ANTONIO TOSCANO
Switching treatment to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease
Autori
- ANTONIO TOSCANO (ERN-NMD CENTER FOR NEUROMUSCULAR DISORDERS OF MESSINA, DEPARTMENT OF CLINICAL AND EXPERIMENTAL MEDICINE, UNIVERSITY OF MESSINA, MESSINA, ITALY – NEUROGENETIC DISEASES (NEUROMUSCULAR AND NEURODEGENERATIVE DISEASES))
- BARRY J. BYRNE (UNIVERSITY OF FLORIDA, GAINESVILLE, FL, USA – PEDIATRIC CARDIOLOGY)
- KRISTL G. CLAEYS (DEPARTMENT OF NEUROLOGY, UNIVERSITY HOSPITALS LEUVEN, AND LABORATORY FOR MUSCLE DISEASES AND NEUROPATHIES, DEPARTMENT OF NEUROSCIENCES, KU LEUVEN, LEUVEN BRAIN INSTITUTE (LBI), LEUVEN, BELGIUM – NEUROMUSCULAR DISORDERS)
- PAULA R. CLEMENS (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE AND VA PITTSBURGH HEALTHCARE SYSTEM, PITTSBURGH, PA, USA – GENETIC MYOPATHIES )
- JORDI DÍAZ-MANERA (JOHN WALTON MUSCULAR DYSTROPHY RESEARCH CENTRE, NEWCASTLE UNIVERSITY, NEWCASTLE UPON TYNE, UK; NEUROMUSCULAR DISORDERS UNIT, NEUROLOGY DEPARTMENT, HOSPITAL DE LA SANTA CREU I SANT PAU, BARCELONA, SPAIN; CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED DE ENFERMEDADES RARAS (CIBERER), MADRID, SPAIN – NEUROMUSCULAR DISEASES, TRANSLATIONAL MEDICINE AND GENETICS)
- MAZEN M. DIMACHKIE (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF KANSAS MEDICAL CENTER, KANSAS CITY, KS, USA – NEUROMUSCULAR DISORDERS)
- PRIYA S. KISHNANI (DUKE UNIVERSITY MEDICAL CENTER, DURHAM, NC, USA – CLINICAL AND BIOCHEMICAL GENETICS; LYSOSOMAL STORAGE DISORDERS)
- HANI KUSHLAF (DEPARTMENT OF NEUROLOGY & REHABILITATION MEDICINE, UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE, CINCINNATI, OH, USA – NEUROMUSCULAR DISORDERS)
- TAHSEEN MOZAFFAR (DEPARTMENT OF NEUROLOGY, UNIVERSITY OF CALIFORNIA, IRVINE, CA, USA – NEUROMUSCULAR DISORDERS)
- MARK ROBERTS (SALFORD ROYAL NHS FOUNDATION TRUST, SALFORD, UK – NEUROLOGY)
- NOEMI HUMMEL (CERTARA GMBH, LÖRRACH, GERMANY – STATISTICAL AND ECONOMIC MODELLING)
- MITCHELL GOLDMAN (AMICUS THERAPEUTICS, INC., PRINCETON, NJ, USA – TRANSLATIONAL MEDICINE, BIOMARKERS, AND PATIENT-REPORTED OUTCOMES)
- FRED HOLDBROOK (AMICUS THERAPEUTICS, INC., PRINCETON, NJ, USA – BIOSTATISTICS )
- SIMON SHOHET (AMICUS THERAPEUTICS LTD, MARLOW, UK – CELL BIOLOGY AND HEALTH ECONOMICS AND OUTCOMES RESEARCH)
- BENEDIKT SCHOSER (FRIEDRICH-BAUR-INSTITUTE AT THE DEPARTMENT OF NEUROLOGY, LMU UNIVERSITY HOSPITAL, LMU MUNICH, MUNICH, GERMANY – NEUROLOGY)
- ON BEHALF OF THE ATB200-07 STUDY GROUP
Presentatore
ANTONIO TOSCANO
Modalità
Oral Communication
Abstract
Late-onset Pompe disease (LOPD) substantially impacts motor function and health-related quality of life (HRQoL). The Phase III PROPEL trial (NCT03729362) assessed efficacy and safety of cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) in adults with LOPD. Here we evaluated the impact of switching to cipa+mig on motor function and HRQoL in the prespecified population of enzyme replacement therapy (ERT)-experienced patients in PROPEL. PROPEL included motor function assessments (6 minute walk test; Gait, Stairs, Gowers’ manoeuvre, and Chair) and patient-reported outcomes (PROs: EQ-5D-5L; Rasch-built Pompe-specific Activity; Subject Global Impression of Change [SGIC]; Patient-Reported Outcomes Measurement Information System [PROMIS]). Group-level analyses estimated between-group differences (least square means) for motor function and PRO change from baseline to week 52 using analysis of covariance adjusted for baseline age, gender, height, weight, and ERT status. Patient-level responder analyses of PROs compared the proportion of patients satisfying literature-based responder thresholds via chi-square/Fisher’s exact tests. Group-level analyses favoured cipa+mig versus alg+pbo in most motor function assessments and PROs, with nominal significance for walking tests and SGIC’s “ability to move around” and “energy level”. Patient-level responder analyses showed a greater proportion of patients improved with cipa+mig versus alg+pbo for most PROs. Differences in proportions of responders between cipa+mig versus alg+pbo were nominally significant for SGIC’s “overall wellbeing”, “ability to move around”, “muscle function”, and “energy level”. These analyses highlight the patient perspective and provide evidence that switching from alg+pbo to cipa+mig benefits patients’ motor function and HRQoL. Study supported by Amicus Therapeutics Inc.