A single ALS center experience with clinical use of sodium phenylbutyrate‐taurursodiol

• Published in: Muscle & nerve Vol. 70; no. 1; pp. 148 - 151
• Main Authors: Quinn, Colin, Baer, Michael, Amado, Defne A., Kelley, Mary, Elman, Lauren
• Format: Journal Article
• Language: English
• Published: Hoboken, USA John Wiley & Sons, Inc 01-07-2024; Wiley Subscription Services, Inc
• Subjects: Adult; Aged; ALS; Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - drug therapy; Clinical trials; Demographics; Demography; Drug Combinations; FDA approval; Female; Humans; Insurance; Male; Middle Aged; neuromuscular therapeutics; Patients; Phenylbutyrates - therapeutic use; Phenylbutyric acid; Retrospective Studies; Side effects; Sodium; Taste; ALS; neuromuscular therapeutics; clinical trials
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/mus.28096

Introduction/Aims
The aim of this study was to examine clinical utilization and discontinuation rates of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) in a single Amyotrophic Lateral Sclerosis (ALS) center. PB‐TURSO was approved by the United States Food and Drug Administration (FDA) in September 2022. Prior experience has been limited to clinical trials or expanded access protocols. In this manuscript, we discuss insurance approval rates, patient uptake, and discontinuation of PB‐TURSO in a large academic center.
Methods
Records of patients seen for clinical visits between January 2022 and May 2023 were reviewed. Demographic and clinical characteristics of our clinic population and those initiating PB‐TURSO were obtained from our clinical database.
Results
A total of 228 patients were seen during the observation period and 122 requested PB‐TURSO prescriptions. 77% (94) were approved by insurance. 66% (65) of those who were approved or received free drug chose to start medication. 51% (34) of those who initiated PB‐TURSO continued to take it through the end of the observation period. Four patients discontinued due to death during the observation period. Of the 29 patients who survived and discontinued, the main reasons for discontinuation were GI symptoms (17, 58.6%) and taste (8, 29.6%).
Discussion
PB‐TURSO was approved by insurance for most patients. The discontinuation rate was high and was driven largely by GI side effects and taste. Future considerations would include deeper examination of demographic trends, patient costs, side effects, and potential benefits in clinical practice.