Preferences of Patients with Amyotrophic Lateral Sclerosis for Intrathecal Drug Delivery: Choosing between an Implanted Drug-Delivery Device and Therapeutic Lumbar Puncture

Jaein Seo, Sandeep Saurkar, Gabriela S. Fernandez, Anup Das, Stephen A. Goutman and Sebastian Heidenreich ()
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Jaein Seo: Patient-Centered Research, Evidera
Sandeep Saurkar: Biogen
Gabriela S. Fernandez: Patient-Centered Research, Evidera
Anup Das: Patient-Centered Research, Evidera Ltd
Stephen A. Goutman: University of Michigan
Sebastian Heidenreich: Patient-Centered Research, Evidera Ltd

The Patient: Patient-Centered Outcomes Research, 2024, vol. 17, issue 2, No 5, 177 pages

Abstract: Abstract Background Novel intrathecal treatments for amyotrophic lateral sclerosis (ALS) may require delivery using lumbar puncture (LP). Implanted drug-delivery devices (IDDDs) could be an alternative but little is known about patients’ preferences for intrathecal drug-delivery methods. Objective We aimed to elicit preferences of patients with ALS for routine LP and IDDD use. Methods A discrete choice experiment (DCE) and a threshold technique (TT) exercise were conducted online among patients with ALS in the US and Europe. In the DCE, patients made trade-offs between administration attributes. Attributes were identified from qualitative interviews. The TT elicited maximum acceptable risks (MARs) of complications from device implantation surgery. DCE data were analyzed using mixed logit to quantify relative attribute importance (RAI) as the maximum contribution of each attribute to a preference, and to estimate MARs of device failure. TT data were analyzed using interval regression. Four scenarios of LP and IDDD were compared. Results Participants (N = 295) had a mean age of 57.7 years; most (74.2%) were diagnosed

Date: 2024
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DOI: 10.1007/s40271-023-00665-4

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