Other Constitutional Bleeding Disorders, Severe Forms of Hemophilia
Conditions
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.
Interventions
BEHAVIORALEvaluation of psychological and psychopathological
Sponsors
Assistance Publique Hopitaux De Marseille
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 12 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed | 2 years | Treatment Observance |
Secondary
| 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 |
Countries
France
Outcome results
None listed