Response to sapropterin hydrochloride (Kuvan®) in children with phenylketonuria (PKU): a clinical trial

AbstractBackgroundPhenylketonuria (PKU) is one of the most common types of inborn error of metabolism. The mainstay of therapy for PKU has been dietary phenylalanine (Phe) restriction. Sapropterin dihydrochloride has been shown to be effective in reducing Phe levels in PKU patients.MethodsThis study was a clinical trial performed in the pediatric endocrine clinic of Imam Reza Hospital, Mashhad, Iran.ResultsAll children between 1 and 10 years of age with a diagnosis of PKU whose serum Phe levels were between 120 and 360 μmol/L, in Khorasan Razavi province in the north-east of Iran, were enrolled. Twenty-four patients were enrolled in the study.Intervention: A free diet for 72 h was allowed and then a 20-mg/kg/day dose of Kuvan®was administered. More than 30% reduction in blood Phe levels was described as responsive. Eight patients responded to the loading test and were eligible for the second stage of the study. In this stage, Phe powder in combination with Kuvan was provided. Patients’ serum Phe was measured weekly for 3 months. All eight patients showed Phe tolerance in 3 months, and their serum Phe levels remained within the range.ConclusionsTreatment with Kuvan can help reduce blood Phe levels in our pediatric PKU population and allows patients to follow a more liberal diet.

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Improving MUSLE performance for sediment yield prediction at micro-watershed level using seasonal classified data

Water Practice & Technology ◽

10.2166/wpt.2018.061 ◽

2018 ◽

Vol 13 (3) ◽

pp. 505-512

Author(s):

Hamzeh Noor ◽

Mohammad Rostami Khalaj

Keyword(s):

Sediment Yield ◽

Traditional Approach ◽

Estimation Error ◽

Superior Performance ◽

North East ◽

Erosion Processes ◽

The North ◽

East Of Iran ◽

Different Seasons

Abstract Separating erosion data and assessing season-based models are of great importance considering the variation in soil erosion processes in different seasons, especially in semi-arid regions. However, evaluation of an erosion model using seasonal classification of data and at a micro-watershed level have rarely been considered. Therefore, the present study was conducted to evaluate the modified universal soil loss equation (MUSLE): 1) with the seasonal classification of data and 2) with the traditional approach (no classification of data), in the Sanganeh research micro-watershed. This watershed has an area of 1.2 ha and is located in the north east of Iran. The results showed that the original MUSLE overestimated the sediment yield in the study watershed. Also, after calibration of MUSLE, the seasonal classification of data (with a relative estimation error (RE) of 34%) showed its superior performance compared with the traditional calibration approach (with a RE of 62%). In this regard, the obtained REs of 33, 40, and 31% respectively for spring, autumn, and winter are within or close to the acceptable range.

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