G71.0 D69.1
Conditions
Interventions
Group 1: RETROSPECTIVE PART: recording of concurrent medication, status of ambulation, occurence of thromboembolic events, occurence of bleeding tendencies
PROSPECTIVE PART: determination of FVIII-ac
Sponsors
Universitätsklinikum Freiburg- Zentrum für Kinder- und JugendmedizinKlinik für Neuropädiatrie und Muskelerkrankungen
Eligibility
Sex/Gender
Male
Age
6 Years to No maximum
Inclusion criteria
Inclusion criteria: RETROSPECTIVE PART Inclusion criteria: -registration in the DMD patient registry of the TREAT-NMD-network PROSPECTIVE PART Inclusion criteria: -male patients, aged 6 and above -assured DMD diagnosis, confirmed by genetic results or muscle biopsy -written approval of the person having the custody of the child -written approval of the patient as far as he is by age and stage of development able to understand the character, significance and bearing of the research project and is able to declare hereafter his intention -sufficient ability for cooperation in echocardiographic examination (during routine examination)
Exclusion criteria
Exclusion criteria: RETROSPECTIVE PART: Exclusion criteria: -none PROSPECTIVE PART: Exclusion criteria: -overweight with Body Mass Index > 30 body weight/m² -anamnesis or any hint of an intracranial and/or malignant tumor -known or persistent medical drug abuse, illegal drug abuse or alcohol abuse
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| RETROSPECTIVE PART: Primary Outcome: Documentation of clinically relevant thromboembolic events in patients registered in the DMD-Registry. PROSPECTIVE PART: Primary Outcome: Comprehensive examination of platelet function in DMD-patients via exmination of - vWF-Antigen - vWF-Collagenbindingcapacity (vWF:CB) - Multimer structural analysis - aggregation of thrombocytes - flow cytometry of thrombocytes | — |
Secondary
| Measure | Time frame |
|---|---|
| RETROSPECTIVE PART: Secondary Outcome: Documentation of general medication and ambulatory status in DMD-patients. PROSPECTIVE PART: Secondary Outcome Examination of - Factor VIII-activity - haemogramm - D-Dimers - Prothrombinfragments 1+2 - C-reactive Protein - Markers of Thrombosis Documentation of clinical course and medicaments - Echocardiography (when planned in routine-visit). | — |
Countries
Germany
Contacts
Public ContactDavid Schorling
Zentrum für Kinder- und Jugendmedizin - Klinik für Neuropädiatrie und Muskelerkrankungen.
Outcome results
None listed