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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: HEMLIBRA

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.

• Study Type: Observational
• Enrollment (Actual): 71

Contacts and Locations

Study Locations

• United States
  • Connecticut
    • Hartford, Connecticut, United States, 06106
      • Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center
    • New Haven, Connecticut, United States, 06510
      • Yale Hemophilia Treatment Center
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03766
      • Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital
  • New Jersey
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel
  • New York
    • Buffalo, New York, United States, 14202
      • Western NY Blood Care - Research Foundation for SUNY
    • New York, New York, United States, 10065
      • Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)
    • Rochester, New York, United States, 14621
      • Mary M Gooley Hemophilia Center, Inc.
    • Staten Island, New York, United States, 11030
      • Northwell Health Hemostasis and Thrombosis Center
    • Syracuse, New York, United States, 13210
      • SUNY Upstate Hemophilia Treatment Center - HTC 058
    • The Bronx, New York, United States, 10467
      • Children's Hospital at Montefiore

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 3 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients 0 to 36 months of age, diagnosed with severe, moderate or mild Hemophlia A at the time of initiating treatment with Emicizumab.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

What is the study measuring?

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.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data	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.	Within a year.

Collaborators and Investigators

• Sponsor: Montefiore Medical Center
• Collaborators: Genentech, Inc.; Rutgers Robert Wood Johnson Medical School; Regional Comprehensive Hemophilia Treatment Center (Mount Sinai); Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center
• Investigators: Principal Investigator: Jennifer Davila, MD, Children's Hospital at Montefiore

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2024
• Primary Completion (Actual): July 18, 2025
• Study Completion (Actual): July 18, 2025

Study Registration Dates

• First Submitted: February 10, 2022
• First Submitted That Met QC Criteria: February 10, 2022
• First Posted (Actual): February 21, 2022

Study Record Updates

• Last Update Posted (Estimated): September 8, 2025
• Last Update Submitted That Met QC Criteria: September 2, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; emicizumab
• Other Study ID Numbers: 2021-13264; ML43506 (Other Identifier: Sponsor)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No