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

Assistance Publique - Hôpitaux de Paris

Status: Not yet enrolling

Conditions: Metabolic Diseases

Treatments: Other: Questionnaire; Other: Calculation of the metabolic balance score

Study type: Observational

Funder types: Other

Identifiers

NCT07356557

APHP251413

Details and patient eligibility

About

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.

Full 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.

Enrollment: 200 estimated patients

Sex: All

Ages: 7 to 20 years old

Volunteers: No Healthy Volunteers

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.

Trial design

200 participants in 1 patient group

Patients

Description:

Patients, children aged at least 7 years and adolescents/young adults followed in the pediatric department of hereditary metabolic diseases of Necker Hospital, having specific oral drug treatment.

Treatment:

Other: Calculation of the metabolic balance score

Other: Questionnaire