Presentation
Abstract We present the case of a woman with Gaucher disease who was being given alglucerase as enzyme replacement therapy. She was found to be pregnant: the treatment was continued. [haematologica.org]
Results The results of treatment are presented in Table 1. All the patients had a decrease in the size of the liver and spleen (if one was present) except Patient 14, who had no hepatomegaly at the start of treatment. [nejm.org]
Entire Body System
- Precocious Puberty
One case of precocious puberty has been reported to date, however due to the recent introduction of manufacturing steps designed to reduce the level of hCG in Ceredase (alglucerase injection) ®, the likelihood of this occurrence is reduced. [rxlist.com]
One case of precocious puberty has been reported to date, however due to the recent introduction of manufacturing steps designed to reduce the level of hCG in Ceredase ®, the likelihood of this occurrence is reduced. [drugs.com]
- Intravenous Administration
Therapeutic response to intravenous administration of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci USA 1990;87:1913–6. Google Scholar Barton NW, Brady RO, Dambrosia JM, di Bisceglie AM, Doppelt SH, Hill SC, et al. [link.springer.com]
Nonetheless, after intravenous administration of alglucerase, improvements are evident within 6 months of therapy. Patients have increased haemoglobin levels and platelet counts, and decreased incidences of epistaxis and bruising. [ncbi.nlm.nih.gov]
Workup
Serum
- Erythrocytosis
Patient 13 had impaired pulmonary function due to his Gaucher's disease and was undergoing frequent therapeutic phlebotomies to control his erythrocytosis. [nejm.org]
Treatment
Results The results of treatment are presented in Table 1. All the patients had a decrease in the size of the liver and spleen (if one was present) except Patient 14, who had no hepatomegaly at the start of treatment. [nejm.org]
Prior to the development of enzyme replacement therapy, treatment for Gaucher's disease was mainly symptomatic relief. Primary treatment with glucocerebrosidase focuses on removal of the lipid metabolite that causes the pathology. [pubmed.ncbi.nlm.nih.gov]
(verify) Alglucerase was a biopharmaceutical drug for the treatment of Gaucher's disease. [en.wikipedia.org]
Enzyme replacement (glucocerebrosidase) Comment Enzyme replacement therapy product Treatment of Gaucher disease Target GBA* [HSA_VAR:2629v1] [HSA:2629] [KO:K01201] Brite Anatomical Therapeutic Chemical (ATC) classification [BR:br08303] A ALIMENTARY [genome.jp]
Home treatment with intravenous enzyme replacement therapy for Gaucher disease: an international collaborative study of 33 patients. Blood 1993;82(4):1107–9. [link.springer.com]
Prognosis
Another form of pulmonary involvement in GD is pulmonary hypertension (PHT): a severe complication associated with poor prognosis. [kundoc.com]
Assessment of sequence generation low: if allocation sequence is suitable to prevent selection bias (i.e. computer‐generated lists, coin tossing, shuffling cards or envelopes, random number table, etc); high: if allocation sequence could be related to prognosis [cochranelibrary.com]
Epidemiology
As an alternative, analyses of observational data from a large international disease registry using standard epidemiologic matching and modeling methods may offer a feasible and methodologically sound approach. [nature.com]
Pathophysiology
Four possible pathophysiological mechanisms might account for the development of PTH in the individual with GD: 1. perivascular infiltration by Gaucher cells with vascular obliteration; 2. plugging of the capillaries with Gaucher cells; 3. occlusion of [kundoc.com]
The end points selected are traditional, but insufficient, surrogates for the full spectrum of GD pathophysiology. [cochranelibrary.com]
Prevention
Alendronate slows bone loss while increasing bone mass, which may prevent bone fractures. Alendronate is used to treat or prevent postmenopausal osteoporosis and steroid-induced osteoporosis. [emedicinehealth.com]
Uptake by both of these types of cell is prevented by mannan. The results suggest that the cellular mechanisms responsible for improvement of Gaucher patients receiving alglucerase treatment is probably more complicated than previously recognized. [ncbi.nlm.nih.gov]
[…] alimentary tract and metabolism products 1.1 A16AA Amino acids and derivatives 1.2 A16AB Enzymes 1.3 A16AX Various alimentary tract and metabolism products 2 QA16Q Other alimentary tract and metabolism products for veterinary use 2.1 QA16QA Drugs for prevention [en.wikipedia.org]
[…] the hydrolysis of the glycolipid, glucocerebroside, to glucose and ceramide as part of the normal degradation pathway for membrane lipids; enzyme replacement for conditions with functional deficiency in beta-glucocerebrosidase enzymatic activity to prevent [reference.medscape.com]
Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. [drugs.com]