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Histiocytosis and Lipid Storage Diseases
Published in Harold R. Schumacher, William A. Rock, Sanford A. Stass, Handbook of Hematologic Pathology, 2019
Salwa Shabbir Sheikh, David F. Garvin
e. Nervous System. Gaucher cells are present in perivascular Virchow-Robin spaces in the brain parenchyma. There is widespread neuronal loss; dentate nucleus of cerebellum is almost always involved. Multiple myeloma, chronic lymphocytic leukemia, and amyloidosis are associated with Gaucher’s disease more commonly than in the normal population. Pregnancy is not contraindicated, but is considered to be high risk because of thrombocytopenia, and coagulation defects that might lead to bleeding. Pseudo-Gaucher cells are morphologically similar to Gaucher cells, but do not contain tubular structures characteristic of the Gaucher cells. These can be seen in various conditions, e.g., acute lymphoblastic leukemia, multiple myeloma, chronic granulocytic leukemia, plasmacytoid lymphoma, Hodgkin’s lymphoma, and AIDS with Mycobacterium avium infection. Patients with Gaucher’s disease receiving alglucerase treatment show a false-positive pregnancy test, as alglucerase is a placenta-derived drug and contains human chorionic gonadotrophin.
Non-viral liver disease
Published in Michael JG Farthing, Anne B Ballinger, Drug Therapy for Gastrointestinal and Liver Diseases, 2019
John ML Christie, Roger WG Chapman
Treatment of Gaucher’s disease is with enzyme replacement with alglucerase,89 which has been shown to decrease the size of the liver and spleen over relatively short time intervals. The disease can be cured by bone marrow transplantation.
Genetic Disorders, Dysplasias and Malformations
Published in Louis Solomon, David Warwick, Selvadurai Nayagam, Apley and Solomon's Concise System of Orthopaedics and Trauma, 2014
Louis Solomon, David Warwick, Selvadurai Nayagam
Bone pain may need symptomatic treatment. For the acute crisis, analgesic medication and bed rest followed by a period of non-weightbearing is recommended. Specific therapy is available (albeit costly) in the form of the replacement enzyme, alglucerase. This has been shown to reverse the blood changes and reduce the size of the liver and spleen. The bone complications also are diminished.
Pharmacological treatment of pediatric Gaucher disease
Published in Expert Review of Clinical Pharmacology, 2018
Punita Gupta, Gregory Pastores
Alglucerase was subsequently replaced by human recombinant imiglucerase (CerezymeTM, Sanofi/Genzyme), generated using Chinese hamster ovary (CHO) cells, in the mid-1990s; this preparation continues to be the most widely used ERT in clinics across the world. In 2009, a vesivirus 2117 infection of the bioreactors in which imiglucerase was manufactured led to a global shortage of imiglucerase and expedited the approval of two ‘new’ enzymes: gene-activated human recombinant velaglucerase alfa (VPRIVTM, Shire) derived from a human fibrosarcoma cell line and plant-cell-derived human recombinant taliglucerase alfa (ElelysoTM Protalix/Pfizer). All three recombinant enzyme formulations have exposed mannosyl-residues, although the process involved in achieving this is unique for each product. These three ERTs are not biosimilar products, and there appears to be no major difference in their safety profile [45–48].