The real practice of clinical and economic research of drugs included in the Federal Program of High-Cost Nosologies

Background. Real world data help to provide more information on the effects and tolerability of therapy. However, data on the use of perampanel in Russian population of children remain limited.Aim. To conduct a retrospective analysis of perampanel efficacy and tolerability in children and adolescents with epilepsy in real clinical practice.Materials and methods. A total of 106 children aged 4–18 years receiving perampanel as part of combination therapy for epilepsy were observed at 18 centers. Seizure frequency at 1–3, after 6 and 12 months of treatment, presence and type of adverse events (AEs) were analyzed. The main assessed efficacy parameters were seizure control, significant (≥50 %) decrease in seizure frequency. All other outcomes (<50 % decrease, no change or increased seizure frequency) were considered to be no effect. The assessed safety parameters were the percentage of patients with or without AEs and the percentage of perampanel withdrawals due to AEs.Results. We defined 3 age groups for comparison: children 4–6, 7–11 and 12–17 years old. Perampanel efficacy was 69 % (seizure control – 23.6 %, ≥50 % decrease in seizure frequency – 45.3 %). The mean duration of the effect was 7.3 ± 4.1 months. No significant difference in efficacy between age groups was found. However, some greater efficacy of perampanel was noted in adolescents: absence of effect in the form of remission or significant decrease in seizures frequency was noted only in 25.5 %, with 40 % in children 4–11 years old. Among those with inefficacy, 3.8 % reported seizure aggravation. AEs occurred in only 23 % of patients, with the least frequent occurrence in adolescents (11.8 %) and the most frequent in children aged 7–11 years (40 %). The most frequent AEs was sluggishness and/or drowsiness. Discontinuation of perampanel due to AEs was required in 7.6 %.Conclusion. Perampanel has demonstrated high efficacy and good tolerability in real clinical practice among children from 4 years of age and adolescents with partial (focal) and secondary generalized seizures. The AEs that developed were not serious and very rarely led to withdrawal of the therapy. The results are comparable to those of phase III studies and previous real-world data. The usage of perampanel in children with primary generalized seizures should be further investigated.

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The first combined Russian experience of using perampanel in children and adolescents with epilepsy in everyday clinical practice

Rakhmanina

Volkov

Volkova

et al. 2022

Rus. ž. det. nevrol.

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Medical rehabilitation of patients with menopausal syndrome and surgical menopause: contribution of magnesium deficiency correction

et al. 2023

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Background. The increase in the proportion of patients with menopausal syndrome (MS) and surgical menopause, including women in the recovery phase after radical surgical treatment of the reproductive system cancer, is a characteristic trend of the 21st century. Part of them receive menopausal hormone therapy (MHT). This determines the relevance of the analysis of the specified cohort of patients to enhance rehabilitation programs designed to improve the quality of life (QoL) in this category of gynecological patients.Objective: subanalysis of primary data from the MAGYN study cohort of MHT treated women with MS and surgical menopause.Material and methods. The MAGYN observational non-interventional study of the real clinical practice included 9168 women, of whom 1528 represented a group of previously not characterized MHT treated women with MS and surgical menopause. To determine the number of patients with magnesium deficiency (MD), a Magnesium Deficiency Questionnaire (MDQ) was used, a biochemical blood test with an indicator of serum magnesium concentration was evaluated. The profile of the participants was analyzed by the presence of general somatic pathologies, obstetric, gynecological history, complaints, symptoms of MD on a visual-analog scale. The QoL was assessed with the help of The World Health Organization Quality of Life Brief Version (WHOQOL-BREF) before the start of a 4-week course of MD supplementation and after treatment.Results. According to the MDQ results, the prevalence of DM was 79.4%, which exceeded the results of all other groups in the study. Women with MD had a higher incidence of viral infections (19.2% vs. 22.7%; p=0.028), vegetative-vascular dystonia (26.2% vs. 29.7%; p=0.0466) and osteochondrosis (42.9% vs. 46.8%; p=0.0453). A subgroup of participants with verified MD demonstrated significantly more pronounced symptoms, such as irritability (4.9±3.5 vs. 4.6±3.4 points in the general group; p=0.0437), sleep disorders (4.4±2.3 vs. 4.2±2.3 points; p=0.0491), back pain (3.9±2.0 vs. 3.7±2.0; p=0.0405), and increased fatigue (4.6±2.3 vs. 4.4±2.3 points; p=0.0444). After the end of the course of therapy with fixed dose combination of magnesium citrate and pyridoxine, the sum of MDQ scores decreased from 46.0±12.7 to 29.2±15.1 points (p<0.001) in combination with an increase in the plasma concentration of magnesium to 0.79±0.23 mmol/l; women's satisfaction with their physical, psychological, social well-being increased (from 21.1±4.5 to 26.2±3.5 points; from 24.8±4.9 to 28.1±4.4 points, and from 9.3±2.7 to 11.0±2.8 points, respectively; p<0.001); self-perception increased from 18.2±3.7 to 22.2±3.6 points. Thus, there was a significant decrease in the severity of MD and a significant improvement in QoL according to WHOQOL-BREF, which is important in the rehabilitation of such patients.Conclusion. The profile of patients with MS and surgical menopause receiving MHT, including the period of recovery after radical surgical treatment of reproductive cancer, is characterized by MD and decreased QoL. It seems effective to introduce MD correction into the complex rehabilitation program of such patients. It is necessary to conduct further studies to improve the rehabilitation measures in this cohort of patients.

