Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0023473 (chronic myeloid leukemia)
 18,916 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

A 32-year-old woman presented with chronic myeloid leukemia 20 years after 2 198Au synoviortheses to her knees for severe juvenile rheumatoid arthritis. She progressed to blast phase of her disease and died from complications of therapy. The literature on rheumatoid arthritis, its therapy, radioisotopes and leukemia is reviewed.
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PMID:Chronic myeloid leukemia in a woman with Still's disease treated with 198Au synoviorthesis. 186 20

We present the clinical data and the management of twelve patients with symptomatic pericardial effusion (PE). The etiology of PE was: Chronic renal failure, viral infection, cardiac surgery, juvenile rheumatoid arthritis and chronic myelocytic leukemia. Four cases were diagnosed as idiophatic. PE in childhood is usually asymptomatic. When symptoms are present they are non-specific and don't help to know the size of the effusion; therefore, it's necessary to practice an echocardiography to demostrate the presence of PE. The hemodynamic findings permit to diferentiate patients with and without cardiac tamponade. The treatment of first choice is aspirin. In patients with cardiac tamponade the treatment should be pericardiocentesis.
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PMID:[Symptomatic pericardial effusions in childhood]. 269 67

Gaucher disease is the most common lysosomal storage disorder. Incidence of disease is around 1:40-60,000 inhabitants and it is assuming that in Croatia we have 20-30 patients with Morbus Gaucher. Morbus Gaucher is recognized in three types. Type I or so called non-neuropatic from is most common in Europe while so called neuropatic forms (type II and III) are much rare (5-10%). Type II and III are characterized by central nervous system involvement and usually are diagnosed in childhood. Disease manifestations are observed in various human organs. The most common disease features are involved liver, spleen, bones, lungs and brain. Enzyme replacement therapy with imiglucerase (Cerezyme) is now day's therapeutic gold standard. Imiglucerase prevents progressive manifestation of disease and patients have normal life. Cost of the treatment is high due to the cost of the recombinant technology which was used to product imiglucerase. The cost of the enzyme replacement therapy is 150-200,000 EUR per year for imiglucerase in a typical adult patient. Because of high cost for the patient treatment Ministry of Health of Republic of Croatia, and Croatian Health Insurance Company in year 2002. established a special program for so called "Expensive drug treatment". This program covering treatment costs for patients with inherited metabolic disorders, adenosine deaminase deficiency, chronic myeloid leukemia, AIDS, multiple sclerosis, juvenile arthritis and ovarian cancer. Until now 11 adult patients and 2 children with Gaucher disease were diagnosed in Croatia and all are on enzyme replacement therapy with imiglucerase. According to our experience administration of imiglucerase decreased spleen and liver size and number of bone pain crisis as well as normalization of platelet and red blood cells. Administration of the imiglucerase do no revert bone changes e.g. avascular hip necrosis or vertebra collapses, but prevent further bone deterioration. According to this, treatment with imiglucerase should be started immediately after establishing diagnosis to prevent irreversible changes on human organs.
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PMID:[Diagnosis and treatment of Gaucher disease in Croatia]. 1895 62