Objective: To investigate how patient reported outcomes (PROs) perform to document the effects of a botulinum neurotoxin (BONT) treatment of movement disorders in the clinical routine

Background: BONT therapy is the established standard of care for a variety of movement disorders and effectiveness has been documented in numerous controlled trials. However, there is no consensus and a large heterogeneity how treatment (effects) are assessed and documented in the clinical routine.

Method: We prospectively assessed established PROs using tablet PCs at each BONT-treatment appointment at the outpatient clinic of the department of Neurology at the University Clinic of Düsseldorf Germany. EQ5D5L scores for quality of life, global clinical impression (GCI) from the physician’s perspective and subjective treatment response from the patient’s perspective were recorded for all patients. Disease specific PROs were obtained as appropriate: MIDAS for migraine, PRISM for spasticity, CDQ24 for cervical dystonia, blepharospasm disability scale for blepharospasm, and HFS-7 for facial hemispasm.

Results: Longitudinal data of patients being initiated on BONT therapy were obtained from 239 patients and in addition from 903 patients on stable BONT treatment between 2018 and 2022. Patients with spasticity had the lowest EQ5D5L scores and highest CGI severity scores while the other indication groups did not differ. The CDQ24, MIDAS and HFS-7 scales showed the strongest changes after initiation of a BONT therapy, while PRISM and the blepharospasm disability scale did not change.  The BONT dose was continuously increased during the first 3 treatment cycles for spasticity, cervical dystonia and migraine while the dose was kept stable on the group level in facial hemispasm and blepharospasm.

Conclusion: It is possible to utilize patient reported outcomes to record the impact of neurological BONT therapy in clinical settings. However, certain scales such as CDQ24 and MIDAS appear to be more responsive to treatment effects, whereas others, like the blepharospasm disability scale, may not be as sensitive in detecting the effects of BONT injections.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/putting-the-patients-voice-at-the-venter-tracking-the-impact-of-botulinum-neurotoxin-therapy-for-movement-disorders-using-patient-reported-outcomes/