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

CompletedN/A

Sponsor: Montefiore Medical Center

Conditions: Hemophilia A

Interventions: HEMLIBRA

Countries: United States

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.

Eligibility overview

Sex: ALL

Age: — to 36 Months

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

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.

Locations (10)

Hartford, Connecticut, United States
New Haven, Connecticut, United States
Lebanon, New Hampshire, United States
Newark, New Jersey, United States
Buffalo, New York, United States
New York, New York, United States
Rochester, New York, United States
Staten Island, New York, United States
Syracuse, New York, United States
The Bronx, New York, United States