Quality of Life in Patients With Auto-inflammatory Diseases (MAI-PATIENT)

Creation of a Tool to Assess Quality of Life in Patient With Auto-inflammatory Diseases

This qualitative interview-based study aim to build a tool to assess quality of life in patients (adults or children) suffering from 6 autoinflammatory diseases (FMF, TRAPS, CAPS, MKD, Still and PFAPA).

Study Overview

• Status: Unknown
• Conditions: Hereditary Autoinflammatory Diseases
• Intervention / Treatment: Other: qualitative study

Detailed Description

Recent understanding of the physiopathological mechanisms that underpin autoinflammatory diseases (AIDs) allowed a revolution in the therapeutic management of these patients, particularly through the use of biotherapies. However new treatments need to be evaluated by valid tools. AIDAI score created and validated by our team evaluates activity of 4 autoinflammatory diseases. One of the important components, not explored by the scores currently available, is the impact of these treatments on quality of life (QoL) of patients. The existing quality of life scales are not adapted to these recurrent chronic pathologies. Throught a qualitative study based on interviews of patients and/ or parents, the investigators aim to build a tool to evaluate QoL in patients suffering from 6 aAIDs :familial Mediterranean fever (FMF), mevalonate kinase deficiency (MKD), tumour necrosis factor receptor-associated periodic syndrome (TRAPS), cryopyrin-associated periodic syndromes (CAPS), Still's disease and PFAPA syndrome.

• Study Type: Interventional
• Enrollment (Anticipated): 48
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Le Chesnay, France, 78150
    • Ch Andre Mignot
  • Le Kremlin Bicêtre, France, 94270
    • Dr Maryam PIRAM
  • Paris, France, 75020
    • Tenon

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Any patient (child and adult) with one of the 6 AIDs (FMF, TRAPS, MKD or CAPS, PFAPA or Still's disease) regardless of the activity of the disease. The diagnosis will have been made by the referring physician according to criteria specific to each AIDs
• Volunteer to participate in the study
• With at least one parent present at the consultation if it is a child under 18 years old
• For minors, agreement of the parents or their legal representative or one of the present parents
• Information for children as far as their age and condition allow
• Affiliation to a national health insurance.

Exclusion Criteria:

• patient or relative (according to age)refuse to participate.
• Bad understanding of French.
• Other chronic inflammatory pathology associated (example: Crohn's, SPA, uveitis, psoriasis ...)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: qualitative study; heterogeneous group of patients	Other: qualitative study; Interviews

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Qualitative evaluation of quality of life by interviews of patients and/or parents	The interviews will be recorded in digital audio and then transcribed literally. The analysis will be phenomenological. Four age group will be interviewed : < 6 year-old (parents), 6 to 12 year-old (patients and parents) , 13 to 17 year-old (patients and parents) , > 18 year-old (patients)	45 to 90 min

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start (Actual): June 6, 2018
• Primary Completion (Anticipated): December 1, 2020
• Study Completion (Anticipated): December 1, 2020

Study Registration Dates

• First Submitted: June 15, 2018
• First Submitted That Met QC Criteria: June 25, 2018
• First Posted (Actual): June 26, 2018

Study Record Updates

• Last Update Posted (Actual): July 2, 2020
• Last Update Submitted That Met QC Criteria: July 1, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Genetic Diseases, Inborn; Skin Diseases, Genetic; Familial Mediterranean Fever; Hereditary Autoinflammatory Diseases
• Other Study ID Numbers: K160903J; 2017-A02673-50 (Other Identifier: ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

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