. Imiglucerase has been the standard of care for the treatment of patients with GD1 since its introduction in 1994 [11]. In some countries, an oral therapy based on the principle of reducing substrate load (miglustat, Zavesca 1 , Actelion Pharmaceuticals, Ltd.) is licensed for adult patients with mild to moderate GD1 for whom enzyme replacement therapy is not a therapeutic option due to allergy, hypersensitivity, or poor venous access [12].…”
Section: Introductionmentioning
confidence: 99%
“…Patients with other chronic multisystem disorders, e.g., asthma, diabetes mellitus, and congestive heart failure, have benefited from the introduction and implementation of protocols for disease management including definition of quantifiable therapeutic goals [13][14][15][16][17]. For GD1, therapeutic goals believed to be clinically meaningful, but not necessarily equivalent to normal values [18], and monitoring guidelines [11] have been proposed in the context of a disease management algorithm that may be applied to current and future treatments (see Fig. 1).…”
Section: Introductionmentioning
confidence: 99%
“…Dans la maladie de Gaucher, l'efficacité thérapeutique de l'enzymothérapie sur l'asthénie, l'hépatosplénomégalie, la thrombopénie et l'anémie est remarquable. Un certain nombre d'interrogations subsistent sur la réponse de l'os à l'enzymothérapie, qui nécessite souvent plusieurs années de traitement et reste parfois incomplète [9,10]. Certaines questions sont intéressantes à discuter, comme la dose de maintenance adéquate et la fréquence optimale d'administration de l'imiglucérase [11].…”
Section: Maladie De Gaucherunclassified
“…Quelle est la dose d'α-glucosidase recombinante optimale à administrer ? La question de la production en quantité suffisante de l'enzyme pourrait se poser, puisque les doses perfusées sont considérables (10,20, voire 40 mg de protéines par kilo de poids). Dans le même temps, de nombreux patients adultes sont par ailleurs actuellement en déshérence de soins avec l'espoir de recevoir un jour cette thérapeutique spécifique disponible pour les enfants.…”
Section: Glycogénose De Type II Ou Maladie De Pompeunclassified
“…Based on its review of both published and unpublished data, the Canadian Expert Drug Advisory Committee (CEDAC), on 24 November 2004, made its final recommendation that miglustat not be listed as a therapeutic option for Canadian patients with GD [21]. CEDAC concluded that there was insufficient evidence that miglustat has a clinically meaningful impact on the key hematologic and bone complications and that there is insufficient evidence to show whether treatment reduces fatigue, bone pain, bone complications, or blood product transfusion requirements.…”
Section: Summary Of Statements From the European United States And mentioning
confidence: 99%