Effect on the Quality of Life of a Therapeutic Education Program in Patients With Marfan Syndrome (MYLIFE)

Effect on the Quality of Life of a Therapeutic Education Program in Patients With Marfan Syndrome: an Observational, Prospective and Multicenter Study

This is a before-after, observational, prospective, multicenter cohort study. The study will consist of 2 phases: an initial observational phase of a minimum of 3 months before the Therapeutic Education Program (TEP) intervention, then a phase of evolution analysis of at least 3 months.

Study Overview

• Status: Active, not recruiting
• Conditions: Marfan Syndrome
• Intervention / Treatment: Other: Therapeutic Education Program

Detailed Description

During the first observational phase, the quality of life assessment will be carried out 3 months apart without intervention. The data obtained during this first phase will make it possible to characterize the functional impact in patients, to verify the reproducibility of our evaluations, and to evaluate the spontaneous evolution of the parameters over a period of 3 months.

During the second phase of evolution analysis, the quality of life will be assessed at 3 month intervals (M9, M12) after the therapeutic Education Program (M6). The comparison between the evolution during the two phases, before and after the TEP will allow us to assess the benefits of the TEP.

• Study Type: Observational
• Enrollment (Anticipated): 60

Contacts and Locations

Study Locations

• France
  • Toulouse, France, 31000
    • UHToulouse

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patient with Marphan Syndrom

Description

Inclusion Criteria:

• Patient aged between 7 and 25 years old with Marfan Syndrome or related according to Ghent criteria
• For minors, no oral opposition from the holders of parental authority and consent of the minor.
• For adults, no oral opposition collected
• Patient affiliated to a social security scheme or equivalent

Exclusion Criteria:

• Inability of the patient to understand the content of the TEP sessions or the questionnaire about quality of life (non-French speaking, severe intellectual disability)
• Patient who has already participated in a TEP session for his pathology.
• Protected adult: patient under legal guardianship or curator protection

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
therapeutic Education Program; individual and group sessions will be set up with educational workshops specialized by age group (child / adolescent / transition / adult / entourage / parents), socio-administrative workshops, etc. The objectives will be adapted to each age group and to each patient individually.	Other: Therapeutic Education Program; needs assessment; educational group workshop; specialized workshops by age group: child / adolescent / transition / adult / entourage / separated parents and in parallel; socio-administrative workshop; individual summary (accompanied or not by his entourage / family); satisfaction questionnaire

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
quality of life of children and young adults with Marfan	Answer to the question of PedsQL	Base line
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),	Answer to the question of PedsQL	3 months
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),	Answer to the question of PedsQL	9 months
quality of life of children and young adults with Marfan syndrome after therapeutic education program (ETP),	Answer to the question of PedsQL	12 months

Collaborators and Investigators

• Sponsor: University Hospital, Toulouse
• Investigators: Principal Investigator: Yves DULAC, MD, University Hospital, Toulouse

Study record dates

Study Major Dates

• Study Start (Actual): December 2, 2020
• Primary Completion (Anticipated): January 31, 2024
• Study Completion (Anticipated): January 31, 2024

Study Registration Dates

• First Submitted: December 3, 2020
• First Submitted That Met QC Criteria: January 28, 2021
• First Posted (Actual): February 1, 2021

Study Record Updates

• Last Update Posted (Actual): February 1, 2021
• Last Update Submitted That Met QC Criteria: January 28, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Connective Tissue Diseases; Bone Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Musculoskeletal Abnormalities; Abnormalities, Multiple; Bone Diseases, Developmental; Limb Deformities, Congenital; Syndrome; Marfan Syndrome; Arachnodactyly
• Other Study ID Numbers: RC31/19/0505

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No