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Management and moni...
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe
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- Belmatoug, Nadia (author)
- Beaujon Hospital
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- Di Rocco, Maja (author)
- Gaslini Children's Hospital
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- Fraga, Cristina (author)
- Hospital do Divino Espírito Santo
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- Giraldo, Pilar (author)
- Biomedical Network on Rare Diseases (CIBERER)
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- Hughes, Derralynn (author)
- Royal Free Hospital
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- Lukina, Elena (author)
- National Research Center for Hematology
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- Maison-Blanche, Pierre (author)
- Hopital Bichat-Claude-Bernard AP-HP
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- Merkel, Martin (author)
- Asklepios Klinik St. Georg
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- Niederau, Claus (author)
- University of Duisburg-Essen
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- Plöckinger, Ursula (author)
- Charité - University Medicine Berlin
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- Richter, Johan (author)
- Lund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine,Skåne University Hospital
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- Stulnig, Thomas M. (author)
- Medical University of Vienna
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vom Dahl, Stephan (author)
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- Cox, Timothy M. (author)
- University of Cambridge
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(creator_code:org_t)
- Elsevier BV, 2017
- 2017
- English.
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In: European Journal of Internal Medicine. - : Elsevier BV. - 0953-6205. ; 37, s. 25-32
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Abstract
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- Purpose: In Gaucher disease, diminished activity of the lysosomal enzyme, acid β-glucosidase, leads to accumulation of glucosylceramides and related substrates, primarily in the spleen, liver, and bone marrow. Eliglustat is an oral substrate reduction therapy approved in the European Union and the United States as a first-line treatment for adults with type 1 Gaucher disease who have compatible CYP2D6 metabolism phenotypes. A European Advisory Council of experts in Gaucher disease describes the characteristics of eliglustat that are distinct from enzyme augmentation therapy (the standard of care) and miglustat (the other approved substrate reduction therapy) and recommends investigations and monitoring for patients on eliglustat therapy within the context of current recommendations for Gaucher disease management. Results: Eliglustat is a selective, potent inhibitor of glucosylceramide synthase, the enzyme responsible for biosynthesis of glucosylceramides which accumulate in Gaucher disease. Extensive metabolism of eliglustat by CYP2D6, and, to a lesser extent, CYP3A of the cytochrome P450 pathway, necessitates careful consideration of the patient's CYP2D6 metaboliser status and use of concomitant medications which share metabolism by these pathways. Guidance on specific assessments and monitoring required for eliglustat therapy, including an algorithm to determine eligibility for eliglustat, are provided. Conclusions: As a first-line therapy for type 1 Gaucher disease, eliglustat offers eligible patients a daily oral therapy alternative to biweekly infusions of enzyme therapy. Physicians will need to carefully assess individual Gaucher patients to determine their appropriateness for eliglustat therapy. The therapeutic response to eliglustat and use of concomitant medications will require long-term monitoring.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Annan klinisk medicin (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Other Clinical Medicine (hsv//eng)
Keyword
- Drug interactions
- Drug metabolism
- Eliglustat
- Enzyme replacement/augmentation therapy
- Substrate reduction therapy
- Type 1 Gaucher disease
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- By the author/editor
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Belmatoug, Nadia
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Di Rocco, Maja
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Fraga, Cristina
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Giraldo, Pilar
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Hughes, Derralyn ...
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Lukina, Elena
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show more...
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Maison-Blanche, ...
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Merkel, Martin
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Niederau, Claus
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Plöckinger, Ursu ...
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Richter, Johan
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Stulnig, Thomas ...
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vom Dahl, Stepha ...
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Cox, Timothy M.
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Other Clinical M ...
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European Journal ...
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Lund University