2023 American Academy of Neurology Abstract Website

Michele Potashman¹, Isabella Huang¹, Susan Durham¹, Victoria Wirtz¹, Gilbert L'Italien¹, Vladimir Coric¹, Andrea De Palma², Triza Brion², Günter Höglinger³, Horacio Kaufmann⁴, Phillip Low⁵, Nikolaus McFarland⁶, Wassilios Meissner⁷, Patricio Millar Vernetti⁸, Huw Morris⁹, Werner Poewe¹⁰, Klaus Seppi¹⁰, Wolfgang Singer⁵, Steven Vernino¹¹, Irfan Qureshi¹
¹Biohaven Pharmaceuticals Inc, ²ICON, ³Hannover Medical School, ⁴NYU Langone Health - NYU Dysautonomia Center, ⁵Mayo Clinic, ⁶University of Florida, ⁷Bordeaux Neurocampus, ⁸NYU School of Medicine, ⁹Department of Clinical Neuroscience, UCL Queen Square Institute of Neurology, ¹⁰Department of Neurology, Medizinische Universität Innsbruck, ¹¹UT Southwestern Medical Center

Objective:

To capture the multiple system atrophy (MSA) patient experience from a series of patient concept elicitation interviews to evaluate a modified version of the Unified Multiple System Atrophy Rating Scale (UMSARS).

Background:

The UMSARS was designed to evaluate signs and symptoms of MSA that progress over a patient’s course of disease. A subset of 9-items from the UMSARS was proposed for use as a clinically meaningful and sensitive measure of disease progression in a clinical trial conducted over 1-year. Patient cognitive interviews were conducted to evaluate the clinical meaningfulness of the selected items (speech, cutting food and handling utensils, dressing, hygiene, walking, urinary function, arising from chair, posture, gait).

Design/Methods:

Semi-structured patient interviews were conducted among 31 patients, probing on symptoms, impact of same to activities of daily living (ADLs), level of concern for symptom worsening and treatment goals. Interviews were audio recorded, transcribed, and analyzed by MAXQDA software to generate the library of qualitative findings. The library was supplemented by key quotes documented by the interviewer.

Results:

The conceptual model for MSA signs and symptoms indicates patients are concerned with (listed by frequency of report): balance (76%), speech (66%), urinary functioning (66%), loss of fine motor control (52%), lightheadedness (45%), sleep (34%), constipation (34%), blood pressure issues (31%), coordination (28%), and fatigue (28%). Functional impacts include a range of ADLs, physical functioning, emotional functioning, social functioning, and work/employment impacts. Evaluation of the 9-items of the modified UMSARS with the interview findings indicate the items in this scale adequately reflects the interviewed patient experiences.

Conclusions:

Patient cognitive interviews demonstrate the complexity of the signs, symptoms and functional impacts of MSA. While all items contained in the modified UMSARS map to aspects of disease that are relevant to the patient experience, it does not comprehensively address all aspects of patient concern.