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Biomedical subjects

P Garver

Publications and source records attributed to P Garver.

13 recordsLinked to original sources
PubMed1995

The clinical course of treated and untreated Gaucher disease. A study of 45 patients.

One hundred nineteen patients with Gaucher disease were examined in the past 13 years. Of these 45 were examined 3 or more times over a time-span exceeding one year and all such patients are included in this study. Adult patients showed little progression of disease. There were few alterations in the blood counts, no increase in size of liver and spleen, and changes in skeletal lesions were largely confined to pre-existing lesions. Some children appeared to have more progressive disease, but since many of the children in this study were treated with alglucerase, it is difficult to draw conclusions about the natural progression of the disease at earlier ages. Treatment with alglucerase resulted in gradual normalization of blood counts, decrease in the size of liver and spleen, and parallel decreases in the serum angiotensin converting enzyme and chitotriosidase levels. Skeletal symptoms were decreased in all patients, and skeletal lesions showed modest improvement in patients treated for two years or more. The response of patients to low dose/high frequency (2.3 U/Kg 3 x weekly; 30 U/Kg/Mo) therapy was indistinguishable from the response observed and previously reported by others with much larger doses. Changing the dosage from 30 U/Kg/Mo to 120 U/Kg/Mo was not attended by any significant changes in response. Criteria for the selection of patients for treatment with alglucerase are proposed. We suggest that a starting dose of 15 to 30 U/Kg/month, fractionated 3 times weekly be used for all patients, regardless of severity or site of involvement, and that upward dosage adjustments be made only in such rare patients who may not respond adequately to this dose in 6 to 12 months.

Adolescent↗
PubMed1994

Comparison of magnetic resonance imaging and ultrasound in evaluating liver size in Gaucher patients.

Comparison is made between the reliability of two imaging methods (ultrasound and magnetic resonance imaging) to measure changes in the size of the liver in patients undergoing treatment for Gaucher disease. The reliability of either modality in estimating liver size is comparable, as is the reliability of demonstrating changes in the size of the liver with time. The precision of the two measurements is roughly the same.

Adolescent↗
PubMed1992

A less costly regimen of alglucerase to treat Gaucher's disease.

BACKGROUND: Alglucerase (Ceredase) provides effective enzyme-replacement treatment for patients with Gaucher's disease, but at the usually recommended dose of 60 U per kilogram of body weight every two weeks (130 U per kilogram per month), it costs $382,200 per year for a 70-kg patient. Theoretical considerations suggest that more frequent administration would be more efficient. METHODS: Fourteen patients with type 1 Gaucher's disease that was moderately severe to severe were given 30 U of alglucerase per kilogram per month, in divided doses given either daily or three times weekly, or 120 U given three times weekly. The effect of the treatment on the size of the liver and spleen and on blood counts was compared with published data on patients who received a total dose four to five times as large as the lower dose we used and who received treatment every two weeks. RESULTS: The response to 30 U of alglucerase per kilogram per month, fractionated into three or seven doses weekly, was approximately the same as that reported after the administration every two weeks of a dose four or five times as large, given in the large infusions usually recommended. A fourfold increase in the dose given three times weekly, from 2.3 to 9.2 U per kilogram, did not substantially increase the rate of improvement. CONCLUSIONS: The treatment of Gaucher's disease with smaller total doses of alglucerase given more frequently yields satisfactory results. A dose of 2.3 U per kilogram three times weekly yields major financial benefits with no sacrifice of therapeutic effect. Even taking into account the increased ancillary costs of more frequent administration, this method of administering alglucerase reduces the annual cost of the drug for a 70-kg patient to about $100,000.

Administration, Oral↗
PubMed1992

Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients.

