Patient Reported Outcomes and Measures of Disease Severity in Lysosomal Disease

Wednesday, February 11, 2015 from 11:30 AM – 12:45 PM

This program provided a discussion of challenges to data collection and ways to overcome these challenges to produce valid and reliable research data. There were short presentations in the context of a panel discussion, along with time for audience Q&A.

Learning Objectives

At the end of this program, the participant will be able to:

  1. Identify key features of current measurement tools: including QOL, pain, behavioral, adaptive and ADL measures
  2. Utilize these measurement tools can help define disease progression and measure treatment outcomes.
  3. Discuss how patient reported outcomes correlate with objective disease state.

Agenda

11:30 AM
Registration and Check-in

11:40-11:45 AM
Introduction to patient and parent reported outcomes
Elsa G. Shapiro, PhD, ABPP, LP

11:45 AM-12:00 PM
Use of patient/parent reported outcomes in natural history studies and clinical trials
Laurie Muldowney, MD

12:00-12:10 PM
Evidence-Based QOL (Quality of Life) Measurements
Julie B. Eisengart, PhD, LP

12:10-12:20 PM
Measurement of Adaptive Function
Kathleen A. Delaney, CCRC, CSP
​
12:20-12:​25 PM
Measures of Behavior — Example from MPS IIIA​
Elsa G. Shapiro, PhD, ABPP, LP

12:​​25-12:35 PM
Disease-specific developed measure (MPS HAQ) vs. general validated tool (EQ-5D-5L) in MPS IVA
Christian J. Hendriksz, MBChB, MSc, FRCPCH

12:35-12:50 PM
Panel Discussion and Audience Q&A
Faculty

Invited Faculty

Kathleen A. Delaney, CCRC, CSP
Community Program Specialist,
Division of Clinical Behavioral Neuroscience,
Department of Pediatrics,
University of Minnesota
Minneapolis, Minnesota, USA

Julie B. Eisengart, PhD, LP
Assistant Professor of Pediatrics,
Pediatric Neuropsychology,
Division of Clinical Behavioral Neuroscience,
University of Minnesota
Minneapolis, Minnesota, USA

Christian J. Hendriksz, MBChB, MSc, FRCPCH
Clinical Lead, Consultant Transitional Metabolic Disorders,
Salford Royal Foundation NHS Trust, Mark Holland Metabolic Unit
Salford, United Kingdom
Honorary Senior Lecturer,
Faculty of Medical and Human Sciences – Imaging, Genomics and Proteomics,
Manchester Academic Health Science Centre,
The University of Manchester
Manchester, United Kingdom

Laurie Muldowney, MD
Medical Officer,
Division of Gastroenterology and Inborn Error Products,
CDER/FDA
Silver Spring, Maryland, USA

Elsa G. Shapiro, PhD, ABPP, LP
Professor of Pediatrics and Neurology,
Pediatric Neuropsychology, Division of Pediatric Clinical Neuroscience,
Department of Pediatrics,
University of Minnesota
Minneapolis, Minnesota, USA