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At least 73 records · Page 4Linked to original sources
PubMed2004

Gaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapy.

Symptomatic patients with Gaucher disease (GD) (acid beta-glucosidase [Gcase] deficiency) are treated with injectable human recombinant GCase. Treatment results in significant decreases in lipid storage in liver, spleen, and bone marrow, but the generalized osteopenia and focal bone lesions present in many adult patients are refractory to treatment. A double-blind, 2-arm, placebo-controlled trial of alendronate (40 mg/d) was performed in adults with GD who had been treated with enzyme for at least 24 months. Primary therapeutic endpoints were improvements in (1) bone mineral density (BMD) and content (BMC) at the lumbar spine, and (2) focal lesions in x-rays of long bones assessed by a blinded reviewer. There were 34 patients with GD type 1 (age range, 18-50 years) receiving enzyme therapy who were randomized for this study. After 18 months, DeltaBMD at the lumbar spine was 0.068 +/- 0.21 and 0.015 +/- 0.034 for alendronate and placebo groups, respectively (P =.001). Long-bone x-rays showed no change in focal lesions or bone deformities in any subject in either arm. Alendronate is a useful adjunctive therapy in combination with enzyme replacement therapy (ERT) for the treatment of GD-related osteopenia in adults, but it cannot be expected to improve focal lesions.

Adult↗
PubMed1997

Gaucher disease: enzyme therapy in the acute neuronopathic variant.

The responses to regular intravenous enzyme infusions were compared in two sibs with Gaucher disease type 2, the acute neuronopathic variant. Enzyme administration was begun at 7 months in patient 1 who had severe progressive visceral and neuronopathic disease. No significant effect of enzyme infusions was noted. Death occurred at 9 months. Patient 2 was prenatally diagnosed and enzyme infusions were initiated at age 4 days. Overall development progressed at a rate similar to her unaffected full sib until her death at 15.1 months. Slowly progressive esotropia, ocular paresis and dysphagia began at 8 months as did infiltrative pulmonary disease. Comparison of these clinical courses show significant visceral and neurologic effects of anticipatory enzyme therapy, but with unaltered outcome, for Gaucher disease type 2.

Gaucher Disease↗
PubMed1997

[Enzyme therapy in children with severe forms of Gaucher's disease].

The enzyme therapy with Ceredase in patients with Gaucher's disease is at present probably the most expensive treatment in the whole world. One-year treatment of an adult patient with Gaucher's disease costs more than 7 million crowns. Indications for treatment in individual patients as well as financial provisions are so far problematic in the Czech Republic. From a total of 28 patients of varying age with Gaucher's disease diagnosed by the authors Ceredase was administered to two boys with a severe course of the disease. Within one year of treatment the health status of both children improved, growth became normal, the spleen diminished in size by 20-35%, haematological manifestations of hypersplenism are receding, there was a 32-46% decline of the activity of serum chitotriosidase and biochemical parameters of the disease improved.

Child↗
PubMedDate not supplied

Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1.

BACKGROUND: Enzyme replacement therapy (ERT) for Gaucher's disease with alglucerase or imiglucerase is efficacious, well-tolerated and safe. However, cost considerations, visits to medical facilities, potentially duration of theray for life, are issues of major concern to a proportion of treated patients and has, in some cases, led to the withdrawal of therapy. AIMS: To elucidate whether an extension of the interval between enzyme infusions to once every three weeks is as effective in maintaining the clinical responses achieved with the bi-monthly regimen. MATERIALS AND METHODS: Four patients with an optimal response to ERT (at 30 units/kg every two weeks for an average of 27 months), were subjected to enzyme dose/frequency changes that essentially constituted a reduction in cumulative dose over the treatment period. Patients were assessed every 6 months for alterations in haematological parameters, plasma chitotriosidase levels, liver and spleen size, and bone symptoms. RESULTS: All patients had to resume the previous infusion schedule of once every two weeks; one because of new bone marrow infiltrates, two because of visceral enlargement, and the fourth due to progressive anaemia. CONCLUSIONS: This limited experience suggests that a reduction in enzyme dose associated with an extended interval between infusions may lead to variable disease control, and underscores the need for individualization of enzyme therapy.

Adolescent↗
PubMed1997

[Systemic enzyme therapy in orthopedics and traumatology].

In 57 patients the preparations of systemic enzyme therapy vobenzim and flogenzim were applied during conservative or operative treatment of locomotor system injuries. Anti-edema, antiinflammatory and analgetic action of preparations was established.

Humans↗
PubMed1980

Enzyme therapy and immuno-adsorption by an extra-corporeal device.

An extra-corporeal chamber has been constructed and used for diagnosis, enzyme therapy and immuno-adsorption. The chamber is made from polymethyl-acrylate and the dimensions of the chamber are 10 x 18 x 7 cm. It contains 23 plates with a total area of 0.5 m2. In most cases no pump is used. The active substance is bound covalently to the plate surfaces. The time for each hemoperfusion has been 3-6 hr. No infections, decrease of thrombocytes or other side effects have been observed. By immobilized homologous antisera (F (ab')2), three putative tumour-associated antigens were isolated from cases of hypernephroma. Two children and one adult patient with malignant lymphoma were treated with extra-corporeal L-asparaginase, the latter with repeated remissions of metastases. The level of antibodies against Factor VIII was significantly decreased by chamber immunoadsorption in two cases of hemophilia. A number of kidney transplantation cases received extra-corporeal L-asparaginase pre- and post-operatively to counteract rejection. In all around 50 patients have received extra-corporeal treatment without any adverse effects. The use of the extra-corporeal chamber in other diseases is discussed.

Adenocarcinoma↗
PubMed1996

[Systemic enzyme therapy].

The paper summarizes the hitherto knowledge concerning the absorption of perorally administered enzymes in the digestive tract. After the transport through the intestinal wall the enzymes are protected against immunity recognition and they reach the affected regions via blood and lymphatic routes. A survey of enzymes taken into consideration and the essence of their action are presented. In the final part, the fields are summed up in which systemic enzymotherapy proves useful.

Enzyme Therapy↗
PubMed1990

[The sociomedical efficacy of the enzyme therapy of gonorrhea patients].

The study has involved 3702 male patients with gonorrhea. Of these 62 percent were fresh cases, 38 percent chronic ones; there were 218 patients (5.9 percent) with recurrences, of these 63.3 percent with new relapses and 36.7 percent with chronic ones. 85.3 percent of patients developed monorelapses. The recurrences were equally frequent after antibiotic therapy (50.2 percent) and after combined treatment (49.8 percent). Proteolytic enzymes (chymotrypsin, trapsin 5 mg twice daily with a 12 hrs interval) in combination with an antibiotic were administered to 242 patients with gonorrhea complicated by epididymo-orchitis and with gonorrhea relapses. Etiologic cure was achieved in all of them; postgonorrheal conditions were recorded in 6-7 percent and depended on the antibiotic administered; in reference patients recurrences were recorded in 4.2-6.5 percent of cases, postgonorrheal conditions in 14-21.4 percent of cases. Enzymic therapy helped reduce the length of inpatient therapy of a patient by 5.93 days on an average in cases with orchidoepididymitis and by 14.64 days in gonorrhea relapses, with the economic effect per worker being 131.4 and 307.2 rubles, respectively.

Anti-Bacterial Agents↗