Blepharospasm (BPS) is one of the most frequent types of facial dystonia and, at the same time, one of the most disabling, being able to trigger functional blindness if not treated. Our aim with this work was to evaluate the efficacy and safety of long-term onabotulinum A toxin (BAT) treatment in a cohort of patients with BPS. The retrospective study was conducted on consecutive patients with BPS treated with subcutaneous BAT. The selection of muscles and dose was made based on each patient's needs. The clinical and demographic characteristics, number of sessions, dose, duration and effectiveness of treatment, and adverse events were analysed. 130 patients were included in the study. The median (95% confidence interval) length of follow-up was 14 (13-15.6) years with an average of 20.5 sessions (range from 10 to 57). Regarding the efficacy of the treatment, 114 (87.7%) experienced satisfactory results with functional and aesthetics recovery. Patient evaluation of global response suggested a clear improvement without adverse events in 72 (55.4%) patients. Adverse events developed at least once during the treatment in 39% of patients, with transient ptosis and haematoma the most common reported both by physician and patient. The results of our study suggest that botulin toxin A is a safe and effective long-term treatment for blepharospasm with mild, transient and well-tolerated side effects when they appear.
Background
Dopamine replacement therapy reduces most motor and nonmotor features of Parkinson's disease. However, with disease progression, adjustments of dopaminergics and the application of advanced therapies must be considered.
Objectives
To validate the OPTIMIPARK questionnaire as a tool to help clinicians make therapeutic decisions on patients treated with levodopa.
Methods
We tested a questionnaire including 9 items encompassing motor and nonmotor signs, complications, and disability in a multicenter, observational, cross‐sectional study. A neurologist (neurologist 1 [N1]) assessed patients according to regular clinical practice and blinded to the OPTIMIPARK questionnaire score. Therapeutic decisions were classified as “no changes,” “adjustment of conventional treatment,” and “advanced therapy indicated.” External neurologists (neurologist 3 [N3] and neurologist 4 [N4]), who only knew the patient age, years of disease, and current treatment, made their therapeutic decisions based on the OPTIMIPARK score. Concordance between the criterion of the N1 versus the OPTIMIPARK‐based N3–N4 consensus was analyzed applying weighted κ. The area under Receiving Operating Characteristic (ROC) curves was calculated for OPTIMIPARK scores.
Results
A total of 113 patients with Parkinson's disease were included. The OPTIMIPARK‐based decision led to a higher proportion of patients requiring therapeutic modification than N1 assessment (74% vs. 60%; P = 0.002). Concordance between the N1 and N3–N4 decisions was moderate, whereas interobserver agreement among N3 and N4 was high. Area Under the Curve(AUC) values of 0.83 and 0.82 were found for “no changes” and “advanced therapy indicated” decisions by the N1 neurologist.
Conclusions
OPTIMIPARK might be more sensitive than regular clinical practice in suggesting the need for a therapeutic change. Furthermore, the low and high scores identify with high accuracy well‐adjusted patients and candidates for advanced therapy, respectively.
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