PracticeUpdate Conference Series_WORLDSymposium 2019

Elosulfase α Improves Outcomes Long Term in Patients With MPS IVA The finding is in line with previously reported data.

P atients with mucopolysaccharidosis (MPS) IVA receiving elosulfase α for ≥2 years show stable or improved patient-reported outcomes, in line with previously reported stabilization/improvement of clinical outcomes with the drug. This outcome of a 24-month assessment using sev- eral measurement tools was reported at the meeting. Jacqueline Adam, PhD, BSc, of MPS Commercial in Amersham, UK, and colleagues evaluated and reported baseline and 24-month assessments (20-month assess- ments for the Beck Depression Inventory) of patients with MPS IVA who had a managed access agreement. As of 2018, 36 patients had received treatment in the managed access agreement for ≥2 years (26 initiated treatment in clinical trials, 10 initiated in the managed access agreement). Mean treatment duration was 7 ± 1.4 and 2.1 ± 0.3 years for each group, respectively (range in clinical trial patients 4.1–8.9 years, range in patients initiating treatment in the managed access agreement 0.2–2.2 years). Among patients in the clinical trial, the fve- item EuroQol 5D (n=22) and Mucopolysaccharidosis Health Assessment Questionnaire Caregiver Domain (n=24) remained unchanged from baseline through 24 months (EuroQol 5D 5L, mean change –0.04 ± 0.29, Mucopolysaccharidosis Health Assessment Questionnaire Caregiver Domain 0.38 ± 6.27). Patients initiating treatment in the managed access agreement who completed the EuroQol

5D 5L (n=8) and Mucopolysaccharidosis Health Assessment Questionnaire Caregiver Domain (n=10) also remained unchanged (EuroQol 5D 5L 0.08 ± 0.53, Mucopolysaccharidosis Health Assessment Questionnaire Caregiver Domain –1.3 ± 16.79). From baseline through 20 months, the Beck Depression Inventory remained unchanged for clinical trial patients (n=19, –0.47 ± 2.34). Those initi- ating treatment in the managed access agreement improved (n=4, –5.75 ± 6.85). Patients in the clinical trial (n=11) and those initiating treatment in the managed access agreement (n=9) assessed using the Adolescent Pediatric Pain Tool improved from baseline through 24 months (–3.27 ± 1.62 and –2.33 ± 2.83, respectively). Only 6 patients in the clinical trial were assessed. Brief Pain Inventory results remained unchanged (–0.17 ± 2.23). No patients discontinued treatment due to intolerability or lack of efficacy. Elosulfase α is the only approved treatment for MPS IVA. In England, access to treatment is granted on a conditional basis through a managed access agreement. One criterion to continue treatment is stabilization/ improvement in quality of life/activities of daily living as assessed by patient-reported outcomes. Patient-reported outcomes assessed quality of life/activities of daily living (EuroQol 5D 5L and/ or Mucopolysaccharidosis Health Assessment

PRACTICEUPDATE CONFERENCE SERIES • WORLDSymposium 2019 12