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

NCT ID: NCT02834130

Last Updated: 2016-07-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-04-30

Brief Summary

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.

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.

Conditions

Severe Forms of Hemophilia; Other Constitutional Bleeding Disorders

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

children from 2 to 10 years with haemophilia or allied HBD, wh

Group Type: OTHER

Evaluation of psychological and psychopathological

Intervention Type: BEHAVIORAL

Interventions

Evaluation of psychological and psychopathological

Intervention Type: BEHAVIORAL

Other Intervention Names

Evaluation of quality of life

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique Hopitaux De Marseille

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Urielle DESALBRES

Role: STUDY_DIRECTOR

Assistance Publique Hôpitaux de Marseille

Locations

Hopital Enfants de la Timone Assistance Publique Hôpitaux de Marseille

Marseille, , France

Countries

France

Other Identifiers

2014-A00367-40

Identifier Type: REGISTRY

Identifier Source: secondary_id

2014-05

Identifier Type: -

Identifier Source: org_study_id