Optimized Trientine-dihydrochloride Therapy in Pediatric Patients With Wilson Disease: Is Weight-based Dosing Justified?

OBJECTIVES: The aim of the study was to investigate the efficacy and safety of trientine-dihydrochloride (TD) in pediatric patients with Wilson disease (WD) and the effect of different weight-based dosages on their clinical and biochemical outcome. METHODS: We retrospectively reviewed the clinical data of 31 children with WD receiving TD therapy ages under 18 years at the time of diagnosis. Outcome measures included parameters of copper metabolism and liver function tests. To examine the impact of different weight-based dosages, 2 dosage subgroups were analyzed. Group 1 received less than 20 mg/kg TD per day, group 2 more than 20 mg ·â€Škg-1 ·â€Šday-1. RESULTS: Median follow-up was 60 (5-60) months in the total study group. During TD therapy, nonceruloplasmin-bound copper was reduced from mean 1.53 (0.01-6.95) at baseline to 0.62 (0.01-4.57) µmol/l. 24h-urinary copper excretion diminished to 1.85 (0.8-9.6) µmol/day approximating the therapeutic goal of 1.6 µmol/day. Seven of 31 patients (22.6%) required discontinuation of TD treatment, in 4 cases it was because of adverse events (ulcerative colitis, gingival and breast hypertrophy, hirsutism, elevation of transaminases).Investigations about weight-based dosage showed no significant difference of any laboratory parameter between the 2 cohorts. But in terms of clinical safety, adverse effects because of TD were only found in 6.7% of children in group 1 (<20 mg ·â€Škg-1 ·â€Šday-1, median follow-up 60 [9-60] months), whereas in group 2 (>20 mg ·â€Škg-1 ·â€Šday-1, median follow-up 60 [14-60] months), it was 63.6%. CONCLUSIONS: TD proves to be an efficacious alternative chelating agent for children with WD. Weight-based dosages above the recommended 20 mg ·â€Škg-1 ·â€Šday-1 may increase the rate of adverse effects in pediatric patients.

A surprising new species of Exapate Hübner, 1825 (Lepidoptera, Tortricidae) from Armenia.

A new species of cold-season moths, Exapate aidasi sp. nov., is described from male specimens collected in Armenia. This is also the first record of the genus Exapate in Armenia and south of the Caucasus. The new species differs from the two previously known congeneric taxa both in phenotypic characteristics and in the structure of the male genitalia. In addition, DNA barcodes (cytochrome c-oxidase subunit 1) are clearly divergent from those of other species. Adult and male genitalia of all three species of Exapate are shown for comparison.

Surprising discovery of an enigmatic geometrid in Croatia: Mirlatiaarcuata, gen. nov., sp. nov. (Lepidoptera, Geometridae).

A new monotypic genus of Geometridae, Mirlatiagen. nov., and a new species, M.arcuatasp. nov., are described from Croatia. Based on external and genitalia characters, the new genus is tentatively placed in the subfamily Larentiinae. However, the new genus takes a highly isolated position by having unique characters of the tympanum and showing an unusually long pectination of female antennae. Genetic analysis of a fragmented DNA barcode (mtDNA; cytochrome c oxidase 1) did not result in a clear assignation to any geometrid subfamily or tribe. Adults, male and female genitalia, and habitat photos of the type locality of the new species are illustrated.