Evaluating the patient needs and tolerability of Clobazam liquid formulation (Likozam® 1 mg/mL): A French patient and care-givers' centered survey.

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Tác giả: Caroline Delattre, Anne Emmanuel Leiber, Rima Nabbout, Giovanna Scorrano

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Epilepsy & behavior : E&B , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 89782

BACKGROUND: Clobazam (CLB) is a 1,5-benzodiazepine, currently used as add-on anti-seizure medication in complex and drug-resistant epilepsies. In France, CLB is available in three galenic forms and in a masterful one. The aim of this survey is to assess the experience of patients and families on CLB galenic forms focusing on CLB liquid formulation, Likozam® 1 mg/ml exclusively available in hospitals' pharmacies. METHODS: We performed a survey on the use of benzodiazepines with the Dravet syndrome Alliance (ASD) France. The survey was distributed on their site (http://www.dravet.fr/) and addressed to caregivers of patients who received CLB in any galenic form. The survey was co-developed by ASD and the Reference Centre for Rare Epilepsies, Necker. It included both open and closed questions. We used a 5-point Likert scale to assess the caregiver opinion on every formulation received, addressing medication palatability, dose adaptability, ability to take the drug without help, easiness of swallowing and access to treatment. Finally, we had some additional questions for the CLB liquid form. RESULTS: Eighty-seven patients aged 2 to 41 years (mean age 13, standard deviation ± 6.6) participated in this study. They presented Dravet syndrome (DS) (71/87, 82 %), Lennox-Gastaut syndrome (LGS) (4/87, 4 %), infantile epileptic spasms syndrome (IESS) (1/87, 1 %), and epilepsy with myoclonic atonic seizures (EMAtS) (1/87, 1 %). The epilepsy syndrome was unknown for 10/87 (12 %). The mean age at the first CLB prescription was 9 years (standard deviation ± 5.9, range age 1-35 years). Most patients (69/87, 79.3 %) had polytherapy. CLB most frequent formulations were the liquid (Likozam® 1 mg/mL) (44/87, 50.5 %) and the pill formulation (Urbanyl® 5 mg) (43/87, 49.4 %). The liquid formulation was selected as the most appropriate by 49,4% of caregivers, due to the greater easiness of administration and the dose adaptability. The easiness of access to treatment was the major issue against this formulation as it is currently available only in hospitals' pharmacies. CONCLUSION: Our study revealed an unmet need of CLB liquid form mainly in infants and young children, where easy to swallow and adaptable small doses are required during CLB introduction or discontinuation. However, some impactful issues such as access to treatment, palatability or liquid quantity administered should be optimized to improve patient and families' acceptability of liquid CLB.
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