136 research outputs found
Sanitary report as to Russian-Hebrew colonies in New Jersey
No full textThis report was a part of the 16th Annual Report of the Board of Health. The main purpose of it was to examine the level of sanitation at the Russian Jewish communities in New Jersey. It also gives a brief description of the industries of each colony and how the colony was founded
Review of the safety and efficacy of imiglucerase treatment of Gaucher disease
No full textDeborah Elstein, Ari ZimranGaucher Clinic, Shaare Zedek Medical Center, Jerusalem, IsraelAbstract: Most patients who suffer from symptomatic Gaucher disease will benefit from enzyme replacement therapy (ERT) with imiglucerase. The safety profile is excellent, only a small percentage of those exposed developing antibodies; similarly, very few patients require pre-medication for allergic reactions. Within 3 to 5 years of imiglucerase therapy, best documented at doses of 30 to 60 units/kg/infusion, hepatosplenomegaly can be expected to be reduced so that the liver volume will be maintained at 1 to 1.5 times normal (30% to 40% reduction from advent of ERT) and spleen volume to ≤2 to 8 times normal (50% to 60% reduction from advent of ERT). For anemic and thrombocytopenic patients, with 2 to 5 years of imiglucerase, hemoglobin levels are expected to be ≥11 g/dL for women and children and ≥12 g/dL for men; and platelet counts in patients with an intact spleen, depending on the baseline value, should approximately be doubled. Bone crises and bone pain but not irreversible skeletal damage will improve in most patients. Nonetheless, some features and some symptomatic patients apparently do not respond equally well and/or perhaps inadequately. The benefit for patients with the neuronopathic forms is primarily in improved visceral and hematological signs and symptoms. There are still several unresolved issues, the high per-unit cost being an important one, which have spurred the development of biosimilar enzymes as well as chaperone therapies currently in clinical trials.Keywords: Gaucher disease, enzyme replacement therapy, imiglucerase, substrate reduction therapy, pharmacological chaperones, cos
Plasma lipids are altered in Gaucher disease: Biochemical markers to evaluate therapeutic intervention
No full textEnzyme replacement therapy has been in clinical practice for the non-neuronopathic form of Gaucher disease for 15 years. However, the wide phenotypic variability in this disorder poses challenges to clinicians to assess patient severity and disease progression in order to effectively manage patients. Once therapy is initiated, methods to monitor the complex biochemical changes associated with the disease, and the response of these changes to therapy, are required in order to tailor therapy regimens to individual patients. We have evaluated the suitability of plasma sphingolipids and phospholipids as biochemical markers of disease burden and the efficacy of therapy to reduce that burden. Over 60 lipid species were measured using electrospray ionization-tandem mass spectrometry in plasma from controls and Gaucher patients, pre- and post-therapy. Glucosylceramide, molecular species of phosphatidylglycerol and G(M3) ganglioside were elevated in Gaucher disease, whereas species of ceramide, dihexosylceramide and sphingomyelin were decreased. Multivariate analysis enabled us to calculate the combined response of these lipids to therapy in Gaucher patients and correlate them with patient severity. Plasma lipids are proposed to be useful biomarkers for Gaucher disease.Peter J. Meikle, Philip D. Whitfield, Tina Rozaklis, David Blacklock, Stephen Duplock, Deborah Elstein, Ari Zimran, Eugen Mengel, Paul Cannell, John J. Hopwood and Maria Fullerhttp://www.elsevier.com/wps/find/journaldescription.cws_home/622796/description#descriptio
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