Hemophilia A
Conditions
Brief summary
Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity \< 1%, bleeding may frequently occur spontaneously, most commonly in joints, leading to painful hemophilic arthropathy and loss of joint function. Patients with moderate or mild hemophilia A, defined as FVIII clotting activity between 1-\<5% and 5-40% respectively, are less likely to have spontaneous bleeding however can have significant bleeding with trauma or surgery. Perioperative management by a hematologist who specializes in hemophilia is needed to ensure hemostasis during surgery. Hemophilia is an X-linked recessive disorder affecting 1 in 5000 to 10,000 males.
Detailed description
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 has been developed. Data on any pre-, peri and post-surgical practices while on emicizumab is being collected. Pediatricians are being asked if there are plans to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when this is planned on being done. The hope is 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. 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. 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.
Interventions
DRUGHEMLIBRA
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.
Sponsors
Genentech, Inc.
Regional Comprehensive Hemophilia Treatment Center (Mount Sinai)
Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center
Rutgers Robert Wood Johnson Medical School
Montefiore Medical Center
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 36 Months
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The proportion of patients that are being treated with emicizumab | Within a year. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Patients who receive additional doses of factor concentrate while on emicizumab | Within a year. | Data collected will also include proportion of patients who receive additional doses of factor concentrate while on emicizumab for trauma or surgical interventions. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data | Within a year. | Additionally, we will characterize the 37+ patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data (indication for emicizumab ie. primary vs secondary prophylaxis. |
Countries
United States
Outcome results
None listed