Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

Study Overview
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Brief Summary
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We have developed a questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

Detailed Description
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This will be a multi-institutional, retrospective review of pediatric patients ages 0 to 36 months of age who are currently receiving and/or have received emicizumab-kxwh as part of their treatment for hemophilia A with or without inhibitors. Participating sites are part of the New England Region (plus New Jersey and New York-Region II) of hemophilia treatment centers. Currently 11 out of the 23 regional centers have committed to collaborating on this study (Appendix A). Each institution will be contributing subjects who have been treated or who are currently being treated with emicizumab, from October 4th, 2018 up to the point of IRB approval. Additionally, each institution will determine the best way to identify eligible patients and keep track of patients enrolled in the study. The Children's Hospital at Montefiore will be the coordinating center.

Official Title
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Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: HEMLIBRA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must meet the following criteria for study entry: Patients who have been prescribed Emicizumab Patients who are 0-36 months of age at the time of starting treatment with Emicizumab Diagnosis of congenital mild, moderate or severe hemophilia with or without an inhibitor Exclusion Criteria: Patients with acquired Hemophilia A Patients with Hemophilia A and another congenital or acquired bleeding disorder.

Ages Eligible for Study
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Minimum Age: 0 Months
Maximum Age: 36 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: HEMLIBRA|Emicizumab (Hemlibra, also known as ACE910 and RO5534262) is a humanized monoclonal modified immunoglobulin G4 (IgG4) antibody with a bispecific antibody structure produced by recombinant DNA technology in Chinese hamster ovary (CHO) cells.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The proportion of patients that are being treated with emicizumab | The primary outcome will include the proportion of patients that are being treated with emicizumab for primary prophylaxis, as well as identifying breakthrough bleeding while on emicizumab. | Within a year. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patients who receive additional doses of factor concentrate while on emicizumab | Data collected will also include proportion of patients who receive additional doses of factor concentrate while on emicizumab for trauma or surgical interventions. | Within a year. |