Input of a Multidisciplinary Device to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families (ACADHEM)

Input of a Multidisciplinary Device Including a Psychological Approach, to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families

Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection.

The investigators propose to set up a single-centre, descriptive, transversal pilot study, designed for the inclusion of around thirty children from 2 to 10 years, with haemophilia or allied hereditary bleeding disorders (HBD), who were referred to the Hemophilia Treatment Center (HTC) of Marseille at diagnosis. For those children who were able to benefit from the totality or part of the device accompanying the diagnostic announcement in the past 10 years.

Study Overview

• Status: Completed
• Conditions: Severe Forms of Hemophilia; Other Constitutional Bleeding Disorders
• Intervention / Treatment: Behavioral: Evaluation of psychological and psychopathological

Detailed Description

Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection. The recurrence of joint bleedings is responsive of multifocal arthropathy in the natural history, but nowadays, the orthopedic outcome is good thanks to prophylactic treatments.

However, the announcement of the diagnosis of such hereditary bleeding disorders at a very young age represents a real psychological trauma for parents. Such an announcement may induce some psycho-developmental impairment.

An innovative support has been implemented at the Hemophilia Treatment Center in Marseille, in conjunction with the French Hemophilia Society. This program is based on a multidisciplinary partnership that favors the psychological support and the reinsurance of the parents.

With Rare Diseases Foundation support, the investigators have initiated this pilot study that will include more than 20 patients with severe hemophilia or constitutional bleeding disorder, who benefit from the device. This descriptive, monocentric, interventional study will permit to describe the psychological status, the quality of life of patients and their parents, but also to evaluate the impact of the device on initiation and observance of treatments like Long Term Prophylaxis.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Marseille, France, 13354
    • Hopital Enfants de la Timone Assistance Publique Hôpitaux de Marseille

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients aged between 3 and 12 years
• Patients diagnosed and treated at the Center until inclusion
• Patients with FVIII :C < 2% or with FIX :C <2% or with FVII:C <2% or with FXIII:C <2% or fibrinogen <0,2 g/l, or with type 3 von Willebrand diseases or Thrombasthenia of Glanzmann diseases.
• Patient who participate to multidisciplinary support and announcement of diagnosis device
• Voluntarily given fully informed written and signed consents obtained before any study-related procedures are conducted

Exclusion Criteria:

• Patient with other clotting disorder or disease.
• Patient diagnosed or treated in other centre
• Patient with psychiatric disorder or disease
• Patient with other severe chronic disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• 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: children from 2 to 10 years with haemophilia or allied HBD, wh	Behavioral: Evaluation of psychological and psychopathological; Other Names: Evaluation of quality of life

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed	Treatment Observance	2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Assessment of patient's behavioral condition using the Child Behavior Check-list (CBCL) 1,5-5	2 Years
Assessment of the patient's body consciousness through the "CORP-R" test (french test of body mapping)	2 years
Assessment of patient's quality of life using the "Kidscreen" questionnaire for children	2 years
Assessment of the patient's parents' Qaulity of life through the Short Form Health Survey (SF-36)	2 Years

Collaborators and Investigators

• Sponsor: Assistance Publique Hopitaux De Marseille

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): April 1, 2016

Study Registration Dates

• First Submitted: October 21, 2015
• First Submitted That Met QC Criteria: July 12, 2016
• First Posted (Estimate): July 15, 2016

Study Record Updates

• Last Update Posted (Estimate): July 19, 2016
• Last Update Submitted That Met QC Criteria: July 18, 2016
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Hematologic Diseases; Hemostatic Disorders; Blood Coagulation Disorders; Hemorrhagic Disorders
• Other Study ID Numbers: 2014-05; 2014-A00367-40 (Registry Identifier: Ansm)