The ALS Natural History Study protocol was developed with the goal of sharing longitudinal natural history data from several ALS multidisciplinary clinics participating in the ALS Natural History Consortium.

 Edaravone was approved by the FDA in May 2018 as a new treatment for ALS. There is not yet much data available regarding edaravone use in the United States. We report on edaravone use in our clinics since its approval.

To report percentage and general information on patients receiving edaravone in the clinics members of ALS Natural History Study Consortium.

All patients followed regularly in seven multidisciplinary ALS clinics are being offered participation in the study. Consenting participants are assigned a Neurological Global Unique Identifier (NeuroGUID), and a predefined clinical dataset is captured in NeuroBANK™.  All medications, including edaravone, are recorded. 

The dataset will be queried for edaravone use, duration of use, and selected clinical and demographic information.

As July of 2018, 105 of 419 consented PALS had received edaravone. Ten PALS were not included due to incomplete data. Of the remaining 95, 61 are male, 34 are female, and age range is from 37 to 82 years. Sixteen PALS (17%) have stopped edaravone, on average after 2.6 months of onset of treatment (range: <1 month through 6.6 months after onset of treatment). Average ALSFRS-R score of all patients on edaravone was 33.6, and of the patients that discontinued the medication was 29.8.

Enrollment has accelerated since that time and updated results, vital capacity slopes, and data for the aggregate population through March 2019 will be reported at the meeting.

The ALS Natural History Study Consortium provides an opportunity to participating sites to aggregate heterogeneous patient population, and to provide usage and efficacy information on concomitant medications, including post-marketing review of approved drugs as edaravone.