PracticeUpdate Conference Series_WORLDSymposium 2019

Questionnaire Caregiver Domain, Beck Depression Inventory), and pain (Adolescent Pediatric Pain Tool or Brief Pain Inventory). Dr. Adam concluded that in the managed access agreement, patients receiving elosulfase α for ≥2 years showed stable or improved patient-reported outcomes, in line with previously reported sta- bilization/improvement of clinical outcomes with elosulfase α. Dr. Adam’s team, while collecting these patient- reported outcomes, undertook their own research to capture changes reported by patients. Patients (or parents of patients under 18 years of age) were asked to describe, in their own words, any changes they noticed after 4, 8 and 12 months on treatment and then yearly thereafter (enzyme replacement therapy-naive patients) or yearly from entry to the managed access agreement (patients who received enzyme replacement therapy previ- ously in a clinical trial). Testimonies were collected for 42 patients age 1–58 years. At 4 months, the most commonly reported changes among treatment-naive patients were: energy levels (10/49, 20%), walking/movement (9/49, 18%) and pain (8/49, 16%). At 8 months, the most commonly reported changes among treatment-naive patients were: general health (22/79, 28%), walking/movement (17/79, 22%) and energy levels (13/79, 16%).

" The findings may act as a guide to the selection of suitable tools or development of disease-specific measures for use in future studies. "

At 12 months, the most commonly reported changes among treatment-naive patients were: general health (24/95, 25%), energy levels (19/95, 20%), and sleep/ tiredness (16/95, 17%). At 12 months, the most commonly reported changes among patients in clinical trials were: energy levels (11/53, 21%), general health (10/53, 19%), and specific health benefits andwalking/movement (both 7/53, 13%). At 24 months, the most commonly reported changes among patients in clinical trials were: energy levels (4/19, 21%), walking/movement, sleep/tiredness, think- ing/learning, and general health (all 3/19, 16%). Dr. Adam concluded that the study highlights a range of outcomes important to patients’ lives. These out- comes may not be collected via current standard patient-reported outcome tools. The findings may act as a guide to the selection of suitable tools or development of disease-specific measures for use in future studies.

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WORLDSymposium 2019 • PRACTICEUPDATE CONFERENCE SERIES 13