We have reviewed our experiences with the clinical, laboratory, radiologic, and genetic features of 53 patients with Gaucher disease. Most were evaluated during early adult life, with a mean age of 33 years. Our patients were evaluated in a referral center, and therefore the data need to be interpreted with caution when applied to the general patient population, which includes a greater proportion of very mild cases. Thirty-nine patients were Ashkenazi Jews, 13 were non-Jewish and 1 was half-Jewish. The most common presenting symptom was bleeding related to splenomegaly and thrombocytopenia. The chronic symptoms, evaluated an average of 20 years after the diagnosis had been established, were mainly skeletal. Splenectomy had been performed in 43% of our patients and there was no evidence that this procedure accelerated the progression of liver and bone involvement. DNA from the patients was examined for 20 different mutations. The association between the 1226G/1226G genotype and a milder clinical course, and between the 1226G/84GG and 1226/1448C genotypes with more severe clinical manifestations, was confirmed. Repeated follow-up examinations in 29 patients revealed that in the majority of the patients, progression of the disease occurs during childhood, adolescence, or early adulthood with a marked tendency for stabilization thereafter. This observation suggests that Gaucher disease in most of the patients is not a relentless progressive disorder but a rather stable disorder during adulthood. The indications for the newly introduced intravenous enzyme replacement therapy as well as of future experimental treatments should be examined in the light of the natural history of the disease.

Adolescent↗
PubMed1991

Enzyme replacement therapy for Gaucher disease.

Four patients with moderately severe type I Gaucher disease were treated with commercially available mannose terminated glucocerebrosidase (Ceredase; Genzyme, Boston, MA) for up to 13 months. The enzyme was administered at the rate of three to four times weekly at one fourth the total recommended dosage, greatly decreasing the cost. Marked regression of hepatomegaly and improvement in liver function tests, peripheral blood counts, and serum angiotensin-converting enzyme levels were documented. The two patients with pulmonary involvement manifested improvement in pulmonary function tests. Skeletal disease remained unchanged.

Adult↗
PubMed1987

Grandmother functions in multigenerational families: an exploratory study of black adolescent mothers and their infants.

This qualitative study explores the functions of 19 Black grandmothers who are engaged in the care of their adolescent daughters' infants. Interviews with grandmothers revealed seven functions related to this role: managing, caretaking, coaching, assessing, nurturing, assigning, patrolling. Suggestions for future research with Black grandmothers are given.

Adolescent↗
PubMed1983

The effect of abstinence on left ventricular performance in asymptomatic chronic alcoholics.

Twelve asymptomatic men who were chronic alcoholics (42.3 +/- 10.7 years, mean age +/- 1 SD) underwent supine bicycle exercise and gated cardiac blood pool imaging 4-7 days after alcohol withdrawal and then again 32-65 days after abstinence (42.2 +/- 15.0 days). Workloads and exercise stages were identical during both exercise studies. Rest and exercise heart rates, blood pressures, cardiac outputs, double products, and systemic vascular resistances were similar in both studies. Ejection fraction (EF) was higher after abstinence at peak exercise (0.68 +/- 0.07 vs. 0.61 +/- 0.08 P less than 0.05); end-systolic volume (ESV) was smaller at rest and at peak exercise after abstinence (P less than 0.05). During the first exercise study, 6 of 12 (50%) subjects did not increase their EF by 0.05 units and 4 of 12 (33%) had no EF increase after abstinence. Even the original "normal" responders had greater rest and exercise EFs after abstinence. In the first exercise study end diastolic volume (EDV) rose during exercise (P less than 0.05) while ESV did not change. After abstinence, EDV did not change during exercise, while ESV declined (P less than 0.05). These results show that latent cardiac dysfunction exists in asymptomatic chronic alcoholics, which is partially although not completely resolved by abstinence of brief periods.

Adult↗
PubMed1982

Melorheostosis of the axial skeleton with associated fibrolipomatous lesions.

Two patients with melorheostotic-like lesions of the axial skeleton are described. In each case adjacent soft tissue masses containing both fatty and fibrous tissues were evident. The presence of such soft tissue tumors as well as other soft tissue abnormalities in melorheostosis emphasizes that the disease should not be regarded as one confined to bone. The precise pathogenesis of the osseous and soft tissue abnormalities in melorheostosis remains obscure.

Adult↗