A subpopulation analysis of study TKT032 was conducted to assess the efficacy and safety of velaglucerase alfa among children aged 2-17. Of 25 patients in the trial, 7 pediatric patients (28%) were enrolled and randomized to receive velaglucerase alfa at 60 U/kg (n=4) or 45 U/kg (n=3) as a 1-hour infusion, every other week for 12 months. In this analysis the two dose groups were pooled and results are shown as a percent change from baseline.
Following 12 months treatment with velaglucerase alfa the mean hemoglobin concentration increased 20%, mean platelet counts increased 54%, mean spleen volume, normalized by body weight, decreased 47% (median value 5.0 multiples of normal (MN) decreased from 13.5 MN). Mean liver volume normalized by body weight decreased 13% (median value 1.04 MN decreased from 1.40 MN). Outcomes reported in children were consistent with those seen in the overall population. Due to the small sample sizes, the results did not achieve statistical significance.
Most frequently reported adverse events in the pediatric subpopulation were headache, nasopharyngitis, pyrexia, nasal congestion, productive cough, vomiting, and injury. No pediatric participants experienced severe or serious treatment-emergent events. No pediatric patients developed antibodies to velaglucerase alfa.
"We are very pleased with the opportunity to present the findings from the first of three Phase III studies for velaglucerase alfa," said Whaijen Soo, MD, PhD, Senior Vice President, Research and Development, Shire Hum
|SOURCE Shire plc|
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