Objective: To assess tolerability and efficacy of high dose Safinamide treatment in Young Onset Parkinson’s Disease (YOPD) patients with dystonia and history of impulse controls disorder (ICD).

Background: Safety of high dose (up to 300 mg daily) Safinamide therapy has previously been assessed in dose-finding studies on epilepsy. ICD is far more frequent in hereditary Parkinson’s Disease (PD) and YOPD than in so-called Idiopathic Parkinson’s Disease (IPD). In these patients, the advised use of Dopaminoagonist (DA) is very often burdened by the occurrence of severe ICD, including binge-eating, hypersexuality and compulsive shopping.

Method: In this study, 9 patients were enrolled, aged 18-40 (5 males). Mean duration of disease was 6.5 years. Six patients were definite carriers of PARK mutations (2, 6), whereas GCH mutation was excluded. Informed consent was obtained from all patients. All patients were receiving L-Dopa monotherapy with a mean daily intake of 300-400 mg. All of them had severe ICD, appeared during DA treatment, which was introduced to decrease L-Dopa daily intake. ICD was assessed by means of Questionnaire for Impulsive-Compulsive Disorders in Parkinson’s Disease – Rating Scale (QUIP-RS). Also, all of them had painful dystonia, unrelated to L-Dopa therapy. Movement Disorder Society-Unified Parkinson’s Disease Rating Scale 4.6 item (MDS-UPDRS 4.6) for Painful Dystonia in “off” state was administered to quantify this disturbance.

Results: Mean QUIP-RS score at baseline was 9.8. Mean MDS-UPDRS 4.6 score at baseline was 2.6. Patients were treated with Safinamide add-on therapy with the following titration: during first week they received 50 mg daily, in the second week 100 mg daily, then, since the third week, 150 mg daily. At three-month follow-up, mean MDS-UPDRS 4.6 score was decreased at 1.4, whereas mean QUIP-RS score showed no statistical significantly variations (Table 1). Symptoms as Visual Hallucination (VH) or acute psychosis were not observed after Safinamide add-on therapy.

Conclusion: Safinamide was proven to be an effective treatment option, even at high doses off-label up to 150 mg daily, in our cohort of YOPD patients. ICD symptoms, which had led to DA withdrawal, did not worsen with Safinamide, while painful dystonia in “off” states had remarkable improvement.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/high-dose-off-label-safinamide-treatment-in-young-onset-parkinsons-disease-patients/