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.
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
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.
Patients followed in the department of hereditary metabolic diseases at Necker Hospital, having specific oral drug treatment, and having a scheduled care appointment at Necker Hospital as part of their usual follow-up, during the study inclusion period.
Case-only
Prospective
Questionnaire
Other interventionA 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.
Calculation of the metabolic balance score
Other interventionFor 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.
Assessment of therapeutic adherence
Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire.
Number of treatments taken each day
Quantification of the number of medications taken daily based on the declarations of patients and their parents to the study questionnaire.
Knowledge of the disease
Assessment of patients' and their parents' knowledge of the disease establish from the declarations of patients and their parents to the study questionnaire.
Knowledge of treatments
Accordance of patients' and their parents' knowledge of treatments, based on the declarations of patients and their parents in the study questionnaire and the patient's prescriptions in the medical record.
Correlation between the therapeutic adherence score and the number of medications taken daily by patients
The therapeutic adherence score and the number of medications taken daily are established from the declarations of patients and their parents to the study questionnaire.
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Assistance Publique - Hôpitaux de Paris
Lead sponsor
URC-CIC Paris Descartes Necker Cochin
Collaborator
