Assessment of Burden Disease in Patients With Mast Cell Disorders (MCD&BuDi)

Assessment of Personal, Psychosocial, Work, and Economic Burden in Patients With Mast Cell Disorders,

The symptoms caused by mast cell disorders can have a significant impact on the state of health of individuals, constituting a real burden for them, and consequently altering their quality of life. It therefore seems important to clarify the impact on the quality of life, on the psycho-affective sphere, on professional life and on the direct and indirect costs caused by the disease, as well as on the "patient's remaining burden". It seems possible by a longitudinal study (patient follow-up over 1 year).

Primary objective is Assessment of quality of life in adult patient with mast cell diseases at M0.

Study Overview

• Status: Unknown
• Conditions: Mastocytosis; Cutaneous
• Intervention / Treatment: Behavioral: Score SMI

Detailed Description

Mast cell activation symptoms are due to the release of mast cell mediators after uncontrolled activation of mast cells. The release by mast cells of mast cell mediators (tryptase, histamine, prostaglandins, serotonin) results in mast cell activation symptoms, found in mast cell activation syndrome but also in mastocytosis. These symptoms interested the skin, gastrointestinal gut, pulmonary, cardiovascular and neuropsychiatric organs, etc.

In mastocytosis, along with these symptoms of mast cell activation, the infiltration of different organs by abnormal mast cells produces many clinical signs related to the excess of monoclonal mast cells present in the organs.

According to our clinical experience, the impact of mast cell disorder on various dimensions of life, including economic life, seems important, but it has never accurately evaluated.

• Study Type: Observational
• Enrollment (Anticipated): 200

Contacts and Locations

• Study Contact: Name: LIVIDEANU Cristina, MD; Phone Number: +33 0567778135; Email: livideanu.c@chu-toulouse.fr
• Study Contact Backup: Name: NEGRETTO Mathilde; Phone Number: +33 05 67 77 81 68; Email: negretto.m@chu-toulouse.fr

Study Locations

• France
  • Toulouse, France
    • Recruiting
    • Cristina BULAI LIVIDEANU
    • Contact: Cristina BULAI LIVIDEANU, MD; Phone Number: +33 567778135; Email: livideanu.c@chut-oulouse.fr
    • Contact: Isabelle OLIVIER, PhD; Phone Number: +33 0561777051; Email: olivier.i@chut-oulouse.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Prospective, descriptive study, both transversal and longitudinal, open, monocentric, performed in 200 patients with mast cell disease and follow-up at the CEREMAST Toulouse

Description

Inclusion Criteria:

• Adult patient (> 18 years old) with confirmed mast cell disorder according to international criteria
• Patient affiliated to social security regime

Exclusion Criteria:

• Patient under legal protection (guardianship, curators or court order)
• Patient does not speak French

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
face-to-face patients; During this visit, the investigator will complete the SMI score.	Behavioral: Score SMI; the investigator will complete the SMI score
Email patients; this visit at M0 + 7d will correspond to the emailing of the Mc_QoL and Burden_MCD questionnaires completed by the patient.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Quality of life of mast cell disorder patients	Quality of life is evaluated with the WHOQOL-bref questionary. This validated questionnaire in French assesses six dimensions of quality of life.	Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mast cell disorder patient's life quality	Quality of life is evaluated with the WHOQOL-bref.	6 months
Mast cell disorder patient's quality of life	Quality of life is evaluated with the WHOQOL-bref.	12 months

Collaborators and Investigators

• Sponsor: University Hospital, Toulouse
• Investigators: Principal Investigator: LIVIDEANU Cristina, MD, University Hospital, Toulouse

Study record dates

Study Major Dates

• Study Start (Actual): January 28, 2020
• Primary Completion (Anticipated): January 28, 2021
• Study Completion (Anticipated): September 15, 2022

Study Registration Dates

• First Submitted: June 16, 2020
• First Submitted That Met QC Criteria: November 3, 2020
• First Posted (Actual): November 4, 2020

Study Record Updates

• Last Update Posted (Actual): November 4, 2020
• Last Update Submitted That Met QC Criteria: November 3, 2020
• Last Verified: November 1, 2020

More Information

Terms related to this study

• Keywords: quality of life; Burden; mast cell disease; work impairment
• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Hypersensitivity; Neoplasms, Connective Tissue; Immune Complex Diseases; Mastocytosis
• Other Study ID Numbers: RC31/19/0354

Drug and device information, study documents

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