Protein and calorie intakes in adult and pediatric subjects with urea cycle disorders participating in clinical trials of glycerol phenylbutyrate

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• 作者：Debra Hook^a ; George A. Diaz^b ; Brendan Lee^c ; James Bartley^a ; Nicola Longo^d ; William Berquist^e ; Cynthia Le Mons^f ; Ingrid Rudolph-Angelich^g ; Marty Porter^g ; Bruce F. Scharschmidt^g ; Masoud Mokhtarani^g ; ^{masoud.mokhtarani@horizonpharma.com" class="auth_mail" title="E-mail the corresponding author}
• 关键词：UCD ; urea cycle disorder ; GPB ; glycerol phenylbutyrate ; NaPBA ; sodium phenylbutyrate ; NHANESs ; National Health and Nutrition Examination Surveys ; RDA ; Recommended Daily Allowances ; FAO ; Food and Agriculture Organization ; WHO ; World Health Organization ; OTC ; ornithine transcarbamylase
• 刊名：Molecular Genetics and Metabolism Reports
• 出版年：2016
• 出版时间：March 2016
• 年：2016
• 卷：6
• 期：Complete
• 页码：34-40
• 全文大小：1001 K

文摘

Little prospectively collected data are available comparing the dietary intake of urea cycle disorder (UCD) patients to UCD treatment guidelines or to healthy individuals.

Objective

To examine the protein and calorie intakes of UCD subjects who participated in clinical trials of glycerol phenylbutyrate (GPB) and compare these data to published UCD dietary guidelines and nutritional surveys.

Design

Dietary data were recorded for 45 adult and 49 pediatric UCD subjects in metabolic control during participation in clinical trials of GPB. Protein and calorie intakes were compared to UCD treatment guidelines, average nutrient intakes of a healthy US population based on the National Health and Nutrition Examination Survey (NHANES) and Recommended Daily Allowances (RDA).

Results

In adults, mean protein intake was higher than UCD recommendations but lower than RDA and NHANES values, while calorie intake was lower than UCD recommendations, RDA and NHANES. In pediatric subjects, prescribed protein intake was higher than UCD guidelines, similar to RDA, and lower than NHANES data for all age groups, while calorie intake was at the lower end of the recommended UCD range and close to RDA and NHANES data. In pediatric subjects height, weight, and body mass index (BMI) Z-scores were within normal range (− 2 to 2).

Conclusions

Pediatric patients treated with phenylbutyrate derivatives exhibited normal height and weight. Protein and calorie intakes in adult and pediatric UCD subjects differed from UCD dietary guidelines, suggesting that these guidelines may need to be reconsidered.