Clinical trial NCT02834130: Input of a Multidisciplinary Device to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Severe Forms of Hemophilia
Other Constitutional Bleeding Disorders
Lead sponsor: Assistance Publique Hopitaux De Marseille
Sponsor class: OTHER
First posted: 2016-07-15
Enrollment: 30
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.

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

Primary outcomes:
measure: Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed
timeFrame: 2 years
Secondary outcomes:
measure: Assessment of patient's behavioral condition using the Child Behavior Check-list (CBCL) 1,5-5
timeFrame: 2 Years
measure: Assessment of the patient's body consciousness through the "CORP-R" test (french test of body mapping)
timeFrame: 2 years
measure: Assessment of patient's quality of life using the "Kidscreen" questionnaire for children
timeFrame: 2 years
measure: Assessment of the patient's parents' Qaulity of life through the Short Form Health Survey (SF-36)
timeFrame: 2 Years
Other outcomes:
Locations:
city: Marseille
country: France
facility: Hopital Enfants de la Timone Assistance Publique Hôpitaux de Marseille
Study officials:
affiliation: Assistance Publique Hôpitaux de Marseille
name: Urielle DESALBRES
role: STUDY_DIRECTOR
References: