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

Description

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.

Conditions

Hemophilia A

Study Overview

Study Details

Study overview

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.

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

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

Condition
Hemophilia A
Intervention / Treatment

-

Contacts and Locations

Hartford

Connecticut Children's Hemophilia Treatment Center, Hartford, Connecticut, United States, 06106

New Haven

Yale Hemophilia Treatment Center, New Haven, Connecticut, United States, 06510

Boston

Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center, Boston, Massachusetts, United States, 02114

Lebanon

Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital, Lebanon, New Hampshire, United States, 03766

New Brunswick

Rutgers Hemophilia Treatment Center, New Brunswick, New Jersey, United States, 08901

Newark

Newark Beth Israel, Newark, New Jersey, United States, 07112

Bronx

Children's Hospital at Montefiore, Bronx, New York, United States, 10467

Buffalo

Western NY Blood Care - Research Foundation for SUNY, Buffalo, New York, United States, 14202

New York

Regional Comprehensive Hemophilia Treatment Center (Mount Sinai), New York, New York, United States, 10029

New York

Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell), New York, New York, United States, 10065

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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
  • * Patients with acquired Hemophilia A
  • * Patients with Hemophilia A and another congenital or acquired bleeding disorder.

Ages Eligible for Study

to 36 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Montefiore Medical Center,

Jennifer Davila, MD, PRINCIPAL_INVESTIGATOR, Children's Hospital at Montefiore

Study Record Dates

2025-08