PS-036 Factors Affecting Compliance With Enzyme Replacement Therapy With Idursulfase In Children With Hunter Syndrome: Data From The Hunter Outcome Survey

Abstract

Background and aims Manifestations of Hunter syndrome typically become apparent between 2 and 4 years of age; affected children may be treated with enzyme replacement therapy with idursulfase (Shire). This long-term treatment consists of weekly infusions generally administered over 3 h. Patients may sometimes miss scheduled infusions. This analysis investigated the frequency of, and reasons for, missed idursulfase infusions and stopping treatment in children. Methods This analysis used data from the Hunter Outcome Survey (HOS), a global, observational registry sponsored by Shire that collects real-world clinical information on the natural history of Hunter syndrome and the long-term effectiveness and safety of idursulfase. Results As of January 2014, data on missed infusions and stopping treatment between HOS entry and last clinical evaluation recorded in HOS/treatment end (median, 35.4 months) were available for 483 children followed prospectively in HOS aged Conclusions Analysis of HOS data reveals that a variety of factors affect treatment compliance; the most common reason for missing an infusion was illness. However, 72.0% of children receiving idursulfase did not miss a single infusion during this analysis period, and few children stopped treatment.