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

Not Applicable

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Prophylaxis with Emicizumab

• Registration Number: NCT05279924
• Lead Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Brief Summary

Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast

Detailed Description

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).

Study Timeline

• Study Start: 2021-07-01
• Study First Submitted: 2022-02-25
• Study First Submitted QC: 2022-03-05
• Study First Posted: 2022-03-15
• Status Verified: 2022-10
• Primary Completion: 2022-10-20
• Study Completion: 2022-10-20
• Last Update Submitted: 2022-10-24
• Last Update Posted: 2022-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 33

Inclusion Criteria

severe hemophilia A with or without inhibitors -

Exclusion Criteria

parents refusal

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ivoirian Boys with severe Hemophilia A treated with Emicizumab	Prophylaxis with Emicizumab	All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab

Primary Outcome Measures

Name	Time	Method
Change in annual bleeding rate	change in annual bleeding rate from baseline at 6 months and 12 months	Using the patients' logbook
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab	Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D at 6 months.	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
Satisfaction of the parents about the treatment with Emicizumab after 6 month	Assessment at 6 months after initiation of Emicizumab	Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied)

Trial Locations

Locations (1)

CHU de Yopougon; 🇨🇮 Abidjan, Côte D'Ivoire