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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-A00367-40 | Registry Identifier | Ansm |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| children from 2 to 10 years with haemophilia or allied HBD, wh | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Evaluation of psychological and psychopathological | Behavioral |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed | Treatment Observance | 2 years |
| Measure | Description | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Urielle DESALBRES | Assistance Publique Hôpitaux de Marseille | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hopital Enfants de la Timone Assistance Publique Hôpitaux de Marseille | Marseille | 13354 | France |
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| 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 |