Metabolic Diseases
Conditions
Keywords
Metabolic disease, Daily oral drug treatment, Medication adherence, Therapeutic education workshop
Brief summary
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.
Detailed description
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.
Interventions
OTHERQuestionnaire
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.
OTHERCalculation 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.
Sponsors
Assistance Publique - Hôpitaux de Paris
URC-CIC Paris Descartes Necker Cochin
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
7 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of therapeutic adherence | Time 0 | Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of treatments taken each day | Time 0 | 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 | Time 0 | 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 | Time 0 | 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 | Time 0 | 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. |
| Correlation between the therapeutic adherence score and the age of patients | Time 0 | Therapeutic adherence score, established from the declarations of patients and their parents to the study questionnaire. |
| Correlation between the therapeutic adherence score and patients' and their parents' knowledge of the disease | Time 0 | The therapeutic adherence score and the knowledge of the disease are established from the declarations of patients and their parents to the study questionnaire. |
| Correlation between the therapeutic adherence score and patients' and their parents' knowledge of treatments | Time 0 | The therapeutic adherence score and the knowledge of treatments are established from the declarations of patients and their parents to the study questionnaire and on the patient's prescription. |
| Correlation between therapeutic adherence score and metabolic balance for patients cared for urea cycle deficiency | Time 0 | 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 based on the following clinical data: * Number of ammonia levels \> 100 µmol/L, * Mean and median follow-up ammonia levels, * Number of glutamine levels \> 1000 µmol/L, * Number of hospitalizations for decompensation. |
| Assessment of medication adherence in the event of a travel | Time 0 | Assessment of patients' and their parents' medication adherence in the event of a travel, established from the declarations of patients and their parents to the study questionnaire. |
Countries
France
Contacts
CONTACTMargaux GASCHIGNARD, M.D.
CONTACTHélène Morel
PRINCIPAL_INVESTIGATORMargaux MD GASCHIGNARD, M.D.
Assistance Publique - Hôpitaux de Paris
STUDY_DIRECTORPascale de LONLAY, M.D., PhD
Assistance Publique - Hôpitaux de Paris
Outcome results
None listed