A Pilot Study of Fluorodeoxyglucose Positron Emission Tomography Findings in Patients with Phenylketonuria before and during Sapropterin Supplementation

BACKGROUND AND PURPOSE: PET scanning with fluorodeoxyglucose (FDG-PET) is a non-invasive method that measures regional glucose metabolic rate. Phenylalanine (Phe) and its metabolites appear to impair several aspects of brain energy metabolism. 1) To evaluate brain glucose metabolism with FDG-PET imaging in phenylketonuria (PKU) patients before and 4 months after sapropterin therapy; 2) to evaluate neurodevelopmental changes, blood Phe levels and dietary Phe tolerance before and after sapropterin therapy; 3) to generate pilot data to assess the feasibility of evaluating brain glucose metabolism with FDG-PET imaging and to explore potential trends resulting from the administration of sapropterin therapy. METHODS: We enrolled 5 subjects, ranged in age from 22 years to 51 years, with PKU. Subjects underwent FDG-PET brain imaging, blood tests for Phe and tyrosine levels, and neurocognitive evaluations before and 4 months after sapropterin therapy (20 mg/kg/day). All subjects' Phe and tyrosine levels were monitored once a week during the study. Subjects kept 3 day diet records that allow calculation of Phe intake. RESULTS: None of the subjects responded to sapropterin therapy based on 30% decrease in blood Phe level. The data show that glucose metabolism appeared depressed in the cerebellum and left parietal cortex while it was increased in the frontal and anterior cingulate cortices in all five subjects. In response to sapropterin therapy, relative glucose metabolism showed significant increases in left Broca's and right superior lateral temporal cortices. Interestingly, there was corresponding enhanced performance in a phonemic fluency test performed during pre- and postneurocognitive evaluation. CONCLUSIONS: Further studies with a larger sample size are needed to confirm the above changes in both sapropterin non-responsive and responsive PKU patients.

Feasibility of a peer-led, school-based asthma education programme for adolescents in Jordan

The Adolescent Asthma Action programme [Triple A] has been used successfully to promote asthma knowledge, awareness and quality of life in adolescents with asthma in Australia. We describe the feasibility and acceptability of an adaptation of this English-language, peer-led, asthma education programme in a girls' high school in Northern Jordan. The pilot was conducted by bilingual health workers. Feasibility, acceptability and adaptability were measured through participation rates, open-ended questionnaires completed by peer leaders, a focus group for junior students and reflective journal notes. The programme was well-received by staff and students, with high levels of participation. The peer-led approach was viewed positively. Students reported that they enjoyed the interactive learning activities and the opportunity to practise English. The students reported increased asthma knowledge and awareness, with students with asthma reporting receiving more support from peers. A peer-led asthma education programme is feasible and acceptable in the Jordanian school context