Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast

Study Overview
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Brief Summary
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Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast

Detailed Description
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Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast. Duration of the study: 1 year after initiation of prophylaxis with Emicizumab Inclusion criteria: boys aged > 2 years, affected with severe hemophilia A with or without inhibitors Evaluated outcomes: annual bleeding rate, days of absence from school, the need for additional facteur VIII consumption, satisfaction about the treatment and quality of life. Tools used to assess quality of life are the Ivoirian version of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version 2 (CHO-KLAT) and EQ-5D The satisfaction of the parents will be assessed using the patients' global impression of change scale (PGIC).

Official Title
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Impact of Implementation of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Prophylaxis with Emicizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: severe hemophilia A with or without inhibitors - Exclusion Criteria: parents refusal -

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: outcomes of prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ivoirian Boys with severe Hemophilia A treated with Emicizumab<br>All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab | Drug: Prophylaxis with Emicizumab<br>* Prophylaxis with Emicizumab in Ivoirian boys with severe hemophilia A<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in annual bleeding rate | Using the patients' logbook | change in annual bleeding rate from baseline at 6 months and 12 months | 
| Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab | Using the CHO-KLAT and EQ-5D. The scales range from 0 to 100 with 0 representing the worst quality of life and 100 the best quality of life score | Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D at 6 months. | 
| Satisfaction of the parents about the treatment with Emicizumab after 6 month | Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied) | Assessment at 6 months after initiation of Emicizumab |