OBJECTIVES: The study objective was to characterize the population of fixed-dose combination sodium phenylbutyrate and taurursodiol (PB&TURSO) users in the Amyotrophic Lateral Sclerosis (ALS) Research Collaborative Data Commons (ARC) database.

METHODS: The ALS ARC is a database of deidentified natural history data of persons living with ALS (PLWALS). Data are entered by PLWALS and/or their caregivers and includes demographics, treatment history, functional, and activity assessments. Duration of PB&TURSO use, and timing and use of concomitant ALS treatments were summarized using descriptive statistics. Categorical variables were summarized using counts and percentages and continuous variables were summarized using mean (SD). For assessment of functional status, conducted using ALS Functional Rating Scale (ALSFRS-R), only participants meeting pre-specified criteria of minimum 3 assessments were included.

RESULTS: Of n=738 identified PLWALS, 67 were identified as PB&TURSO users who met the study inclusion criteria. Of these, 51% were female, 94% were white and 16% had family history of ALS. The mean age of symptom onset was 54 years (SD 11). Median time from symptom onset to PB&TURSO initiation was 36 months. Riluzole and edaravone were used at some point in patient journey in 91% and 63% of participants, respectively. Median ALSFRS-R change (slope) was -0.22 prior to PB&TURSO initiation (range -1.49 to -0.01). Initiation of riluzole or edaravone prior to PB&TURSO occurred in 81% and 93% of combination users, respectively.

CONCLUSIONS: PLWALS participating in the ARC database and using PB&TURSO represent a slower progressing population than studied in previous PB&TURSO clinical trials. This will provide a robust source for ongoing real-world study of PB&TURSO effectiveness and overall ALS treatment patterns. Concomitant use of PB&TURSO with other ALS medications was common among PLWALS.