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Emicizumab in Children with Severe Hemophilia A
Usha Rani Thota1, Sreelatha Martha1, Chaitanya Jyothi Ravula1
1Department of Pediatrics, Institute of Child Health - Niloufer Hospital for Women and Children, Osmania Medical College, Hyderabad, Telangana, India.
Insights
Emicizumab prophylaxis significantly reduced bleeding events in children with severe hemophilia A (HA). This novel non-factor replacement therapy demonstrated high effectiveness and safety, improving patient quality of life.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Hemophilia A (HA) is a rare bleeding disorder characterized by a deficiency in clotting factor VIII.
- Current treatments for HA include factor replacement therapy and immune tolerance induction, which can be burdensome and less effective in some patients.
- Emicizumab represents a novel therapeutic approach as a non-factor replacement agent for HA management.
Purpose of the Study:
- To evaluate the efficacy and safety of emicizumab prophylaxis in pediatric patients with severe hemophilia A.
- To assess the impact of emicizumab on bleeding event rates and joint health.
- To determine the tolerability and adverse event profile of emicizumab in this population.
Main Methods:
- An observational, pre-post study design was employed.
- Eighteen pediatric patients (1 month to 12 years) with severe HA, with or without factor VIII inhibitors, were enrolled.
- Patients transitioned from previous therapies to emicizumab prophylaxis and were followed for 52 weeks.
Main Results:
- The mean annualized bleeding rate decreased dramatically from 12.7 events before emicizumab to 0.28 events after prophylaxis (p < 0.001).
- 72.2% of patients experienced no bleeding events while on emicizumab prophylaxis.
- Mean annualized joint bleeding rate significantly reduced, with 100% target joint resolution and no reported adverse events.
Conclusions:
- Emicizumab is an effective and safe prophylactic treatment for severe hemophilia A, including in patients with factor VIII inhibitors.
- Emicizumab prophylaxis offers a promising strategy to optimize treatment outcomes and enhance the quality of life for children with severe HA.
Objectives:
To assess the effectiveness and tolerability of emicizumab prophylaxis in hemophilia A (HA). Emicizumab is a novel therapeutic drug which is the first and only non-factor replacement agent licensed for use in people with HA.
Methods:
Pediatric patients aged 1 mo to 12 y with severe HA and frequent / life threatening bleeding events, with or without coagulation protein factor VIII inhibitors were enrolled (n = 18) in this observational pre-post study. Patients were switched from therapy involving on-demand or prophylactic factor VIII/bypassing agents/immune tolerance induction to emicizumab prophylaxis and followed up for 52 wk.
Results:
One year before initiating emicizumab, a total of 229 bleeding events occurred among the enrolled children. After emicizumab prophylaxis, 5 patients had one episode of bleeding event each with a mean bleeding duration of 1.2 d in one year. The mean annualized bleeding rate significantly reduced from 12.7 ± 8.61 events pre-emicizumab prophylaxis to 0.28 ± 0.46 events post-emicizumab prophylaxis (p < 0.001). Out of the total cohort (n = 18), 72.2% of patients (n = 13) had no bleeding events (95% Confidence interval: 46.4-89.3) while on emicizumab. The mean annualized joint bleeding rate reduced from 9.72 ± 7.44 to 0.17 ± 0.38 (p < 0.001). The target joint resolution was 100% and no adverse events were noted.
Conclusions:
Emicizumab was found to be effective and safe as a prophylactic agent for the treatment of severe HA with and without factor VIII inhibitors. Emicizumab prophylaxis can optimize treatment outcomes and promote a better quality of life in children with severe HA.
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