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Perampanel in adjunctive therapy of patients with brain tumor-related epilepsy: real-world data

Samsonova¹,

Жидкова

2023

Èpilepsiâ paroksizmalʹnye sostoâ.

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Background. Brain tumor-related epilepsy (BTRE) is an important and insufficiently studied interdisciplinary problem. In a significant part of brain tumor patients, the disease onsets with epileptic seizures. The course of tumor-associated epilepsy is often pharmacoresistant and requires rational polytherapy. To date, there are no uniform recommendations on the choice of an antiepileptic drug (AED) for the initial therapy of BTRE.Objective: retrospective analysis of the efficacy/tolerability of adjunctive therapy with perampanel in relation to epileptic seizures in patients with epilepsy associated with glial brain tumors and metastases.Material and methods. The analysis included 51 patients with glial tumors and brain metastases who were prescribed perampanel as part of adjunctive therapy. Its effectiveness against focal seizures (FS) and bilateral tonic-clonic seizures (BTCS) was evaluated at follow-up periods of >1≥3≥6 months. A decrease in the frequency of seizures by 50% or more (responders) or by 100% (seizure freedom) was analyzed. An analysis of the influence of intervening factors in a multifactorial model, an assessment of the effectiveness of perampanel as a whole and a stratified assessment of intervening factors were carried out. The frequency and profile of adverse events (AEs) were also evaluated, including their possible association with the use of other AED.Results. In the multifactorial model, independent predictors of the clinical effect were the onset of the disease in the form of FS at >1≥3≥6 months follow-up. None of AEDs used in the first line of therapy demonstrated an impact on the clinical effect. There were no predictors of clinical effect in patients with BTCS during the entire follow-up period. Out of 51 patients, 48 (94.1%) were responders, and in 36 of them (70.6%) the seizure freedom was registered. Among patients with FS, the proportion of responders was 83.3–90.9% at different follow-up periods, including 31.2–50.0% who showed seizure freedom. Among patients with BTCS, 86.7–92.3% became responders, including 56.1–88.5% who achieved seizure freedom. AEs were noted in 7 (13.7%) patients, the most common was aggression – 4 patients (7.8%). There were no cases required reduction the dose or discontinuation the treatment with perampanel due to AEs. In most patients at >1≥3≥6 months follow-up, the median was 6 mg/day.Conclusion. The study performed in real-world practice confirmed the high efficiency and safety of perampanel in the adjunctive therapy of epileptic seizures associated with glial brain tumors and metastases, which together with the low potential of drug interactions allow us recommend the drug to this contingent of patients.

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The real practice of clinical and economic research of drugs included in the Federal Program of High-Cost Nosologies

Cited by 7 publications

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The first combined Russian experience of using perampanel in children and adolescents with epilepsy in everyday clinical practice

The first combined Russian experience of using perampanel in children and adolescents with epilepsy in everyday clinical practice

Medical rehabilitation of patients with menopausal syndrome and surgical menopause: contribution of magnesium deficiency correction

Perampanel in adjunctive therapy of patients with brain tumor-related epilepsy: real-world data

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