Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate
Study Details
- Unmapped
- Drug: Tranexamic Acid
- Drug: Von Willebrand Factor-Containing Concentrates
- Age 0-18 years.
- Residents of Assiut Governorate or receiving care at Assiut University Children's Hospital.
- Suspected or confirmed von Willebrand disease (VWD) based on clinical bleeding symptoms or referral for evaluation.
- Patients diagnosed with VWD using standard laboratory tests, including:
- VWF antigen (VWF:Ag).
- VWF ristocetin cofactor activity (VWF:RCo).
- Factor VIII activity.
- Other inherited bleeding disorders, such as:
- Hemophilia A or B.
- Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).
- Platelet function disorders.
- Acquired bleeding disorders, including:
- Liver disease.
- Renal insufficiency.
- Vitamin K deficiency.
- Disseminated intravascular coagulation (DIC).
- Use of medications that may interfere with coagulation testing (e.g., anticoagulants, antiplatelet drugs).
- Incomplete clinical or laboratory data (for retrospective cases).
- Refusal of consent for participation (for prospective cases).
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.