Evaluation of Therapeutic Adherence Among Patients Followed in the Department of Hereditary Metabolic Diseases at Necker Hospital

Обзор

The purpose of this study is to evaluate treatment adherence among patients followed in the Department of Inherited Metabolic Diseases at Necker Hospital, in order to assess the need for implementing a therapeutic education workshop focused on medication adherence.

Подробное описание

There is limited data in the literature regarding treatment adherence in children with metabolic disorders. A 2011 study on phenylketonuria highlighted a decline in dietary adherence during adolescence. Another study, dating from 1996 and focusing on urea cycle disorders, emphasized the importance of good treatment adherence for maintaining disease control. Furthermore, larger-scale research in epileptic patients has identified several barriers to treatment compliance, highlighting the crucial role of treatment adherence in improving disease control.

The purpose of this study is to evaluate treatment adherence among patients followed in the Department of Inherited Metabolic Diseases at Necker Hospital, in order to assess the need for implementing a therapeutic education workshop focused on medication adherence.

Вмешательства

• Другое Questionnaire
  A pseudonymized questionnaire assessing treatment adherence, understanding of the illness, knowledge of medications, and the number of oral medications taken is given to patients and/or their parents during a follow-up visit, whether in a consultation, day hospital, or conventional hospitalization. It is completed by parents and children aged 7 and older, as well as by adolescents and young adults. The responses will not be disclosed to the physicians in the department.
• Другое Calculation of the metabolic balance score
  For patients with urea cycle deficiency, a metabolic balance score will be calculated independently from retrospective data from the medical record, in the last two years preceding the completion of the questionnaire.

Первичные конечные точки

• Assessment of therapeutic adherence [Срок оценки: Time 0]

Вторичные конечные точки (9)

• Number of treatments taken each day [Срок оценки: Time 0]
• Knowledge of the disease [Срок оценки: Time 0]
• Knowledge of treatments [Срок оценки: Time 0]
• Correlation between the therapeutic adherence score and the number of medications taken daily by patients [Срок оценки: Time 0]
• Correlation between the therapeutic adherence score and the age of patients [Срок оценки: Time 0]
• Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease [Срок оценки: Time 0]
• Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments [Срок оценки: Time 0]
• Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency [Срок оценки: Time 0]
• Assessment of medication adherence in the event of a travel [Срок оценки: Time 0]

Критерии участия

Критерии включения

• All patients followed in the Hereditary Metabolic Diseases department of Necker Hospital during their visit to the department for their usual care and having a specific daily oral medication treatment.
• Children aged at least 7 years and adolescents/young adults
• Holders of parental authority and children or adolescents or adults' patients informed and consenting to participate in the study

Критерии исключения

• Metabolic disease without oral medication (intravenous treatments, amino acid mixtures, and dietary regimens are not evaluated).
• Patient and parents not proficient in French.
• Refusal by the patient's holders of parental authority or adult patient to participate in the study and/or refusal of the child/adolescent.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Только случаи

Центры проведения

Франция · 1 центр

• Hôpital Necker-Enfants Malades — Paris

Публикации

• Modi AC, et al. "Adolescent adherence to antiepileptic drug therapy: determinants and clinical outcomes." JAMA, 2011.
• Maestri NE, et al. "Outcome of urea cycle disorder patients: importance of metabolic control and compliance." Journal of Pediatrics, 1996.
• Ten Hoedt AE, et al. "Adherence to treatment in phenylketonuria: challenges and improvements." Molecular Genetics and Metabolism, 2011.

Идентификаторы

NCT: NCT07356557 · APHP251413

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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