Other Constitutional Bleeding Disorders Clinical Trial
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
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.
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.
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Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care